Geographic Atrophy
Conditions
Keywords
Geographic Atrophy, GA, Danicopan, ALXN2040, Complement Inhibition, AMD
Brief summary
This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD. The study consists of a Screening Period of up to 6 weeks, a 104-week masked Treatment Period, followed by a 30-day Follow-up after the last dose. This study will have 4 treatments arms: 100 milligrams (mg) twice daily (bid), 200 mg bid, 400 mg once daily (qd), and matching placebo.
Interventions
DRUGDanicopan
Oral tablet.
DRUGPlacebo
Oral tablet.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Masking of treatment allocation will be observed until Week 104.
Eligibility
Sex/Gender
ALL
Age
60 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Vaccination for Neisseria meningitidis. * Capable of giving signed informed consent. * Presentation of GA secondary to AMD in at least 1 eye * The entire GA lesion must be \> 1 μm outside of the foveal center Key
Exclusion criteria
* GA in the study eye due to cause other than AMD. * Have previously received intravitreal anti-vascular endothelial growth factor injections in study eye for intraocular vascular disease. * Have previously received any stem cell/gene therapy for any ophthalmological condition in either eye. * Use of any investigational medicinal product (ie, participation in interventional clinical studies for any ophthalmic indications) or use of any regulatory approved treatment for GA in the study eye regardless of route of administration within the last 3 months or 5 half-lives of the last dose of the investigational or commercial product (whichever is longer). * Presence of active ocular diseases in the study eye that in the opinion of the Investigator compromises or confounds visual function or interferes with study assessments. * Known or suspected complement deficiency. * History or presence of any medical or psychological condition that, in the opinion of the Principal Investigator, would make the patient inappropriate for the study. * Hypersensitivity to fluorescein sodium for injection, the investigational drug (danicopan) or any of its excipients. Note: Additional inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF) | Baseline, Week 52 | FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area measurement. The total GA lesion area (square millimeter \[mm\^2\]) was transformed into sqrt (millimeter \[mm\]). Greater area affected means a worse outcome than smaller area affected. The rate of change from Baseline (mm/year) for each treatment group was estimated based on the coefficients for time and the interaction of time and treatment from the mixed models for repeated measures (MMRM). MMRM analysis included the change from Baseline at post-baseline visit in the sqrt GA lesion area as dependent variable. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Baseline, Week 52 and Week 104 | FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area (mm\^2) measurement. Greater area affected means a worse outcome than smaller area affected. |
| Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Baseline, Week 52 and Week 104 | Photoreceptor loss was assessed by measuring the percent of macular EZ total attenuation using SD-OCT. The EZ was considered to be formed mainly by mitochondria within the ellipsoid layer of the outer portion of the inner segments of the photoreceptors. In SD-OCT, decreases in the integrity and intensity of the EZ have been correlated with the reduction in cone photoreceptors and retinal sensitivity in retinal degenerative diseases. Lower increase in percentage means a better outcome. Higher increase in percentage means a worse outcome. |
| Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | Photoreceptor degradation was assessed by measuring the percent of macular EZ partial attenuation using SD-OCT. The EZ was considered to be formed mainly by mitochondria within the ellipsoid layer of the outer portion of the inner segments of the photoreceptors. In SD-OCT, decreases in the integrity and intensity of the EZ have been correlated with the reduction in cone photoreceptors and retinal sensitivity in retinal degenerative diseases. Lower increase in percentage means a better outcome. Higher increase in percentage means a worse outcome. |
| Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The ONL - RPE mean central 1 mm subfield thickness refers to the average thickness of the retinal layers between the ONL and RPE measured within a 1 mm radius from the foveal center (the central point of the retina). |
| Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The ONL - RPE mean central macular 2 mm subfield thickness refers to the thickness of the retina in the central part of the macula, specifically within a 2 mm radius. |
| Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The EZ - RPE macular volume refers to the volume of the retinal tissue between the EZ and the RPE, specifically within the macular region. Changes in EZ-RPE volume indicate the presence or progression of diseases affecting the outer retina. |
| Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The EZ - RPE mean central 1 mm subfield thickness is a measurement of the thickness of the ellipsoid zone, a layer within the retina, in the central 1 mm region. Changes in EZ-RPE thickness indicate damage or dysfunction within the photoreceptors, which is associated with various eye diseases like AMD. |
| Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The EZ - RPE mean central macular 2 mm thickness measurement reflects the distance between the outer border of the EZ and the inner border of the RPE layer, typically within a central 2 mm area. Decreased EZ-RPE thickness is an early indicator of disease, such as in dry AMD, where a thinning or loss of the EZ is a hallmark. Changes in EZ-RPE thickness is used to monitor treatment response in conditions like AMD. |
| Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The percentage of total attenuation of the EZ in the central 1 mm subfield is reported. |
| Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The percentage of partial attenuation of the EZ in the central 1 mm subfield is reported. |
| Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The percentage of total attenuation of the EZ in the central macular 2 mm subfield is reported. |
| Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The percentage of partial attenuation of the EZ in the central macular 2 mm subfield is reported. |
| Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | Sub-RPE compartment measures reflect the disease burden between the RPE and Bruch's membrane. The sub-RPE central 1 mm mean thickness refers to the average thickness of the retina within a 1-mm diameter circular area centered around the fovea (the central part of the retina). |
| Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | Baseline, Week 104 | FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area measurement. The total GA lesion area (mm\^2) was transformed into sqrt (mm). Greater area affected means a worse outcome than smaller area affected. |
| Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | Sub-RPE compartment measures reflect the disease burden between the RPE and Bruch's membrane. The panmacular sub-RPE volume refers to the total volume of fluid or space located underneath the RPE across the entire macula (the central part of the retina). |
| Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | Macular total RPE attenuation refers to the degree of thinning or loss of the RPE in the macula, the central part of the retina responsible for sharp, detailed vision. This was measured as a percentage of the macular area affected. |
| Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The percentage of total attenuation of the RPE in the central 1 mm subfield is reported. |
| Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The percentage of total attenuation of the RPE in the central macular 2 mm subfield is reported. |
| Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Baseline, Week 52 and Week 104 | ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome). |
| Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Baseline, Week 52 and Week 104 | LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. LLVA was reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters). The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Change from baseline: a negative score indicates decline in LLVA. |
| Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Baseline, Week 52 and Week 104 | LLD score was calculated as the difference between BCVA and LLVA (BCVA-LLVA) scores. BCVA and LLVA were reported as the number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters). The lower the number of letters read correctly on the eye chart, the worse the vision. LLD is a measure of visual acuity impairment in low-light conditions. A higher LLD score indicates a greater difference between vision in bright and dim light, suggesting a more significant loss of visual function in low-luminance settings. |
| Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Baseline, Week 52 and Week 104 | MNRead acuity cards were continuous-text reading-acuity cards suitable for measuring the reading acuity and reading speed of normal and low-vision participants. The MNRead acuity cards consisted of single, simple sentences with equal numbers of characters. A stopwatch was used to record time to a tenth of a second. Sentences that could not be read or were not attempted due to vision should be recorded as 0 for time and 10 for errors. The Radner Reading Cards were suitable for measuring reading speed, reading visual acuity, and critical print size. The reading test was stopped when the reading time was longer than 20 seconds or when the participant was making severe errors. A negative change from baseline indicates a decrease in the monocular reading speed; disease worsening. |
| Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Baseline, Week 52 and Week 104 | The NEI VFQ-25 instrument measures dimensions of self-reported vision-targeted health status of individuals with chronic eye conditions. The NEI VFQ-25 consists of 11 vision related domains: global vision rating, difficulty with near vision activities, difficulty with distance vision activities, limitations in social function related to vision, role limitations, dependency on others due to vision, mental health symptoms due to vision, driving difficulties, limitations with peripheral- and color-vision, and ocular pain. The NEI VFQ-25 also includes a single item measuring general health. A composite score averages the vision related domains and ranges from 0 (worse) to 100 (best). A higher score represents better functioning. A negative change from baseline indicates a decrease in the visual functioning; disease worsening. |
| Plasma Concentration of Danicopan | Day 1 and Week 52 | All values below the limit of quantification (\<0.1 nanograms \[ng\]/milliliter \[mL\]) were set to 0 for calculation of summary statistics. |
| Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity | Week 52 (pre-dose, 2 hours post-dose) | Serum AP functional activity was measured by the Wieslab functional immunoassay method. |
| Change From Baseline in Plasma Bb Fragment of Complement Factor B (Bb) Concentration | Baseline, Week 52 (pre-dose) | — |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Day 1 through Week 108 | An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, resulted in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), or an important medical event or reaction. A TEAE was defined as any AE that started during or after the first dose of study intervention. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | Sub-RPE compartment measures reflect the disease burden between the RPE and Bruch's membrane. The sub-RPE central macular 2 mm subfield thickness refers to the thickness of the retina within a 2-mm radius in the central part of the macula. |
Countries
Australia, Czechia, France, Germany, Hungary, Italy, Japan, Latvia, Slovakia, South Korea, Spain, United Kingdom, United States
Participant flow
Recruitment details
The study included 104-week Masked Treatment Period (Masked Placebo-controlled \[Primary Evaluation Period; up to 52 weeks\] and Masked Active Treatment \[Secondary Evaluation Period; Week 52 to Week 104\]). As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated.
Pre-assignment details
For standardization purposes, the primary and secondary efficacy analyses were measured in the pre-specified study eye per individual participant. In this study, ocular assessments were performed in both eyes at Screening. The eye that met all eligibility criteria was designated as the study eye and the other eye was used as the fellow eye. If both eyes were eligible, the right eye was designated as the study eye.
