Real-world Experience with Spesolimab in Chinese Patients with GPP

Generalized pustular psoriasis (GPP) is a heterogeneous, neutrophilic inflammatory disease associated with chronic skin and systemic symptoms, and potentially life-threatening flares. We present safety and efficacy data from a real-world expanded access program (EAP) in China (12 sites; May 2022–July 2023), which provided early access to spesolimab (an anti-interleukin-36 receptor antibody) for patients not eligible for clinical trials and with no alternative treatment. Spesolimab was later approved in China for the treatment of GPP flares in adults (intravenous [IV] formulation [December 2022])1 and the reduction of occurrence of GPP flare in adolescents from 12 years of age with a body weight of ≥40kg and adults (subcutaneous formulation [March 2024]).2 Patients received a 900mg dose of spesolimab IV for flare treatment (optional second dose after 1 week for persistent flare symptoms). Treatment-emergent adverse events (TEAEs), serious AEs (SAEs), and AEs of special interest (AESIs) were recorded up to Week 16 after the last spesolimab dose. Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) pustulation subscore (PS) and total score (TS) were recorded at baseline and Week 1. Thirty-nine patients (female, n=23) entered the EAP; 12 patients had available GPPGA scores at baseline (PS: 1, n=2; 2, n=2; 3, n=5; 4, n=3; TS: 2, n=4; 3, n=6; 4, n=2). Mean patient age: 39 years (range: 18–65); mean body mass index: 22.9kg/m2. Flare triggers: treatment withdrawal (n=4), pregnancy (n=2), infection (n=1), other (n=7), and unknown (n=25). Thirty-six patients had ≥1 comorbidity and received ≥1 concomitant medication, including immunosuppressants, corticosteroids, topicals, and biologic therapies. Thirty patients (77%) experienced TEAEs, mostly mild/moderate (n=26). One patient had pneumonia and COVID-19 (SAE, AESI), one patient had pneumonia (SAE, AESI) and respiratory failure (SAE), and one patient had worsening of GPP (SAE). No AEs led to discontinuation/death. At Week 1, 9/12 patients (75%) had no pustules (GPPGA PS=0); the other 3 patients had a GPPGA PS of 1 or 2. Six of 12 patients (50%) had a GPPGA TS of 0/1 at Week 1 (clear/almost clear skin). Spesolimab was well tolerated in Chinese patients with GPP, including those with a GPPGA TS of 2, a population not included in EFFISAYIL 1.3 Most patients (75%) demonstrated pustular clearance and AEs were consistent with the context of the EAP (COVID-19 pandemic) and comparable to those in the EFFISAYIL 1 trial.3 Funding: Study supported/funded by Boehringer Ingelheim.