Rapid and sustained improvements in Generalized Pustular Psoriasis Physician Global Assessment scores with spesolimab for treatment of generalized pustular psoriasis flares in the randomized, placebo-controlled Effisayil 1 study

Abstract

Effisayil 1 was a randomized, placebo-controlled study of spesolimab, an anti-IL-36 receptor antibody, in patients presenting with a generalized pustular psoriasis (GPP) flare. We present the effects of spesolimab over the 12-week study. The primary endpoint was a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) pustulation subscore of 0 at Week 1. Patients (N=53) were randomized (2:1) to receive a single intravenous dose of 900 mg spesolimab or placebo on Day 1. Patients could receive open-label spesolimab for persistent flare symptoms on Day 8. Most patients receiving spesolimab achieved a GPPGA pustulation subscore of 0 (60.0%) and GPPGA total score of 0 or 1 (60.0%) by Week 12. In patients randomized to placebo who received open-label spesolimab, the proportion with a GPPGA pustulation subscore of 0 increased from 5.6% at Day 8 to 83.3% at Week 2. No factors predictive of spesolimab response were identified in patient demographics or clinical characteristics. The effect of initial randomization was not determined conventionally beyond Week 1 due to patients receiving OL spesolimab. Rapid control of GPP flare symptoms with spesolimab was sustained over 12 weeks, further supporting its potential use as a therapeutic option for patients.