Participants by arm
| Arm | Count |
|---|---|
| Danicopan 100 mg BID Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period. | 93 |
| Danicopan 200 mg BID Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period. | 91 |
| Danicopan 400 mg QD Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period. | 91 |
| Placebo Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52. | 90 |
| Total | 365 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 | FG004 | FG005 | FG006 |
|---|---|---|---|---|---|---|---|---|
| Primary Evaluation Period (52 Weeks) | Adverse Event | 7 | 9 | 8 | 4 | 0 | 0 | 0 |
| Primary Evaluation Period (52 Weeks) | Death | 0 | 1 | 0 | 3 | 0 | 0 | 0 |
| Primary Evaluation Period (52 Weeks) | Investigator's Discretion | 1 | 1 | 0 | 2 | 0 | 0 | 0 |
| Primary Evaluation Period (52 Weeks) | Liver abnormalities meeting Hy's Law criteria | 1 | 0 | 0 | 0 | 0 | 0 | 0 |
| Primary Evaluation Period (52 Weeks) | Lost to Follow-up | 1 | 0 | 1 | 0 | 0 | 0 | 0 |
| Primary Evaluation Period (52 Weeks) | Other Than Specified | 3 | 0 | 3 | 2 | 0 | 0 | 0 |
| Primary Evaluation Period (52 Weeks) | Withdrawal by Subject | 7 | 13 | 6 | 9 | 0 | 0 | 0 |
| Secondary Evaluation Period (52 Weeks) | Adverse Event | 2 | 3 | 2 | 0 | 0 | 0 | 3 |
| Secondary Evaluation Period (52 Weeks) | Death | 0 | 1 | 1 | 0 | 1 | 0 | 0 |
| Secondary Evaluation Period (52 Weeks) | Investigator's Discretion | 1 | 2 | 1 | 0 | 0 | 0 | 1 |
| Secondary Evaluation Period (52 Weeks) | Lack of Efficacy | 0 | 1 | 2 | 0 | 0 | 0 | 0 |
| Secondary Evaluation Period (52 Weeks) | Liver abnormalities meeting Hy's Law criteria | 0 | 0 | 0 | 0 | 0 | 1 | 0 |
| Secondary Evaluation Period (52 Weeks) | Other Than Specified | 3 | 1 | 3 | 0 | 0 | 1 | 0 |
| Secondary Evaluation Period (52 Weeks) | Study Terminated by Sponsor | 49 | 45 | 53 | 0 | 13 | 17 | 14 |
| Secondary Evaluation Period (52 Weeks) | Withdrawal by Subject | 3 | 0 | 0 | 0 | 5 | 2 | 2 |
Baseline characteristics
| Characteristic | Danicopan 100 mg BID | Danicopan 200 mg BID | Danicopan 400 mg QD | Placebo | Total |
|---|---|---|---|---|---|
| Age, Continuous | 77.0 years STANDARD_DEVIATION 7.2 | 76.0 years STANDARD_DEVIATION 7.24 | 77.8 years STANDARD_DEVIATION 7.52 | 77.8 years STANDARD_DEVIATION 6.75 | 77.1 years STANDARD_DEVIATION 7.19 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 4 Participants | 1 Participants | 4 Participants | 6 Participants | 15 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 75 Participants | 81 Participants | 77 Participants | 75 Participants | 308 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 14 Participants | 9 Participants | 10 Participants | 9 Participants | 42 Participants |
| Race/Ethnicity, Customized Race Asian | 2 Participants | 1 Participants | 3 Participants | 3 Participants | 9 Participants |
| Race/Ethnicity, Customized Race Black or African American | 0 Participants | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized Race Not Reported | 11 Participants | 6 Participants | 6 Participants | 6 Participants | 29 Participants |
| Race/Ethnicity, Customized Race Other | 0 Participants | 0 Participants | 0 Participants | 2 Participants | 2 Participants |
| Race/Ethnicity, Customized Race Unknown | 3 Participants | 3 Participants | 4 Participants | 2 Participants | 12 Participants |
| Race/Ethnicity, Customized Race White | 77 Participants | 81 Participants | 78 Participants | 76 Participants | 312 Participants |
| Sex: Female, Male Female | 68 Participants | 58 Participants | 64 Participants | 61 Participants | 251 Participants |
| Sex: Female, Male Male | 25 Participants | 33 Participants | 27 Participants | 29 Participants | 114 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk | EG008 affected / at risk | EG009 affected / at risk |
|---|---|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 93 | 1 / 91 | 0 / 91 | 3 / 90 | 1 / 73 | 1 / 67 | 1 / 73 | 1 / 24 | 0 / 23 | 0 / 23 |
| other Total, other adverse events | 44 / 93 | 42 / 91 | 33 / 91 | 33 / 90 | 22 / 73 | 14 / 67 | 16 / 73 | 12 / 24 | 7 / 23 | 8 / 23 |
| serious Total, serious adverse events | 14 / 93 | 19 / 91 | 16 / 91 | 15 / 90 | 8 / 73 | 10 / 67 | 8 / 73 | 6 / 24 | 4 / 23 | 4 / 23 |
Outcome results
Primary
Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF)
FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area measurement. The total GA lesion area (square millimeter \[mm\^2\]) was transformed into sqrt (millimeter \[mm\]). Greater area affected means a worse outcome than smaller area affected. The rate of change from Baseline (mm/year) for each treatment group was estimated based on the coefficients for time and the interaction of time and treatment from the mixed models for repeated measures (MMRM). MMRM analysis included the change from Baseline at post-baseline visit in the sqrt GA lesion area as dependent variable.
Time frame: Baseline, Week 52
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) who had any post-baseline FAF GA lesion area data at the Week 52 analysis. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Danicopan 100 mg BID | Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF) | 0.465 mm/year | Standard Error 0.0289 |
| Danicopan 200 mg BID | Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF) | 0.391 mm/year | Standard Error 0.0306 |
| Danicopan 400 mg QD | Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF) | 0.443 mm/year | Standard Error 0.0301 |
| Placebo | Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF) | 0.406 mm/year | Standard Error 0.0304 |
p-value: 0.920590% CI: [-0.01, 0.126]Mixed Models Analysis
p-value: 0.920590% CI: [-0.086, 0.055]Mixed Models Analysis
p-value: 0.920590% CI: [-0.033, 0.106]Mixed Models Analysis
Secondary
Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity
Serum AP functional activity was measured by the Wieslab functional immunoassay method.
Time frame: Week 52 (pre-dose, 2 hours post-dose)
Population: Pharmacodynamic (PD) Analysis Set included all participants who received at least 1 dose (full or partial) of study drug and had at least 1 measurable PD value. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity | Change at Week 52: Predose | -5.9 percentage of activity | Standard Deviation 17.49 |
| Danicopan 100 mg BID | Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity | Change at Week 52: 2 Hour Postdose | -58.5 percentage of activity | Standard Deviation 27.85 |
| Danicopan 200 mg BID | Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity | Change at Week 52: 2 Hour Postdose | -75.3 percentage of activity | Standard Deviation 23.17 |
| Danicopan 200 mg BID | Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity | Change at Week 52: Predose | -21.3 percentage of activity | Standard Deviation 29.9 |
| Danicopan 400 mg QD | Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity | Change at Week 52: Predose | -8.2 percentage of activity | Standard Deviation 24.48 |
| Danicopan 400 mg QD | Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity | Change at Week 52: 2 Hour Postdose | -74.8 percentage of activity | Standard Deviation 28.82 |
| Placebo | Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity | Change at Week 52: Predose | 2.7 percentage of activity | Standard Deviation 17.12 |
| Placebo | Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity | Change at Week 52: 2 Hour Postdose | -23.1 percentage of activity | Standard Deviation 42.12 |
Secondary
Change From Baseline in Plasma Bb Fragment of Complement Factor B (Bb) Concentration
Time frame: Baseline, Week 52 (pre-dose)
Population: PD Analysis Set included all participants who received at least 1 dose (full or partial) of study drug and had at least 1 measurable PD value. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline in Plasma Bb Fragment of Complement Factor B (Bb) Concentration | -0.3 µg/mL | Standard Deviation 0.77 |
| Danicopan 200 mg BID | Change From Baseline in Plasma Bb Fragment of Complement Factor B (Bb) Concentration | -0.7 µg/mL | Standard Deviation 0.94 |
| Danicopan 400 mg QD | Change From Baseline in Plasma Bb Fragment of Complement Factor B (Bb) Concentration | -0.2 µg/mL | Standard Deviation 0.55 |
| Placebo | Change From Baseline in Plasma Bb Fragment of Complement Factor B (Bb) Concentration | -0.1 µg/mL | Standard Deviation 0.55 |
Secondary
Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF
FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area measurement. The total GA lesion area (mm\^2) was transformed into sqrt (mm). Greater area affected means a worse outcome than smaller area affected.
