A poster from the 2022 American Academy of Dermatology Annual Meeting presented subgroup analyses from the Effisayil 1 study that showed the efficacy of spesolimab was consistent across all prespecified patient populations, including those with or without IL-36RN mutations.
A group of investigators conducted a subgroup analysis of the primary and key secondary end points from the Effisayil 1 (NCT03782792) study.1 This study was a multicenter, randomized, double-blind, placebo-controlled trial of spesolimab (BI655130; Boehringer Ingelheim)—a humanized monoclonal antibody that targets interleukin (IL)-36—as treatment for generalized pustular psoriasis (GPP) flares.
The primary end point was a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) pustulation sub score of 0, defined as no visible pustules, and the key secondary end point was the percentage of patients who achieved a GPPGA total score of 0 or 1—clear or almost clear skin. In the study, 54% of patients who were treated with spesolimab achieved the primary end point vs 6% of placebo (one-sided p < .001) and for the key secondary end point it was 43% and 11%, respectively (one-sided p = .0118).2
In the Effisayil 1 study, there were more adverse events (AEs) reported in the spesolimab treated patients compared to placebo, 66% vs 56% respectively, with infections appearing in 17% of spesolimab treated patients at week 1. There were serious AEs in 6% of spesolimab treated patients at week 1. Additionally, 2 patients who were receiving treatment had drug reactions of eosinophilia and systemic symptoms.3
The analysis looked at different subgroups which were created if at least 2 categories of the subgroup included greater than or equal to 5 patients: sex, age, race, BMI, GPPGA pustulation sub score at baseline, GPPGA total score at baseline, Japanese Dermatological Association (JDA) GPP severity score at baseline, presence of plaque psoriasis at baseline, and IL36RN status, according to the poster.
Overall, the spesolimab treatment efficacy in each patient subgroup was similar to those of the general population for both the primary and key secondary end points, but many of these subgroups did not have many patients.
The FDA has accepted a Biologics License Application (BLA) and granted priority review for spesolimab for the treatment of GPP flares. The FDA also granted spesolimab an orphan drug designation for the treatment of GPP and breakthrough therapy designation for the treatment of GPP flares in adults.4
1. Burden AD, Okubo Y, Zheng M, et al. Efficacy of spesolimab for the treatment of GPP flares across prespecified patient subgroups in the Effisayil 1 study. Poster. Presented at: 2022 American Academy of Dermatology Annual Meeting, March 25-29, in Boston, Massachusetts.
2. Choon SE, Lebwohl MG, Marrakchi S, et al. Study protocol of the global Effisayil 1 Phase II, multicenter, randomized, double-blind, placebo-controlled trial of spesolimab in patients with generalized pustular psoriasis presenting with an acute flare. BMJ Open. 2021;11(3):e043666. doi:10.1136/bmjopen-2020-043666
3. New spesolimab data showed significant improvement in patients with generalized pustular psoriasis (GPP) flares. Accessed April 1, 2022. https://www.boehringer-ingelheim.us/press-release/new-spesolimab-data-showed-significant-improvement-patients-generalized-pustular
4. New data released on spesolimab treatment for gpp. Dermatology Times. Accessed April 1, 2022. https://www.dermatologytimes.com/view/new-data-released-on-spesolimab-treatment-for-gpp