@article{03eab14d5c6d427492c7d747092351d4,
title = "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 = "Background: Effisayil 1 was a randomized, placebo-controlled study of spesolimab, which is an anti-IL-36 receptor antibody, in patients presenting with a generalized pustular psoriasis flare. Objective: To assess the effects of spesolimab over the 12-week study. Methods: The primary endpoint of the study was 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. Results: 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 on day 8, the proportion with 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. Limitations: The effect of initial randomization was not determined conventionally beyond week 1 due to patients receiving open-label spesolimab. Conclusion: Rapid control of generalized pustular psoriasis flare symptoms with spesolimab was sustained over 12 weeks, further supporting its potential use as a therapeutic option for patients.",
keywords = "GPP, GPPGA, IL-36, IL-36R, pustular psoriasis, spesolimab",
author = "Elewski, {Boni E.} and Lebwohl, {Mark G.} and Anadkat, {Milan J.} and Jonathan Barker and Kamran Ghoreschi and Shinichi Imafuku and Ulrich Mrowietz and Ling Li and Manuel Quaresma and Christian Thoma and Herv{\'e} Bachelez",
note = "Funding Information: Funding sources: The study was supported and funded by Boehringer Ingelheim.The authors met the criteria for authorship as recommended by the International Committee of Medical Journal Editors (ICMJE). The authors did not receive payments related to the development of this manuscript. Boehringer Ingelheim was given the opportunity to review the manuscript for medical and scientific accuracy as well as intellectual property considerations. Dr Elewski is an investigator for AbbVie, Amgen (previously Celgene), AnaptysBio, Bausch Health (formerly Valeant Pharmaceuticals), Boehringer Ingelheim, Bristol Myers Squibb, Eli Lilly, Incyte, LEO Pharma, Menlo, Merck, Novartis, Pfizer, Regeneron, Sun Pharmaceutical Industries, UCB, and Vanda; and is a consultant for Amgen (previously Celgene), Arcutis, Bausch Health (formerly Valeant Pharmaceuticals), Boehringer Ingelheim, Bristol Myers Squibb, Eli Lilly, LEO Pharma, Novartis, and UCB. Dr Lebwohl is an employee of Mount Sinai and receives research funds from AbbVie, Amgen, Arcutis, Avotres, Boehringer Ingelheim, Cara Therapeutics, Dermavant Sciences, Eli Lilly, Incyte, Janssen Research & Development, LLC, Ortho Dermatologics, Regeneron, and UCB, Inc.; and is a consultant for Aditum Bio, Almirall, AltruBio Inc., AnaptysBio, Arcutis, Arena Pharmaceuticals, Aristea Therapeutics, Avotres Therapeutics, BiomX, Brickell Biotech, Boehringer Ingelheim, Bristol Myers Squibb, Cara Therapeutics, Castle Biosciences, Celltrion, CorEvitas, Dermavant Sciences, Evommune, Inc., Facilitation of International Dermatology Education, Forte Biosciences, Foundation for Research and Education in Dermatology, Hexima Ltd., Incyte, LEO Pharma, Meiji Seika Pharma, Mindera, Pfizer, Seanergy, Trevi, Vial, and Verrica. Dr Anadkat declares paid activities as a current advisor or consultant for BioLinq, Boehringer Ingelheim, Eli Lilly, Novocure, Protagonist, and Springworks; has served as prior consultant or advisor for AbbVie, Adgero, Amgen, Aspire Bariatrics, AstraZeneca, Biogen, Bristol Myers Squibb, ImClone, Innovaderm, OnQuality, Therakos, Veloce, and UCB; and has served as an investigator for AbbVie, AnaptysBio, Biogen, Boehringer Ingelheim, ChemoCentryx, Eli Lilly, Incyte, InflaRx, Hana Biosciences, Lutris, Moonlake, Novartis, OnQuality, UCB, Veloce, XBiotech, and Xoma. Prof Barker declares having attended advisory boards and/or received consultancy fees and/or spoken at sponsored symposia and/or received grant funding from AbbVie, Almirall, Amgen, AnaptysBio, Boehringer Ingelheim, Bristol Myers Squibb, Celgene, Eli Lilly, Janssen, LEO Pharma, Novartis, Pfizer, Samsung, Sienna, Sun Pharmaceutical Industries, and UCB. Prof Ghoreschi declares having attended advisory boards and/or received consultancy fees and/or spoken at sponsored symposia and served as an investigator for AbbVie, Boehringer Ingelheim, Bristol Myers Squibb, Eli Lilly, Janssen-Cilag, LEO Pharma, Novartis, Pfizer, and UCB. Prof Imafuku has served as a consultant and/or paid speaker for and/or accepted a research grant from and/or participated in clinical trials sponsored by companies including AbbVie, Amgen, Eisai, Eli Lilly, Janssen, Kyowa Kirin, LEO Pharma, Maruho Pharmaceutical, Mitsubishi Tanabe, Novartis, Sun Pharmaceutical Industries, Taiho Yakuhin Koryo, Torii Yakuhin, and UCB. Prof Mrowietz has been an advisor and/or received speaker's honoraria and/or received grants and/or participated in clinical trials for AbbVie, Aditxt, Almirall, Amgen (previously Celgene), Aristea, Boehringer Ingelheim, Bristol Myers Squibb, Dr. Reddy's Laboratories, Eli Lilly, Foamix, Formycon, Immunic, Janssen, LEO Pharma, Medac, MetrioPharm, Novartis, Phi-Stone, Pierre Fabre, Sanofi Aventis, and UCB. Li, Quaresma, and Dr Thoma are employees of Boehringer Ingelheim. Prof Bachelez reports receiving grants or contracts from Boehringer Ingelheim, Bristol Myers Squibb, Janssen, LEO Pharma, Novartis, and Pfizer; consulting fees from AbbVie, Almirall, Anaptysbio, Aristea Therapeutics, Biocad, Boehringer Ingelheim, Bristol Myers Squibb, Celgene, Dermavant, Innovaderm, Janssen, Kyowa Kirin, LEO Pharma, Lilly, Mylan, Novartis, UCB, and Xion Pharmaceuticals; honoraria from AbbVie, Boehringer Ingelheim, Celgene, and LEO Pharma; advisory board participation for Avillion; and meeting attendance support from Janssen, Novartis, and UCB.Writing, editorial, and formatting support was provided by James Parkinson, PhD, at OPEN Health Communications (London, UK), and was contracted and funded by Boehringer Ingelheim. Funding Information: Funding sources: The study was supported and funded by Boehringer Ingelheim . Publisher Copyright: {\textcopyright} 2023 American Academy of Dermatology, Inc.",
year = "2023",
month = jul,
doi = "10.1016/j.jaad.2023.02.040",
language = "English",
volume = "89",
pages = "36--44",
journal = "Journal of the American Academy of Dermatology",
issn = "0190-9622",
number = "1",
}