Time frame: Baseline, Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline visit and post-baseline (Week 104) visit FAF GA lesion area data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | 0.892 mm | Standard Deviation 0.4545 |
| Danicopan 200 mg BID | Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | 0.705 mm | Standard Deviation 0.3831 |
| Danicopan 400 mg QD | Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | 0.859 mm | Standard Deviation 0.3468 |
| Placebo | Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | 0.668 mm | Standard Deviation 0.4866 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | 0.585 mm | Standard Deviation 0.1396 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | 0.787 mm | Standard Deviation 0.1858 |
Secondary
Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT
The EZ - RPE macular volume refers to the volume of the retinal tissue between the EZ and the RPE, specifically within the macular region. Changes in EZ-RPE volume indicate the presence or progression of diseases affecting the outer retina.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit EZ-RPE macular volume data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.16 cubic millimeters (mm^3) | Standard Deviation 0.097 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.08 cubic millimeters (mm^3) | Standard Deviation 0.062 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.18 cubic millimeters (mm^3) | Standard Deviation 0.066 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.09 cubic millimeters (mm^3) | Standard Deviation 0.074 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.15 cubic millimeters (mm^3) | Standard Deviation 0.115 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.29 cubic millimeters (mm^3) | Standard Deviation 0.17 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.10 cubic millimeters (mm^3) | Standard Deviation 0.098 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.05 cubic millimeters (mm^3) | Standard Deviation 0.047 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.11 cubic millimeters (mm^3) | Standard Deviation 0.056 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.10 cubic millimeters (mm^3) | Standard Deviation 0.069 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.17 cubic millimeters (mm^3) | Standard Deviation 0.143 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.07 cubic millimeters (mm^3) | Standard Deviation 0.047 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.16 cubic millimeters (mm^3) | Standard Deviation 0.089 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.32 cubic millimeters (mm^3) | Standard Deviation 0.174 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.08 cubic millimeters (mm^3) | Standard Deviation 0.059 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.16 cubic millimeters (mm^3) | Standard Deviation 0.122 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.09 cubic millimeters (mm^3) | Standard Deviation 0.048 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.16 cubic millimeters (mm^3) | Standard Deviation 0.089 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.13 cubic millimeters (mm^3) | Standard Deviation 0.098 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.06 cubic millimeters (mm^3) | Standard Deviation 0.063 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.07 cubic millimeters (mm^3) | Standard Deviation 0.052 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.18 cubic millimeters (mm^3) | Standard Deviation 0.135 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.05 cubic millimeters (mm^3) | Standard Deviation 0.063 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.10 cubic millimeters (mm^3) | Standard Deviation 0.082 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.10 cubic millimeters (mm^3) | Standard Deviation 0.064 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.06 cubic millimeters (mm^3) | Standard Deviation 0.052 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.11 cubic millimeters (mm^3) | Standard Deviation 0.103 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.13 cubic millimeters (mm^3) | Standard Deviation 0.107 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.06 cubic millimeters (mm^3) | Standard Deviation 0.058 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.13 cubic millimeters (mm^3) | Standard Deviation 0.075 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.23 cubic millimeters (mm^3) | Standard Deviation 0.133 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.08 cubic millimeters (mm^3) | Standard Deviation 0.052 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.12 cubic millimeters (mm^3) | Standard Deviation 0.082 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.15 cubic millimeters (mm^3) | Standard Deviation 0.106 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.12 cubic millimeters (mm^3) | Standard Deviation 0.068 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.08 cubic millimeters (mm^3) | Standard Deviation 0.053 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.08 cubic millimeters (mm^3) | Standard Deviation 0.069 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.16 cubic millimeters (mm^3) | Standard Deviation 0.037 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.28 cubic millimeters (mm^3) | Standard Deviation 0.069 |
Secondary
Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT
The EZ - RPE mean central 1 mm subfield thickness is a measurement of the thickness of the ellipsoid zone, a layer within the retina, in the central 1 mm region. Changes in EZ-RPE thickness indicate damage or dysfunction within the photoreceptors, which is associated with various eye diseases like AMD.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit EZ-RPE mean central 1 mm subfield thickness data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below= number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -5.23 µm | Standard Deviation 4.879 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -6.03 µm | Standard Deviation 6.163 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -4.03 µm | Standard Deviation 3.888 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -8.32 µm | Standard Deviation 4.05 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -2.51 µm | Standard Deviation 4.076 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -11.41 µm | Standard Deviation 8.175 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -5.70 µm | Standard Deviation 5.327 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -8.46 µm | Standard Deviation 5.799 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -4.85 µm | Standard Deviation 5.035 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -1.93 µm | Standard Deviation 3.043 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -3.48 µm | Standard Deviation 4.243 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -4.26 µm | Standard Deviation 3.16 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -6.55 µm | Standard Deviation 5.942 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -3.83 µm | Standard Deviation 4.044 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -3.01 µm | Standard Deviation 3.685 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -5.05 µm | Standard Deviation 4.629 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -6.47 µm | Standard Deviation 5.111 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -11.18 µm | Standard Deviation 7.506 |
| Placebo | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -3.16 µm | Standard Deviation 4.466 |
| Placebo | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -6.09 µm | Standard Deviation 6.692 |
| Placebo | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -3.52 µm | Standard Deviation 4.738 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -5.82 µm | Standard Deviation 4.866 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.40 µm | Standard Deviation 2.863 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -9.23 µm | Standard Deviation 9.97 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -4.10 µm | Standard Deviation 7.591 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -5.37 µm | Standard Deviation 5.864 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -3.51 µm | Standard Deviation 4.338 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -9.28 µm | Standard Deviation 7.248 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -6.76 µm | Standard Deviation 6.1 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -3.15 µm | Standard Deviation 3.755 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -3.85 µm | Standard Deviation 6.048 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -3.42 µm | Standard Deviation 4.103 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -6.53 µm | Standard Deviation 7.175 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -6.40 µm | Standard Deviation 7.224 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -3.61 µm | Standard Deviation 5.753 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -3.25 µm | Standard Deviation 3.944 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -7.06 µm | Standard Deviation 7.138 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -4.30 µm | Standard Deviation 5.902 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -2.76 µm | Standard Deviation 2.737 |
Secondary
Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT
The EZ - RPE mean central macular 2 mm thickness measurement reflects the distance between the outer border of the EZ and the inner border of the RPE layer, typically within a central 2 mm area. Decreased EZ-RPE thickness is an early indicator of disease, such as in dry AMD, where a thinning or loss of the EZ is a hallmark. Changes in EZ-RPE thickness is used to monitor treatment response in conditions like AMD.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit EZ-RPE mean central macular 2 mm thickness data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below= number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -5.46 µm | Standard Deviation 4.343 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -5.58 µm | Standard Deviation 5.229 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -3.45 µm | Standard Deviation 2.971 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -10.96 µm | Standard Deviation 7.034 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -7.44 µm | Standard Deviation 3.218 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -2.51 µm | Standard Deviation 3.042 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.75 µm | Standard Deviation 2.633 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -1.93 µm | Standard Deviation 3.082 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -5.21 µm | Standard Deviation 4.657 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -4.11 µm | Standard Deviation 3.792 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -3.38 µm | Standard Deviation 2.564 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -7.40 µm | Standard Deviation 5.216 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -2.76 µm | Standard Deviation 3.147 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -5.68 µm | Standard Deviation 4.637 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -5.46 µm | Standard Deviation 3.4 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -10.06 µm | Standard Deviation 7.266 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -3.20 µm | Standard Deviation 2.622 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -4.89 µm | Standard Deviation 5.228 |
| Placebo | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -3.22 µm | Standard Deviation 3.844 |
| Placebo | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -5.27 µm | Standard Deviation 5.408 |
| Placebo | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.58 µm | Standard Deviation 2.819 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -7.97 µm | Standard Deviation 8.308 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.58 µm | Standard Deviation 3.012 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -5.05 µm | Standard Deviation 4.337 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -3.50 µm | Standard Deviation 3.502 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -3.50 µm | Standard Deviation 6.461 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -5.54 µm | Standard Deviation 5.618 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -8.80 µm | Standard Deviation 7.525 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -2.97 µm | Standard Deviation 3.983 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -5.56 µm | Standard Deviation 3.808 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -5.03 µm | Standard Deviation 5.235 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.70 µm | Standard Deviation 3.011 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -4.05 µm | Standard Deviation 5.544 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -6.80 µm | Standard Deviation 5.094 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -3.80 µm | Standard Deviation 4.506 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -5.23 µm | Standard Deviation 5.584 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -3.00 µm | Standard Deviation 1.925 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.45 µm | Standard Deviation 2.512 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -3.16 µm | Standard Deviation 4.206 |
Secondary
Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye
LLD score was calculated as the difference between BCVA and LLVA (BCVA-LLVA) scores. BCVA and LLVA were reported as the number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters). The lower the number of letters read correctly on the eye chart, the worse the vision. LLD is a measure of visual acuity impairment in low-light conditions. A higher LLD score indicates a greater difference between vision in bright and dim light, suggesting a more significant loss of visual function in low-luminance settings.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit LLD score data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 104 | 5.6 letters | Standard Deviation 17.11 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 52 | 4.3 letters | Standard Deviation 13.3 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 104 | 6.3 letters | Standard Deviation 15.22 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 52 | 2.6 letters | Standard Deviation 13.68 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 52 | 0.3 letters | Standard Deviation 15.67 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 104 | 0.5 letters | Standard Deviation 19.93 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 52 | 2.9 letters | Standard Deviation 15.18 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 104 | 4.4 letters | Standard Deviation 13.83 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 52 | 5.3 letters | Standard Deviation 12.56 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 104 | -2.7 letters | Standard Deviation 24 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 52 | -0.8 letters | Standard Deviation 17.15 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 52 | 4.5 letters | Standard Deviation 15.84 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye | Change at Week 104 | 9.3 letters | Standard Deviation 15.91 |
Secondary
Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart
ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome).
