Phase 2 Study to Evaluate LUM001 in Combination With Ursodeoxycholic Acid in Patients With Primary Biliary Cirrhosis

Pruritus was assessed using ItchRO measure, administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). (ItchRO) scores ranged from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. The weekly sum score was calculated as the sum of the daily scores for the 7 days prior to the time point being reported: 7 days prior to randomization or 7 days prior to Week 13/ET visit.

Time frame: Baseline and Week 13/ET

Population: The mITT population included all participants who were randomized, received at least 1 dose of treatment, and had at least 1 post-baseline ItchRO assessment.

The 5-D itch (validated instrument to measure pruritus) scale was developed for the multidimensional quantification of pruritus that is sensitive to change over time. The 5-D itch scale included 5 domains (duration, degree, direction, disability, and distribution of pruritus). The total 5-D score was obtained by scoring each of the domains separately and then summing them together. 5-D total scores ranged between 5 (no pruritus) and 25 (most severe pruritus).

Time frame: Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET)

Population: mITT Population. Here, n signifies the number of participants evaluable for the respective time points.

Laboratory serum ALP enzyme levels were evaluated using blood samples collected.

Time frame: Baseline, Weeks 4, 8, 13 and Last Post-baseline (Week 13/ET)

Population: mITT Population. Here, n signifies the number of participants evaluable for the respective time points.

C4 7 alpha-hydroxy-4-cholesten-3-one is an intermediate in the biochemical synthesis of bile acids from cholesterol and its concentrations reflect the activity of the bile acid synthetic pathway. Elevated levels of C4 indicate bile acid malabsorption. Laboratory C4 levels were evaluated using blood samples collected.

Time frame: Baseline, Weeks 4, 8, 13 and Last Post-baseline Visit (Week 13/ET)

Population: mITT Population. Here, n signifies the number of participants evaluable for the respective time points.

Laboratory serum bile acid level levels were evaluated using blood samples collected.

Time frame: Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET)

Population: mITT Population. Here, n signifies the number of participants evaluable for the respective time points.

ItchRO scores had a range from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. Adult ItchRO average daily score was the sum of daily scores divided by the number of days adult ItchRO was completed, using the 7 days prior to the reported visit date.

Time frame: Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET)

Population: mITT Population. Here, n signifies the number of participants evaluable for the respective time points.

ItchRO scores had a range from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. The weekly sum score was calculated as the sum of the daily scores for the 7 days prior to the time point being reported: 7 days prior to randomization or 7 days prior to Week 13/ET visit.

Time frame: Baseline, Weeks 4, 8 and 13

Population: mITT Population. Here, n signifies the number of participants evaluable for the respective time points.

An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether or not considered related to the product. A serious adverse event (SAE) was defined as an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life-threatening; persistent or significant disability/incapacity; congenital anomaly or birth defect; an important medical event that did not meet any of the above criteria but jeopardized the participant or required medical or surgical intervention to prevent one of the outcomes listed above. A TEAE was defined as any AE that occurred during the study, from the start of investigational product dosing through the end of the study (13 weeks of treatment period (or ET) + 14 days \]), or that worsened since the start of dosing.

Time frame: From the first dose of study drug until the 13 weeks of treatment period (or ET) + 14 days (approximately 15 weeks)

Population: The Safety Population included all participants who were randomized and received at least 1 dose of the study drug. One participant was randomized to LUM001 10 mg, but was down-titrated to 5 mg dose due to tolerability issues. The safety data has been summarized based on the study dose actually received by the participant.