Spesolimab (BI 655130) Single Dose in Generalized Pustular Psoriasis

Percentage of patients with adverse reactions, defined as drug-related Adverse Events is presented.

Time frame: Up to 140 days from the administration of spesolimab.

Population: Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).

Area under the concentration-time curve of spesolimab in plasma over the time interval from 0 extrapolated to infinity (AUC0-∞) is reported.

Time frame: Within 1.5 hours (h) before and 0.5 h, 24 h, 48 h, 72 h, 96 h, 120 h, 144 h, 312 h, 480 h, 648 h, 1992 h, 3336 h after spesolimab administration.

Population: Pharmacokinetic (PK) parameter set (PKS): All patients in the treated set (TS) who provided at least one secondary PK endpoint not flagged for exclusion due to a protocol violation relevant to the evaluation of PK (to be decided no later than at the Report Planning Meeting) or due to PK non-evaluability (as revealed during data analysis).

Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale scale score at Week 2 is presented. The FACIT-Fatigue scale is a brief and reliable instrument for monitoring fatigue and its effects on patients. It is a comprehensive compilation of questions that measure health-related quality of life in patients with chronic illnesses. It comprises 13 questions, the responses to which are each recorded on a 5-point Likert scale. Scores range from 0 to 52, with lower scores representing greater fatigue, i.e. higher changes from baseline indicate higher improvement (compared to baseline)

Time frame: At baseline and at Week 2.

Population: Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.

The pain VAS is a unidimensional measure of pain intensity. It is a continuous scale comprised of a horizontal or vertical line, usually 10 centimeters (100 millimeters (mm) in length, anchored by word descriptors at each end ('no pain', 'very severe pain'). The pain VAS is self-completed by the respondent. The respondent is asked to place a line perpendicular to the VAS line at the point that represents their pain intensity. Using a ruler, the score is determined by measuring the distance (mm) on the 10-cm line between the 'no pain' anchor and the patient's mark, providing a range of scores from 0-100. A higher score indicates greater pain intensity, i.e., a negative change from baseline indicates an improvement (compared to baseline).

Time frame: At baseline and at Week 2.

Population: Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.

Maximum measured concentration of spesolimab in plasma (Cmax) is reported.

Time frame: Within 1.5 hours (h) before and 0.5 h, 24 h, 48 h, 72 h, 96 h, 120 h, 144 h, 312 h, 480 h, 648 h, 1992 h, 3336 h after spesolimab administration.

Population: Pharmacokinetic (PK) parameter set (PKS): All patients in the treated set (TS) who provided at least one secondary PK endpoint not flagged for exclusion due to a protocol violation relevant to the evaluation of PK (to be decided no later than at the Report Planning Meeting) or due to PK non-evaluability (as revealed during data analysis).

The GPPASI is an adaptation for Generalized Pustular Psoriasis (GPP) patients of the Psoriasis Area and Severity Index (PASI), an established measure of severity and area of psoriatic lesions in patients with psoriasis. It is a tool which provides a numeric scoring for patients overall GPP disease state, ranging from 0 (no disease) to 72 (worse disease state). It is a linear combination of percent of surface area of skin that is affected and the severity of erythema, pustules, and scaling (desquamation) over four body regions. % GPPASI change from baseline=100\* (GPPASI at baseline - GPPASI at post-baseline visit)/(GPPASI at baseline). For %GPPASI change, positive numbers show reduction in GPPASI with higher values representing a larger improvement or recovery of disease, while negative numbers show an increase in GPPASI, i.e. worsening of disease.

Time frame: At baseline and at Week 2.

Population: Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.

GPPGA relies on clinical assessment of the GPP patient's skin presentation. It is a modified Physician Global Assessment (PGA), a physician's assessment of psoriatic lesions, which has been adapted to the evaluation of GPP patients. The investigator (or qualified site personnel) scores the erythema, pustules and scaling of all psoriatic lesions from 0 - 4. Each component is graded separately, the average is calculated and the final GPPGA is determined from this composite score. A lower score then indicates a lesser severity, with 0 being clear and 1 being almost clear. To receive a score of 0 or 1, the patient should be afebrile, in addition to skin presentation requirements.

Time frame: At Week 2.

Population: Full Analysis Set No Response Imputed (FAS NRI). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. NRI: If there were data at the visits both immediately before and immediately after the visit with a missing outcome, a failure to achieve response imputed.