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit BCVA score data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 52 | -2.2 letters | Standard Deviation 8.56 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 104 | -1.3 letters | Standard Deviation 4.71 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 52 | -2.0 letters | Standard Deviation 9.39 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 104 | -2.2 letters | Standard Deviation 12.3 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 52 | -3.5 letters | Standard Deviation 11.35 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 104 | -10.2 letters | Standard Deviation 16.37 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 52 | -1.8 letters | Standard Deviation 5.6 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 104 | -2.7 letters | Standard Deviation 6.62 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 52 | -2.4 letters | Standard Deviation 5.74 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 52 | -2.0 letters | Standard Deviation 4.39 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 104 | -3.2 letters | Standard Deviation 5.54 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 104 | -5.4 letters | Standard Deviation 6.39 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Best-corrected Visual Acuity (BCVA) Scores in the Study Eye as Assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Change at Week 52 | -0.9 letters | Standard Deviation 6.58 |
Secondary
Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart
LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. LLVA was reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters). The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Change from baseline: a negative score indicates decline in LLVA.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit LLVA score data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 104 | -10.3 letters | Standard Deviation 14.27 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 52 | -6.3 letters | Standard Deviation 11.61 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 52 | -4.6 letters | Standard Deviation 11.77 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 104 | -9.9 letters | Standard Deviation 14.15 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 104 | -8.9 letters | Standard Deviation 10.39 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 52 | -4.0 letters | Standard Deviation 11.23 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 52 | -4.8 letters | Standard Deviation 15.96 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 104 | -8.0 letters | Standard Deviation 10.37 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 52 | -7.7 letters | Standard Deviation 13.43 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 104 | -3.9 letters | Standard Deviation 20.88 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 52 | -1.2 letters | Standard Deviation 17.9 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 52 | -5.5 letters | Standard Deviation 16.71 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Monocular Low Luminance Visual Acuity (LLVA) Scores in the Study Eye as Assessed by the ETDRS Chart | Change at Week 104 | -11.8 letters | Standard Deviation 15.86 |
Secondary
Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts
MNRead acuity cards were continuous-text reading-acuity cards suitable for measuring the reading acuity and reading speed of normal and low-vision participants. The MNRead acuity cards consisted of single, simple sentences with equal numbers of characters. A stopwatch was used to record time to a tenth of a second. Sentences that could not be read or were not attempted due to vision should be recorded as 0 for time and 10 for errors. The Radner Reading Cards were suitable for measuring reading speed, reading visual acuity, and critical print size. The reading test was stopped when the reading time was longer than 20 seconds or when the participant was making severe errors. A negative change from baseline indicates a decrease in the monocular reading speed; disease worsening.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit reading speed data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 52 | -137.36 words per minute (wpm) | Standard Deviation 1016.889 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 104 | 16.04 words per minute (wpm) | Standard Deviation 142.422 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 104 | -23.70 words per minute (wpm) | Standard Deviation 57.745 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 52 | -19.80 words per minute (wpm) | Standard Deviation 42.134 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 52 | -13.47 words per minute (wpm) | Standard Deviation 77.927 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 104 | -29.01 words per minute (wpm) | Standard Deviation 65.896 |
| Placebo | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 52 | -14.55 words per minute (wpm) | Standard Deviation 81.313 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 52 | 16.22 words per minute (wpm) | Standard Deviation 104.097 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 104 | -59.41 words per minute (wpm) | Standard Deviation 43.156 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 52 | -41.54 words per minute (wpm) | Standard Deviation 53.752 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 104 | 30.18 words per minute (wpm) | Standard Deviation 144.094 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 52 | -30.46 words per minute (wpm) | Standard Deviation 61.042 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 And Week 104 in Monocular Reading Speeds in the Study Eye as Assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts | Change at Week 104 | -47.13 words per minute (wpm) | Standard Deviation 71.218 |
Secondary
Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores
The NEI VFQ-25 instrument measures dimensions of self-reported vision-targeted health status of individuals with chronic eye conditions. The NEI VFQ-25 consists of 11 vision related domains: global vision rating, difficulty with near vision activities, difficulty with distance vision activities, limitations in social function related to vision, role limitations, dependency on others due to vision, mental health symptoms due to vision, driving difficulties, limitations with peripheral- and color-vision, and ocular pain. The NEI VFQ-25 also includes a single item measuring general health. A composite score averages the vision related domains and ranges from 0 (worse) to 100 (best). A higher score represents better functioning. A negative change from baseline indicates a decrease in the visual functioning; disease worsening.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit NEI VFQ-25 scores data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 104 | -7.676 units on a scale | Standard Deviation 16.3251 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 52 | -0.425 units on a scale | Standard Deviation 9.6019 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 104 | -8.478 units on a scale | Standard Deviation 13.5192 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 52 | -5.816 units on a scale | Standard Deviation 11.9162 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 52 | -4.456 units on a scale | Standard Deviation 9.4963 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 104 | -7.454 units on a scale | Standard Deviation 14.2207 |
| Placebo | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 52 | -1.578 units on a scale | Standard Deviation 11.0757 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 104 | -0.332 units on a scale | Standard Deviation 13.3618 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 52 | -1.058 units on a scale | Standard Deviation 9.1822 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 52 | -4.856 units on a scale | Standard Deviation 11.4575 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 104 | 0.535 units on a scale | Standard Deviation 10.7239 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 52 | 1.156 units on a scale | Standard Deviation 12.0823 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) Composite Scores | Change at Week 104 | -9.944 units on a scale | Standard Deviation 13.4043 |
Secondary
Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT
The ONL - RPE mean central macular 2 mm subfield thickness refers to the thickness of the retina in the central part of the macula, specifically within a 2 mm radius.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit ONL-RPE mean central macular 2 mm thickness data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below= number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -7.42 µm | Standard Deviation 7.354 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -14.33 µm | Standard Deviation 11.799 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -8.39 µm | Standard Deviation 8.989 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -17.49 µm | Standard Deviation 14.707 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -14.66 µm | Standard Deviation 14.939 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -27.07 µm | Standard Deviation 19.859 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -10.74 µm | Standard Deviation 14.465 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -14.86 µm | Standard Deviation 11.321 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -21.71 µm | Standard Deviation 24.931 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -6.56 µm | Standard Deviation 9.96 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -12.09 µm | Standard Deviation 21.562 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -5.91 µm | Standard Deviation 9.783 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -9.01 µm | Standard Deviation 8.981 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -15.47 µm | Standard Deviation 11.523 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -17.60 µm | Standard Deviation 10.975 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -16.18 µm | Standard Deviation 13.333 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -8.04 µm | Standard Deviation 8.589 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -32.26 µm | Standard Deviation 19.723 |
| Placebo | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -13.83 µm | Standard Deviation 12.378 |
| Placebo | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -7.92 µm | Standard Deviation 9.205 |
| Placebo | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -7.25 µm | Standard Deviation 7.219 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -32.27 µm | Standard Deviation 31.178 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -4.85 µm | Standard Deviation 6.749 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -21.03 µm | Standard Deviation 23.221 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -7.28 µm | Standard Deviation 7.784 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -13.48 µm | Standard Deviation 10.615 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -11.01 µm | Standard Deviation 11.299 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -16.20 µm | Standard Deviation 4.746 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -7.04 µm | Standard Deviation 7.522 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -14.62 µm | Standard Deviation 6.409 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -7.53 µm | Standard Deviation 6.631 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -13.05 µm | Standard Deviation 9.983 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -27.58 µm | Standard Deviation 13.28 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -9.46 µm | Standard Deviation 7.805 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -9.32 µm | Standard Deviation 11.75 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -17.56 µm | Standard Deviation 14.952 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -19.46 µm | Standard Deviation 11.807 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -40.44 µm | Standard Deviation 23.788 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -20.98 µm | Standard Deviation 12.521 |
Secondary
Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT
The ONL - RPE mean central 1 mm subfield thickness refers to the average thickness of the retinal layers between the ONL and RPE measured within a 1 mm radius from the foveal center (the central point of the retina).
Time frame: Baseline, Week 52 and Week 104
Population: All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit ONL-RPE mean central 1 mm subfield thickness data. As prespecified, data were collected and reported for participants. Hence, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -7.83 micrometers (µm) | Standard Deviation 13.457 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -13.84 micrometers (µm) | Standard Deviation 25.155 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -11.16 micrometers (µm) | Standard Deviation 16.448 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -22.29 micrometers (µm) | Standard Deviation 31.682 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -12.63 micrometers (µm) | Standard Deviation 23.455 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -4.60 micrometers (µm) | Standard Deviation 13.366 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -7.77 micrometers (µm) | Standard Deviation 18.785 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -4.65 micrometers (µm) | Standard Deviation 12.206 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -10.01 micrometers (µm) | Standard Deviation 29.953 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -4.48 micrometers (µm) | Standard Deviation 13.398 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -12.00 micrometers (µm) | Standard Deviation 6.459 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -17.78 micrometers (µm) | Standard Deviation 28.793 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -4.86 micrometers (µm) | Standard Deviation 13.345 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -16.53 micrometers (µm) | Standard Deviation 25.363 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -11.75 micrometers (µm) | Standard Deviation 21.771 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -28.34 micrometers (µm) | Standard Deviation 39.848 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -12.47 micrometers (µm) | Standard Deviation 23.648 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -7.63 micrometers (µm) | Standard Deviation 15.236 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -3.10 micrometers (µm) | Standard Deviation 10.666 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -9.55 micrometers (µm) | Standard Deviation 20.028 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -7.63 micrometers (µm) | Standard Deviation 15.088 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -22.77 micrometers (µm) | Standard Deviation 38.733 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.51 micrometers (µm) | Standard Deviation 10.404 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -4.60 micrometers (µm) | Standard Deviation 19.446 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -4.69 micrometers (µm) | Standard Deviation 13.097 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -34.67 micrometers (µm) | Standard Deviation 50.039 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -14.28 micrometers (µm) | Standard Deviation 14.915 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -14.72 micrometers (µm) | Standard Deviation 14.931 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -26.53 micrometers (µm) | Standard Deviation 10.412 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.53 micrometers (µm) | Standard Deviation 8.417 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -9.01 micrometers (µm) | Standard Deviation 17.029 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -9.57 micrometers (µm) | Standard Deviation 19.25 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -35.94 micrometers (µm) | Standard Deviation 25.831 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -6.41 micrometers (µm) | Standard Deviation 12.496 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -39.08 micrometers (µm) | Standard Deviation 32.217 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -14.70 micrometers (µm) | Standard Deviation 20.919 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -12.76 micrometers (µm) | Standard Deviation 15.206 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -26.32 micrometers (µm) | Standard Deviation 18.045 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -9.39 micrometers (µm) | Standard Deviation 15.478 |
Secondary
Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT
Sub-RPE compartment measures reflect the disease burden between the RPE and Bruch's membrane. The panmacular sub-RPE volume refers to the total volume of fluid or space located underneath the RPE across the entire macula (the central part of the retina).
Time frame: Baseline, Week 52 and Week 104
Population: All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit panmacular sub-RPE volume data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.04 mm^3 | Standard Deviation 0.052 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.07 mm^3 | Standard Deviation 0.102 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.04 mm^3 | Standard Deviation 0.074 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.08 mm^3 | Standard Deviation 0.076 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.08 mm^3 | Standard Deviation 0.084 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.14 mm^3 | Standard Deviation 0.127 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.06 mm^3 | Standard Deviation 0.073 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.12 mm^3 | Standard Deviation 0.16 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.07 mm^3 | Standard Deviation 0.085 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.09 mm^3 | Standard Deviation 0.098 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.04 mm^3 | Standard Deviation 0.048 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.04 mm^3 | Standard Deviation 0.05 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.17 mm^3 | Standard Deviation 0.128 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.04 mm^3 | Standard Deviation 0.054 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.08 mm^3 | Standard Deviation 0.085 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.08 mm^3 | Standard Deviation 0.088 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.05 mm^3 | Standard Deviation 0.059 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.09 mm^3 | Standard Deviation 0.065 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.04 mm^3 | Standard Deviation 0.068 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.08 mm^3 | Standard Deviation 0.111 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.04 mm^3 | Standard Deviation 0.066 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.06 mm^3 | Standard Deviation 0.079 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.14 mm^3 | Standard Deviation 0.196 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.05 mm^3 | Standard Deviation 0.082 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.09 mm^3 | Standard Deviation 0.142 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.05 mm^3 | Standard Deviation 0.094 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.11 mm^3 | Standard Deviation 0.114 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.05 mm^3 | Standard Deviation 0.05 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.14 mm^3 | Standard Deviation 0.085 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.03 mm^3 | Standard Deviation 0.064 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.10 mm^3 | Standard Deviation 0.063 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.02 mm^3 | Standard Deviation 0.053 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.05 mm^3 | Standard Deviation 0.089 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -0.24 mm^3 | Standard Deviation 0.074 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.06 mm^3 | Standard Deviation 0.062 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.03 mm^3 | Standard Deviation 0.051 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -0.17 mm^3 | Standard Deviation 0.123 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -0.06 mm^3 | Standard Deviation 0.091 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -0.08 mm^3 | Standard Deviation 0.093 |
Secondary
Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT
The percentage of partial attenuation of the EZ in the central 1 mm subfield is reported.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 10.31 percentage of EZ partial attenuation | Standard Deviation 13.668 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 15.74 percentage of EZ partial attenuation | Standard Deviation 20.909 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 24.56 percentage of EZ partial attenuation | Standard Deviation 14.964 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 13.35 percentage of EZ partial attenuation | Standard Deviation 18.08 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 6.80 percentage of EZ partial attenuation | Standard Deviation 14.277 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 31.92 percentage of EZ partial attenuation | Standard Deviation 29.615 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 12.06 percentage of EZ partial attenuation | Standard Deviation 15.349 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 16.86 percentage of EZ partial attenuation | Standard Deviation 18.793 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 5.39 percentage of EZ partial attenuation | Standard Deviation 9.926 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 8.24 percentage of EZ partial attenuation | Standard Deviation 12.342 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 22.85 percentage of EZ partial attenuation | Standard Deviation 20.419 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 9.25 percentage of EZ partial attenuation | Standard Deviation 10.751 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 16.06 percentage of EZ partial attenuation | Standard Deviation 20.365 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 7.26 percentage of EZ partial attenuation | Standard Deviation 12.982 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 24.56 percentage of EZ partial attenuation | Standard Deviation 23.689 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 9.51 percentage of EZ partial attenuation | Standard Deviation 14.02 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 12.57 percentage of EZ partial attenuation | Standard Deviation 16.778 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 12.65 percentage of EZ partial attenuation | Standard Deviation 15.581 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 8.67 percentage of EZ partial attenuation | Standard Deviation 13.691 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 9.92 percentage of EZ partial attenuation | Standard Deviation 16.529 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 16.93 percentage of EZ partial attenuation | Standard Deviation 21.887 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 9.62 percentage of EZ partial attenuation | Standard Deviation 12.575 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 17.58 percentage of EZ partial attenuation | Standard Deviation 20.152 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 14.79 percentage of EZ partial attenuation | Standard Deviation 17.28 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 29.97 percentage of EZ partial attenuation | Standard Deviation 39.643 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 6.65 percentage of EZ partial attenuation | Standard Deviation 7.932 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 14.87 percentage of EZ partial attenuation | Standard Deviation 29.975 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 11.42 percentage of EZ partial attenuation | Standard Deviation 18.493 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 22.86 percentage of EZ partial attenuation | Standard Deviation 18.298 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 9.45 percentage of EZ partial attenuation | Standard Deviation 13.614 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 18.81 percentage of EZ partial attenuation | Standard Deviation 23.297 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 5.76 percentage of EZ partial attenuation | Standard Deviation 11.374 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 27.47 percentage of EZ partial attenuation | Standard Deviation 22.597 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 17.27 percentage of EZ partial attenuation | Standard Deviation 25.273 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 4.08 percentage of EZ partial attenuation | Standard Deviation 5.899 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 10.38 percentage of EZ partial attenuation | Standard Deviation 18.139 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 14.46 percentage of EZ partial attenuation | Standard Deviation 17.962 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 10.61 percentage of EZ partial attenuation | Standard Deviation 22.125 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 7.92 percentage of EZ partial attenuation | Standard Deviation 12.672 |
Secondary
Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT
The percentage of total attenuation of the EZ in the central 1 mm subfield is reported.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 22.31 percentage of EZ total attenuation | Standard Deviation 17.89 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 23.92 percentage of EZ total attenuation | Standard Deviation 21.961 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 16.73 percentage of EZ total attenuation | Standard Deviation 16.914 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 20.59 percentage of EZ total attenuation | Standard Deviation 15.693 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 36.13 percentage of EZ total attenuation | Standard Deviation 29.699 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 9.16 percentage of EZ total attenuation | Standard Deviation 12.8 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 28.93 percentage of EZ total attenuation | Standard Deviation 27.084 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 21.82 percentage of EZ total attenuation | Standard Deviation 27.888 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 20.14 percentage of EZ total attenuation | Standard Deviation 19.616 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 8.33 percentage of EZ total attenuation | Standard Deviation 16.198 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 13.59 percentage of EZ total attenuation | Standard Deviation 16.375 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 15.93 percentage of EZ total attenuation | Standard Deviation 21.716 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 18.68 percentage of EZ total attenuation | Standard Deviation 20.417 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 24.22 percentage of EZ total attenuation | Standard Deviation 22.988 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 61.94 percentage of EZ total attenuation | Standard Deviation 38.776 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 12.68 percentage of EZ total attenuation | Standard Deviation 16.131 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 30.13 percentage of EZ total attenuation | Standard Deviation 26.472 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 39.81 percentage of EZ total attenuation | Standard Deviation 26.391 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 24.82 percentage of EZ total attenuation | Standard Deviation 24.477 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 13.69 percentage of EZ total attenuation | Standard Deviation 16.939 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 13.44 percentage of EZ total attenuation | Standard Deviation 17.791 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 10.43 percentage of EZ total attenuation | Standard Deviation 12.638 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 11.07 percentage of EZ total attenuation | Standard Deviation 13.674 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 28.09 percentage of EZ total attenuation | Standard Deviation 27.09 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 19.40 percentage of EZ total attenuation | Standard Deviation 19.132 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 13.33 percentage of EZ total attenuation | Standard Deviation 23.449 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 22.27 percentage of EZ total attenuation | Standard Deviation 27.116 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 18.21 percentage of EZ total attenuation | Standard Deviation 23.796 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 12.68 percentage of EZ total attenuation | Standard Deviation 18.176 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 12.33 percentage of EZ total attenuation | Standard Deviation 10.834 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 13.11 percentage of EZ total attenuation | Standard Deviation 12.384 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 22.33 percentage of EZ total attenuation | Standard Deviation 20.562 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 28.70 percentage of EZ total attenuation | Standard Deviation 31.841 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 14.51 percentage of EZ total attenuation | Standard Deviation 16.785 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 38.42 percentage of EZ total attenuation | Standard Deviation 27.959 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 29.97 percentage of EZ total attenuation | Standard Deviation 25.421 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 19.61 percentage of EZ total attenuation | Standard Deviation 16.593 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 18.81 percentage of EZ total attenuation | Standard Deviation 17.001 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 17.48 percentage of EZ total attenuation | Standard Deviation 18.703 |
Secondary
Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT
The percentage of total attenuation of the RPE in the central 1 mm subfield is reported.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit central 1 mm subfield total RPE attenuation data. As prespecified, data were collected and reported for participants. Hence, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 16.29 percentage of total RPE attenuation | Standard Deviation 21.116 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 15.56 percentage of total RPE attenuation | Standard Deviation 18.923 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 15.43 percentage of total RPE attenuation | Standard Deviation 13.245 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 7.48 percentage of total RPE attenuation | Standard Deviation 12.846 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 26.10 percentage of total RPE attenuation | Standard Deviation 21.267 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 10.27 percentage of total RPE attenuation | Standard Deviation 13.204 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 9.84 percentage of total RPE attenuation | Standard Deviation 13.9 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 12.79 percentage of total RPE attenuation | Standard Deviation 16.127 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 21.13 percentage of total RPE attenuation | Standard Deviation 18.674 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 8.60 percentage of total RPE attenuation | Standard Deviation 11.277 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 14.77 percentage of total RPE attenuation | Standard Deviation 12.017 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 5.91 percentage of total RPE attenuation | Standard Deviation 10.333 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 19.42 percentage of total RPE attenuation | Standard Deviation 18.209 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 39.11 percentage of total RPE attenuation | Standard Deviation 27.628 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 20.71 percentage of total RPE attenuation | Standard Deviation 18.542 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 8.67 percentage of total RPE attenuation | Standard Deviation 11.815 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 9.60 percentage of total RPE attenuation | Standard Deviation 13.794 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 17.43 percentage of total RPE attenuation | Standard Deviation 19.549 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 16.23 percentage of total RPE attenuation | Standard Deviation 21.394 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 9.69 percentage of total RPE attenuation | Standard Deviation 15.031 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 8.16 percentage of total RPE attenuation | Standard Deviation 12.335 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 15.85 percentage of total RPE attenuation | Standard Deviation 19.996 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 9.85 percentage of total RPE attenuation | Standard Deviation 13.381 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 7.33 percentage of total RPE attenuation | Standard Deviation 10.67 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 13.89 percentage of total RPE attenuation | Standard Deviation 12.33 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 21.88 percentage of total RPE attenuation | Standard Deviation 20.194 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 32.12 percentage of total RPE attenuation | Standard Deviation 28.775 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 28.62 percentage of total RPE attenuation | Standard Deviation 27.097 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 6.46 percentage of total RPE attenuation | Standard Deviation 7.8 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 22.45 percentage of total RPE attenuation | Standard Deviation 22.899 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 9.02 percentage of total RPE attenuation | Standard Deviation 13.346 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 46.58 percentage of total RPE attenuation | Standard Deviation 43.859 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 14.08 percentage of total RPE attenuation | Standard Deviation 18.363 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 18.76 percentage of total RPE attenuation | Standard Deviation 25.857 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 23.19 percentage of total RPE attenuation | Standard Deviation 37.563 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 38.98 percentage of total RPE attenuation | Standard Deviation 66.64 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 14.81 percentage of total RPE attenuation | Standard Deviation 21.405 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 15.79 percentage of total RPE attenuation | Standard Deviation 29.801 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 5.48 percentage of total RPE attenuation | Standard Deviation 9.989 |
Secondary
Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT
The percentage of partial attenuation of the EZ in the central macular 2 mm subfield is reported.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 6.24 percentage of EZ partial attenuation | Standard Deviation 9.738 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 27.52 percentage of EZ partial attenuation | Standard Deviation 23.015 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 18.70 percentage of EZ partial attenuation | Standard Deviation 12.603 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 8.24 percentage of EZ partial attenuation | Standard Deviation 10.598 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 13.27 percentage of EZ partial attenuation | Standard Deviation 14.756 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 13.98 percentage of EZ partial attenuation | Standard Deviation 14.829 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 5.12 percentage of EZ partial attenuation | Standard Deviation 9.238 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 17.70 percentage of EZ partial attenuation | Standard Deviation 15.71 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 6.09 percentage of EZ partial attenuation | Standard Deviation 7.169 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 9.71 percentage of EZ partial attenuation | Standard Deviation 10.869 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 12.71 percentage of EZ partial attenuation | Standard Deviation 13.46 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 7.71 percentage of EZ partial attenuation | Standard Deviation 7.689 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 10.96 percentage of EZ partial attenuation | Standard Deviation 11.518 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 12.49 percentage of EZ partial attenuation | Standard Deviation 18.409 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 6.73 percentage of EZ partial attenuation | Standard Deviation 11.225 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 7.54 percentage of EZ partial attenuation | Standard Deviation 8.63 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 13.61 percentage of EZ partial attenuation | Standard Deviation 15.682 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 22.87 percentage of EZ partial attenuation | Standard Deviation 24.193 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 9.07 percentage of EZ partial attenuation | Standard Deviation 12.589 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 6.37 percentage of EZ partial attenuation | Standard Deviation 8.079 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 13.96 percentage of EZ partial attenuation | Standard Deviation 16.418 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 9.62 percentage of EZ partial attenuation | Standard Deviation 10.501 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 12.12 percentage of EZ partial attenuation | Standard Deviation 11.782 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 12.14 percentage of EZ partial attenuation | Standard Deviation 25.712 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 14.82 percentage of EZ partial attenuation | Standard Deviation 15.686 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 6.67 percentage of EZ partial attenuation | Standard Deviation 7.237 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 22.24 percentage of EZ partial attenuation | Standard Deviation 31.056 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 9.47 percentage of EZ partial attenuation | Standard Deviation 17.096 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 6.93 percentage of EZ partial attenuation | Standard Deviation 9.374 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 8.30 percentage of EZ partial attenuation | Standard Deviation 12.676 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 13.42 percentage of EZ partial attenuation | Standard Deviation 16.367 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 24.67 percentage of EZ partial attenuation | Standard Deviation 24.32 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 17.09 percentage of EZ partial attenuation | Standard Deviation 13.793 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 9.30 percentage of EZ partial attenuation | Standard Deviation 13.802 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 9.17 percentage of EZ partial attenuation | Standard Deviation 14.826 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 14.41 percentage of EZ partial attenuation | Standard Deviation 14.806 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 5.12 percentage of EZ partial attenuation | Standard Deviation 5.509 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 5.46 percentage of EZ partial attenuation | Standard Deviation 7.746 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 13.59 percentage of EZ partial attenuation | Standard Deviation 17.862 |
Secondary
Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT
The percentage of total attenuation of the EZ in the central macular 2 mm subfield is reported.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 23.81 percentage of EZ total attenuation | Standard Deviation 17.076 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 21.45 percentage of EZ total attenuation | Standard Deviation 21.023 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 9.80 percentage of EZ total attenuation | Standard Deviation 11.161 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 27.98 percentage of EZ total attenuation | Standard Deviation 14.597 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 16.04 percentage of EZ total attenuation | Standard Deviation 12.199 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 41.63 percentage of EZ total attenuation | Standard Deviation 25.286 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 19.87 percentage of EZ total attenuation | Standard Deviation 18.112 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 12.76 percentage of EZ total attenuation | Standard Deviation 9.657 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 23.25 percentage of EZ total attenuation | Standard Deviation 15.725 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 13.61 percentage of EZ total attenuation | Standard Deviation 13.797 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 8.84 percentage of EZ total attenuation | Standard Deviation 11.049 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 31.50 percentage of EZ total attenuation | Standard Deviation 18.865 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 16.10 percentage of EZ total attenuation | Standard Deviation 12.422 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 53.57 percentage of EZ total attenuation | Standard Deviation 30.657 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 26.52 percentage of EZ total attenuation | Standard Deviation 18.341 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 22.72 percentage of EZ total attenuation | Standard Deviation 20.095 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 11.54 percentage of EZ total attenuation | Standard Deviation 11.821 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 32.56 percentage of EZ total attenuation | Standard Deviation 15.265 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 12.89 percentage of EZ total attenuation | Standard Deviation 11.554 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 13.21 percentage of EZ total attenuation | Standard Deviation 12.847 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 23.84 percentage of EZ total attenuation | Standard Deviation 19.218 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 21.46 percentage of EZ total attenuation | Standard Deviation 20.555 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 31.12 percentage of EZ total attenuation | Standard Deviation 24.754 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 25.38 percentage of EZ total attenuation | Standard Deviation 19.781 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 12.66 percentage of EZ total attenuation | Standard Deviation 11.515 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 11.59 percentage of EZ total attenuation | Standard Deviation 13.028 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 15.14 percentage of EZ total attenuation | Standard Deviation 14.786 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 32.22 percentage of EZ total attenuation | Standard Deviation 18.972 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 11.79 percentage of EZ total attenuation | Standard Deviation 10.311 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 20.37 percentage of EZ total attenuation | Standard Deviation 14.102 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 11.15 percentage of EZ total attenuation | Standard Deviation 8.184 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 17.50 percentage of EZ total attenuation | Standard Deviation 6.271 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 18.41 percentage of EZ total attenuation | Standard Deviation 13.948 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 14.96 percentage of EZ total attenuation | Standard Deviation 14.456 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 35.83 percentage of EZ total attenuation | Standard Deviation 17.546 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 25.71 percentage of EZ total attenuation | Standard Deviation 23.057 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 12.48 percentage of EZ total attenuation | Standard Deviation 13.098 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 16.20 percentage of EZ total attenuation | Standard Deviation 13.464 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 19.63 percentage of EZ total attenuation | Standard Deviation 7.982 |
Secondary
Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT
The percentage of total attenuation of the RPE in the central macular 2 mm subfield is reported.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in FAS (all randomized participants who received at least 1 dose \[full/partial\] of study drug grouped by randomized treatment) with both baseline and postbaseline (Week 52 and Week 104) visit central macular 2 mm subfield total RPE attenuation data. As prespecified, data were collected and reported for participants. Hence, 'Ns' reported below= number of participants analyzed regardless of which eye was evaluated. Number analyzed= participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 11.07 percentage of total RPE attenuation | Standard Deviation 8.79 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 18.22 percentage of total RPE attenuation | Standard Deviation 13.985 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 17.54 percentage of total RPE attenuation | Standard Deviation 13.638 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 22.11 percentage of total RPE attenuation | Standard Deviation 18.777 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 33.38 percentage of total RPE attenuation | Standard Deviation 19.938 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 8.84 percentage of total RPE attenuation | Standard Deviation 9.019 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 10.86 percentage of total RPE attenuation | Standard Deviation 7.042 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 20.14 percentage of total RPE attenuation | Standard Deviation 11.243 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 17.22 percentage of total RPE attenuation | Standard Deviation 13.792 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 8.99 percentage of total RPE attenuation | Standard Deviation 11.116 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 12.04 percentage of total RPE attenuation | Standard Deviation 10.432 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 27.94 percentage of total RPE attenuation | Standard Deviation 19.154 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 11.21 percentage of total RPE attenuation | Standard Deviation 8.539 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 20.75 percentage of total RPE attenuation | Standard Deviation 13.888 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 42.14 percentage of total RPE attenuation | Standard Deviation 18.854 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 20.96 percentage of total RPE attenuation | Standard Deviation 12.092 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 22.35 percentage of total RPE attenuation | Standard Deviation 9.98 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 10.56 percentage of total RPE attenuation | Standard Deviation 9.026 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 11.50 percentage of total RPE attenuation | Standard Deviation 8.889 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 19.74 percentage of total RPE attenuation | Standard Deviation 14.674 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 10.18 percentage of total RPE attenuation | Standard Deviation 9.265 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 21.58 percentage of total RPE attenuation | Standard Deviation 14.796 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 33.88 percentage of total RPE attenuation | Standard Deviation 27.642 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 14.87 percentage of total RPE attenuation | Standard Deviation 10.962 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 12.20 percentage of total RPE attenuation | Standard Deviation 9.228 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 11.30 percentage of total RPE attenuation | Standard Deviation 7.522 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 22.81 percentage of total RPE attenuation | Standard Deviation 19.741 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 41.73 percentage of total RPE attenuation | Standard Deviation 15.668 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 8.36 percentage of total RPE attenuation | Standard Deviation 7.234 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 20.32 percentage of total RPE attenuation | Standard Deviation 5.212 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 8.89 percentage of total RPE attenuation | Standard Deviation 8.759 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 15.43 percentage of total RPE attenuation | Standard Deviation 11.951 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 26.77 percentage of total RPE attenuation | Standard Deviation 6.514 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 22.13 percentage of total RPE attenuation | Standard Deviation 16.571 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 13.47 percentage of total RPE attenuation | Standard Deviation 11.872 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 14.38 percentage of total RPE attenuation | Standard Deviation 10.698 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 26.85 percentage of total RPE attenuation | Standard Deviation 20.134 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 9.41 percentage of total RPE attenuation | Standard Deviation 9.869 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 40.32 percentage of total RPE attenuation | Standard Deviation 28.798 |
Secondary
Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT)
Photoreceptor loss was assessed by measuring the percent of macular EZ total attenuation using SD-OCT. The EZ was considered to be formed mainly by mitochondria within the ellipsoid layer of the outer portion of the inner segments of the photoreceptors. In SD-OCT, decreases in the integrity and intensity of the EZ have been correlated with the reduction in cone photoreceptors and retinal sensitivity in retinal degenerative diseases. Lower increase in percentage means a better outcome. Higher increase in percentage means a worse outcome.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 52 | 6.31 percentage of EZ total attenuation | Standard Deviation 3.642 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 52 | 13.77 percentage of EZ total attenuation | Standard Deviation 7.285 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 52 | 8.70 percentage of EZ total attenuation | Standard Deviation 4.681 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 104 | 22.18 percentage of EZ total attenuation | Standard Deviation 15.324 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 104 | 12.80 percentage of EZ total attenuation | Standard Deviation 7.605 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 104 | 13.54 percentage of EZ total attenuation | Standard Deviation 7.664 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 52 | 7.48 percentage of EZ total attenuation | Standard Deviation 4.226 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 52 | 11.27 percentage of EZ total attenuation | Standard Deviation 6.892 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 52 | 5.53 percentage of EZ total attenuation | Standard Deviation 3.665 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 104 | 11.72 percentage of EZ total attenuation | Standard Deviation 7.976 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 104 | 9.48 percentage of EZ total attenuation | Standard Deviation 5.396 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 104 | 17.17 percentage of EZ total attenuation | Standard Deviation 13.337 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 104 | 15.27 percentage of EZ total attenuation | Standard Deviation 6.156 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 104 | 14.05 percentage of EZ total attenuation | Standard Deviation 9.509 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 104 | 28.51 percentage of EZ total attenuation | Standard Deviation 12.937 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 52 | 14.94 percentage of EZ total attenuation | Standard Deviation 8.335 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 52 | 6.96 percentage of EZ total attenuation | Standard Deviation 4.684 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 52 | 8.66 percentage of EZ total attenuation | Standard Deviation 4.633 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 52 | 7.87 percentage of EZ total attenuation | Standard Deviation 4.79 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 52 | 7.50 percentage of EZ total attenuation | Standard Deviation 4.814 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 52 | 14.02 percentage of EZ total attenuation | Standard Deviation 8.564 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 52 | 7.31 percentage of EZ total attenuation | Standard Deviation 4.898 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 52 | 6.03 percentage of EZ total attenuation | Standard Deviation 3.147 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 104 | 10.81 percentage of EZ total attenuation | Standard Deviation 6.514 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 104 | 11.82 percentage of EZ total attenuation | Standard Deviation 9.719 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 52 | 12.17 percentage of EZ total attenuation | Standard Deviation 7.625 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 104 | 20.65 percentage of EZ total attenuation | Standard Deviation 15.245 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 104 | 12.34 percentage of EZ total attenuation | Standard Deviation 5.174 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 104 | 12.65 percentage of EZ total attenuation | Standard Deviation 5.163 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 104 | 22.53 percentage of EZ total attenuation | Standard Deviation 7.748 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 52 | 13.43 percentage of EZ total attenuation | Standard Deviation 7.072 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 52 | 7.74 percentage of EZ total attenuation | Standard Deviation 4.469 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 52 | 7.26 percentage of EZ total attenuation | Standard Deviation 4.164 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 104 | 14.19 percentage of EZ total attenuation | Standard Deviation 3.223 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Fellow Eye: Change at Week 52 | 8.94 percentage of EZ total attenuation | Standard Deviation 5.205 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 52 | 8.77 percentage of EZ total attenuation | Standard Deviation 6.206 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Study Eye: Change at Week 104 | 14.76 percentage of EZ total attenuation | Standard Deviation 6.672 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 104 | 28.95 percentage of EZ total attenuation | Standard Deviation 7.702 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Both Eyes Combined: Change at Week 52 | 16.55 percentage of EZ total attenuation | Standard Deviation 10.412 |
Secondary
Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT
Photoreceptor degradation was assessed by measuring the percent of macular EZ partial attenuation using SD-OCT. The EZ was considered to be formed mainly by mitochondria within the ellipsoid layer of the outer portion of the inner segments of the photoreceptors. In SD-OCT, decreases in the integrity and intensity of the EZ have been correlated with the reduction in cone photoreceptors and retinal sensitivity in retinal degenerative diseases. Lower increase in percentage means a better outcome. Higher increase in percentage means a worse outcome.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 6.42 percentage of EZ partial attenuation | Standard Deviation 4.917 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 11.10 percentage of EZ partial attenuation | Standard Deviation 8.11 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 19.85 percentage of EZ partial attenuation | Standard Deviation 14.357 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 5.74 percentage of EZ partial attenuation | Standard Deviation 4.531 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 11.09 percentage of EZ partial attenuation | Standard Deviation 6.771 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 12.48 percentage of EZ partial attenuation | Standard Deviation 7.29 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 4.28 percentage of EZ partial attenuation | Standard Deviation 3.646 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 5.15 percentage of EZ partial attenuation | Standard Deviation 3.806 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 12.67 percentage of EZ partial attenuation | Standard Deviation 10.789 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 8.10 percentage of EZ partial attenuation | Standard Deviation 5.976 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 8.06 percentage of EZ partial attenuation | Standard Deviation 6.728 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 7.87 percentage of EZ partial attenuation | Standard Deviation 5.927 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 26.92 percentage of EZ partial attenuation | Standard Deviation 14.542 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 13.87 percentage of EZ partial attenuation | Standard Deviation 7.495 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 13.79 percentage of EZ partial attenuation | Standard Deviation 9.773 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 5.61 percentage of EZ partial attenuation | Standard Deviation 4.987 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 6.63 percentage of EZ partial attenuation | Standard Deviation 4.599 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 11.69 percentage of EZ partial attenuation | Standard Deviation 8.277 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 6.31 percentage of EZ partial attenuation | Standard Deviation 4.37 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 5.65 percentage of EZ partial attenuation | Standard Deviation 4.365 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 10.90 percentage of EZ partial attenuation | Standard Deviation 7.617 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 4.00 percentage of EZ partial attenuation | Standard Deviation 3.423 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 8.11 percentage of EZ partial attenuation | Standard Deviation 5.784 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 12.92 percentage of EZ partial attenuation | Standard Deviation 10.399 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 4.90 percentage of EZ partial attenuation | Standard Deviation 3.399 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 7.75 percentage of EZ partial attenuation | Standard Deviation 4.677 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 6.20 percentage of EZ partial attenuation | Standard Deviation 6.897 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 5.54 percentage of EZ partial attenuation | Standard Deviation 4.089 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 10.55 percentage of EZ partial attenuation | Standard Deviation 7.536 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 11.52 percentage of EZ partial attenuation | Standard Deviation 8.433 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 5.83 percentage of EZ partial attenuation | Standard Deviation 4.616 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 10.10 percentage of EZ partial attenuation | Standard Deviation 6.989 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 19.77 percentage of EZ partial attenuation | Standard Deviation 11.361 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 13.48 percentage of EZ partial attenuation | Standard Deviation 3.926 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 8.15 percentage of EZ partial attenuation | Standard Deviation 4.563 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 14.60 percentage of EZ partial attenuation | Standard Deviation 8.644 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 10.56 percentage of EZ partial attenuation | Standard Deviation 6.122 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 7.49 percentage of EZ partial attenuation | Standard Deviation 4.946 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 24.04 percentage of EZ partial attenuation | Standard Deviation 8.185 |
Secondary
Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT
Macular total RPE attenuation refers to the degree of thinning or loss of the RPE in the macula, the central part of the retina responsible for sharp, detailed vision. This was measured as a percentage of the macular area affected.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit macular total RPE attenuation data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 18.20 percentage of macular area | Standard Deviation 12.155 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 10.36 percentage of macular area | Standard Deviation 6.652 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 10.66 percentage of macular area | Standard Deviation 6.579 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 10.96 percentage of macular area | Standard Deviation 6.159 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 5.23 percentage of macular area | Standard Deviation 3.594 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 6.11 percentage of macular area | Standard Deviation 3.874 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 5.00 percentage of macular area | Standard Deviation 3.549 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 8.58 percentage of macular area | Standard Deviation 5.429 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 14.14 percentage of macular area | Standard Deviation 11.759 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 5.11 percentage of macular area | Standard Deviation 2.706 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 8.50 percentage of macular area | Standard Deviation 6.472 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 9.18 percentage of macular area | Standard Deviation 5.951 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 5.59 percentage of macular area | Standard Deviation 4.513 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 11.64 percentage of macular area | Standard Deviation 4.956 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 11.49 percentage of macular area | Standard Deviation 8.582 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 11.42 percentage of macular area | Standard Deviation 6.976 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 6.38 percentage of macular area | Standard Deviation 3.718 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 22.52 percentage of macular area | Standard Deviation 12.076 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 5.48 percentage of macular area | Standard Deviation 3.413 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 6.18 percentage of macular area | Standard Deviation 4.043 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 10.62 percentage of macular area | Standard Deviation 7.048 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 10.09 percentage of macular area | Standard Deviation 6.376 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 16.51 percentage of macular area | Standard Deviation 12.246 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 7.86 percentage of macular area | Standard Deviation 4.673 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 5.47 percentage of macular area | Standard Deviation 3.043 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 5.55 percentage of macular area | Standard Deviation 3.474 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 10.38 percentage of macular area | Standard Deviation 7.719 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 9.06 percentage of macular area | Standard Deviation 6.227 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 5.00 percentage of macular area | Standard Deviation 3.328 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 12.68 percentage of macular area | Standard Deviation 6.33 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 11.82 percentage of macular area | Standard Deviation 6.691 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 5.18 percentage of macular area | Standard Deviation 3.74 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 22.14 percentage of macular area | Standard Deviation 10.27 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 12.73 percentage of macular area | Standard Deviation 8.186 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | 11.77 percentage of macular area | Standard Deviation 5.205 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 6.00 percentage of macular area | Standard Deviation 3.916 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | 16.12 percentage of macular area | Standard Deviation 4.807 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | 27.89 percentage of macular area | Standard Deviation 8.76 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | 7.66 percentage of macular area | Standard Deviation 4.545 |
Secondary
Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT
Sub-RPE compartment measures reflect the disease burden between the RPE and Bruch's membrane. The sub-RPE central 1 mm mean thickness refers to the average thickness of the retina within a 1-mm diameter circular area centered around the fovea (the central part of the retina).
Time frame: Baseline, Week 52 and Week 104
Population: All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit sub-RPE central 1 mm subfield mean thickness data. As prespecified, data were collected and reported for participants. Hence, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -3.23 µm | Standard Deviation 9.34 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -3.24 µm | Standard Deviation 11.423 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.84 µm | Standard Deviation 6.699 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.61 µm | Standard Deviation 5.838 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -3.58 µm | Standard Deviation 9.171 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -5.74 µm | Standard Deviation 14.485 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.89 µm | Standard Deviation 4.863 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -4.18 µm | Standard Deviation 6.176 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -4.33 µm | Standard Deviation 7.45 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -1.50 µm | Standard Deviation 3.274 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -3.95 µm | Standard Deviation 5.893 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -7.04 µm | Standard Deviation 9.644 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.87 µm | Standard Deviation 5.842 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -5.26 µm | Standard Deviation 9.398 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -11.72 µm | Standard Deviation 16.323 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -6.73 µm | Standard Deviation 9.984 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -2.42 µm | Standard Deviation 6.345 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -5.05 µm | Standard Deviation 9.529 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -2.04 µm | Standard Deviation 14.876 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.04 µm | Standard Deviation 12.832 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -2.33 µm | Standard Deviation 8.5 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -12.52 µm | Standard Deviation 16.342 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -1.13 µm | Standard Deviation 11.96 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -3.83 µm | Standard Deviation 12.208 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.97 µm | Standard Deviation 5.403 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -10.56 µm | Standard Deviation 11.61 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -3.72 µm | Standard Deviation 7.208 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -9.50 µm | Standard Deviation 12.294 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.13 µm | Standard Deviation 6.814 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -6.38 µm | Standard Deviation 9.288 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -0.59 µm | Standard Deviation 3.463 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -3.90 µm | Standard Deviation 5.368 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -2.60 µm | Standard Deviation 7.605 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | 0.40 µm | Standard Deviation 21.689 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -9.36 µm | Standard Deviation 12.776 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -5.00 µm | Standard Deviation 6.941 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -4.72 µm | Standard Deviation 9.648 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -14.08 µm | Standard Deviation 21.741 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | 5.19 µm | Standard Deviation 20.081 |
Secondary
Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT
Sub-RPE compartment measures reflect the disease burden between the RPE and Bruch's membrane. The sub-RPE central macular 2 mm subfield thickness refers to the thickness of the retina within a 2-mm radius in the central part of the macula.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in FAS (randomized participants who received at least 1 dose \[full/partial\] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit sub-RPE central macular 2 mm subfield mean thickness data. As prespecified, data were collected and reported for participants. Hence, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -4.06 µm | Standard Deviation 6.749 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.75 µm | Standard Deviation 4.229 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -5.80 µm | Standard Deviation 9.514 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -1.66 µm | Standard Deviation 4.75 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -4.42 µm | Standard Deviation 9.773 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -8.61 µm | Standard Deviation 12.28 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -5.35 µm | Standard Deviation 4.29 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -9.15 µm | Standard Deviation 13.049 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -11.28 µm | Standard Deviation 12.963 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -3.07 µm | Standard Deviation 3.575 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -3.34 µm | Standard Deviation 3.314 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -5.51 µm | Standard Deviation 5.298 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -2.93 µm | Standard Deviation 3.552 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -12.22 µm | Standard Deviation 8.602 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -6.33 µm | Standard Deviation 5.931 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -6.22 µm | Standard Deviation 3.954 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -3.02 µm | Standard Deviation 4.064 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -5.67 µm | Standard Deviation 5.79 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -4.50 µm | Standard Deviation 7.649 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -1.88 µm | Standard Deviation 5.55 |
| Placebo | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -3.12 µm | Standard Deviation 5.733 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -12.03 µm | Standard Deviation 16.671 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -2.79 µm | Standard Deviation 7.131 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -5.66 µm | Standard Deviation 7.552 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -3.36 µm | Standard Deviation 4.148 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -9.90 µm | Standard Deviation 10.97 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -3.78 µm | Standard Deviation 7.19 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -8.38 µm | Standard Deviation 5.509 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -2.06 µm | Standard Deviation 3.834 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -4.32 µm | Standard Deviation 2.743 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -1.24 µm | Standard Deviation 3.434 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -5.08 µm | Standard Deviation 2.898 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -3.03 µm | Standard Deviation 4.963 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 52 | -4.77 µm | Standard Deviation 9.77 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 104 | -12.30 µm | Standard Deviation 10.238 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Fellow Eye: Change at Week 52 | -5.06 µm | Standard Deviation 5.37 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 52 | -0.15 µm | Standard Deviation 7.701 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Both Eyes Combined: Change at Week 104 | -17.46 µm | Standard Deviation 11.297 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT | Study Eye: Change at Week 104 | -5.16 µm | Standard Deviation 4.527 |
Secondary
Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF
FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area (mm\^2) measurement. Greater area affected means a worse outcome than smaller area affected.
Time frame: Baseline, Week 52 and Week 104
Population: All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit FAF GA lesion area data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 52 | 2.347 mm^2 | Standard Deviation 1.2972 |
| Danicopan 100 mg BID | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 104 | 4.311 mm^2 | Standard Deviation 2.8803 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 52 | 2.044 mm^2 | Standard Deviation 1.228 |
| Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 104 | 3.223 mm^2 | Standard Deviation 2.3784 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 104 | 4.504 mm^2 | Standard Deviation 2.13 |
| Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 52 | 2.274 mm^2 | Standard Deviation 1.4482 |
| Placebo | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 52 | 2.057 mm^2 | Standard Deviation 1.2016 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 52 | 1.887 mm^2 | Standard Deviation 1.1212 |
| Placebo - Danicopan 200 mg BID | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 104 | 3.309 mm^2 | Standard Deviation 2.5878 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 52 | 1.716 mm^2 | Standard Deviation 0.9095 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 104 | 3.653 mm^2 | Standard Deviation 2.3125 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 104 | 5.098 mm^2 | Standard Deviation 1.825 |
| Placebo - Danicopan 400 mg QD | Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF | Change at Week 52 | 2.553 mm^2 | Standard Deviation 1.3938 |
Secondary
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study
An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, resulted in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), or an important medical event or reaction. A TEAE was defined as any AE that started during or after the first dose of study intervention. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Time frame: Day 1 through Week 108
Population: Safety Set included all participants who received at least 1 dose (full or partial) of study drug grouped by treatment actually received. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Danicopan 100 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Any TEAEs | 79 Participants |
| Danicopan 100 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | SAEs | 14 Participants |
| Danicopan 100 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Events Leading To Discontinuation of Study Drug | 11 Participants |
| Danicopan 200 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Events Leading To Discontinuation of Study Drug | 15 Participants |
| Danicopan 200 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | SAEs | 19 Participants |
| Danicopan 200 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Any TEAEs | 83 Participants |
| Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | SAEs | 16 Participants |
| Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Any TEAEs | 76 Participants |
| Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Events Leading To Discontinuation of Study Drug | 9 Participants |
| Placebo | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Any TEAEs | 71 Participants |
| Placebo | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | SAEs | 15 Participants |
| Placebo | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Events Leading To Discontinuation of Study Drug | 11 Participants |
| Placebo - Danicopan 200 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Any TEAEs | 51 Participants |
| Placebo - Danicopan 200 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Events Leading To Discontinuation of Study Drug | 1 Participants |
| Placebo - Danicopan 200 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | SAEs | 8 Participants |
| Placebo - Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Events Leading To Discontinuation of Study Drug | 2 Participants |
| Placebo - Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Any TEAEs | 37 Participants |
| Placebo - Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | SAEs | 10 Participants |
| Placebo - Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Any TEAEs | 46 Participants |
| Placebo - Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Events Leading To Discontinuation of Study Drug | 4 Participants |
| Placebo - Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | SAEs | 8 Participants |
| Secondary Evaluation Period: Placebo - Danicopan 100 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Any TEAEs | 19 Participants |
| Secondary Evaluation Period: Placebo - Danicopan 100 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | SAEs | 6 Participants |
| Secondary Evaluation Period: Placebo - Danicopan 100 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Events Leading To Discontinuation of Study Drug | 0 Participants |
| Secondary Evaluation Period: Placebo - Danicopan 200 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Events Leading To Discontinuation of Study Drug | 2 Participants |
| Secondary Evaluation Period: Placebo - Danicopan 200 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | SAEs | 4 Participants |
| Secondary Evaluation Period: Placebo - Danicopan 200 mg BID | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Any TEAEs | 12 Participants |
| Secondary Evaluation Period: Placebo - Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Any TEAEs | 17 Participants |
| Secondary Evaluation Period: Placebo - Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | SAEs | 4 Participants |
| Secondary Evaluation Period: Placebo - Danicopan 400 mg QD | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study | Events Leading To Discontinuation of Study Drug | 4 Participants |
Secondary
Plasma Concentration of Danicopan
All values below the limit of quantification (\<0.1 nanograms \[ng\]/milliliter \[mL\]) were set to 0 for calculation of summary statistics.
Time frame: Day 1 and Week 52
Population: Pharmacokinetic (PK) Analysis Set included all participants who received at least 1 dose (full or partial) of study drug and had at least 1 measurable concentration value. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan 100 mg BID | Plasma Concentration of Danicopan | Week 52: Predose | 62.5 ng/mL | Standard Deviation 40.64 |
| Danicopan 100 mg BID | Plasma Concentration of Danicopan | Day 1: Predose | 0.4 ng/mL | Standard Deviation 3.41 |
| Danicopan 100 mg BID | Plasma Concentration of Danicopan | Week 52: 2 Hour Postdose | 381.6 ng/mL | Standard Deviation 219.22 |
| Danicopan 100 mg BID | Plasma Concentration of Danicopan | Day 1: 2 Hour Postdose | 289.6 ng/mL | Standard Deviation 202.88 |
| Danicopan 200 mg BID | Plasma Concentration of Danicopan | Day 1: Predose | 0 ng/mL | Standard Deviation 0 |
| Danicopan 200 mg BID | Plasma Concentration of Danicopan | Week 52: Predose | 127.7 ng/mL | Standard Deviation 100.78 |
| Danicopan 200 mg BID | Plasma Concentration of Danicopan | Day 1: 2 Hour Postdose | 507.5 ng/mL | Standard Deviation 279.71 |
| Danicopan 200 mg BID | Plasma Concentration of Danicopan | Week 52: 2 Hour Postdose | 704.4 ng/mL | Standard Deviation 336.37 |
| Danicopan 400 mg QD | Plasma Concentration of Danicopan | Day 1: 2 Hour Postdose | 844.5 ng/mL | Standard Deviation 563.54 |
| Danicopan 400 mg QD | Plasma Concentration of Danicopan | Week 52: 2 Hour Postdose | 919.5 ng/mL | Standard Deviation 709.94 |
| Danicopan 400 mg QD | Plasma Concentration of Danicopan | Day 1: Predose | 0.2 ng/mL | Standard Deviation 1.67 |
| Danicopan 400 mg QD | Plasma Concentration of Danicopan | Week 52: Predose | 88.1 ng/mL | Standard Deviation 138.95 |