David Rosmarin, MD

Efficacy and Safety of Ruxolitinib Cream for the Treatment of Vitiligo: 24-Week Results From 2 Randomized, Double-Blind Phase 3 Studies

Two multicenter, phase 3, double-blind, controlled studies (TRuE-V1 [NCT04052425]; TRuE-V2 [NCT04057573]) of ruxolitinib cream, a Janus kinase (JAK)1/JAK2 inhibitor, were conducted in patients ≥12 years old with nonsegmental vitiligo in North America and Europe. Patients were randomized 2:1 to receive 1.5% ruxolitinib cream or vehicle twice daily for 24 weeks. TRuE-V1/TRuE-V2 included 221/228 patients who received ≥1 dose of ruxolitinib cream; 109/115 patients received vehicle. Mean ages were 40/39 years; 11% of patients in both studies were adolescents. A facial Vitiligo Area Scoring Index improvement of ≥75% (F-VASI75) at Week 24 (primary endpoint) was achieved by 29.9%/29.9% of patients who applied ruxolitinib cream vs 7.5%/12.9% who applied vehicle (both *P*<0.01). F-VASI50 at Week 24 was achieved by 51.5%/51.4% and 17.2%/23.4% (both *P*<0.0001). Ruxolitinib cream demonstrated clinically meaningful superiority vs vehicle for all primary and key secondary endpoints and was well tolerated in these large phase 3 studies.

Amit G. Pandya, MD, Palo Alto Foundation Medical Group, Mountain View, CA, USA; University of Texas Southwestern Medical Center, Dallas, TX, USA.

Pearl Grimes, MD, The Vitiligo & Pigmentation Institute of Southern California, Los Angeles, CA, USA.

John E. Harris, MD, PhD, University of Massachusetts Medical School, Worcester, MA, USA.

Seemal R. Desai, MD, University of Texas Southwestern Medical Center, Dallas, TX, USA; Innovative Dermatology, Plano, TX, USA.

Mark Lebwohl, MD, Icahn School of Medicine at Mount Sinai, New York, NY, USA.

Mireille Ruer-Mulard, MD, Office of Mireille Ruer-Mulard, MD, Martiques, France.

Thierry Passeron, MD, PhD, Centre Hospitalier Universitaire de Nice, Université Côte d'Azur, Nice, France; INSERM U1065, C3M, Université Côte d'Azur, Nice, France.

Julien Seneschal, MD, PhD, Department of Dermatology and Pediatric Dermatology, National Reference Center for Rare Skin Disorders, Hôpital Saint-André, INSERM, BMGIC, U1035, F-33000, Bordeaux, France.

Albert Wolkerstorfer, MD, PhD, Amsterdam University Medical Center, Amsterdam, Netherlands.

Deanna Kornacki, PhD, Incyte Corporation, Wilmington, DE, USA.

Kang Sun, PhD, Incyte Corporation, Wilmington, DE, USA.

Kathleen Butler, MD, Incyte Corporation, Wilmington, DE, USA.

Khaled Ezzedine, MD, PhD, Henri Mondor University Hospital and Université Paris-Est Créteil Val de Marne, Paris, France.

DR has received honoraria as a consultant for AbbVie, Abcuro, AltruBio, Boehringer Ingelheim, Bristol Meyers Squibb, Celgene, Concert, Dermavant Sciences, Dermira, Janssen, Kyowa Kirin, Lilly, Novartis, Pfizer, Regeneron Pharmaceuticals, Sanofi, Sun Pharmaceuticals, UCB, and VielaBio; has received research support from AbbVie, Amgen, Bristol Myers Squibb, Celgene, Dermira, Galderma, Incyte Corporation, Janssen, Lilly, Merck, Novartis, Pfizer, and Regeneron Pharmaceuticals; and has served as a paid speaker for AbbVie, Amgen, Celgene, Janssen, Lilly, Novartis, Pfizer, Regeneron Pharmaceuticals, and Sanofi.

Coauthor Disclosures:

AGP has served as an investigator for Aclaris Therapeutics, Immune Tolerance Network, Incyte Corporation, and Pfizer; a consultant for AbbVie, Arcutis, Avita Medical, Chromaderm, Immune Tolerance Network, Incyte Corporation, Pfizer, TWi, Viela Bio, and Villaris; and holds stock options for Tara Medical and Zerigo Health. 

PG has served as a consultant for Aclaris Therapeutics, Clarify Medical, DermaForce, Incyte Corporation, Proctor & Gamble, and Versicolor Technologies and a principal investigator for Aclaris Therapeutics, Allergan/SkinMedica, Clinuvel Pharmaceuticals, Incyte Corporation, Johnson & Johnson, L’Oreal, Merz Pharma, Pfizer, Thync Global Inc., and VT Cosmetics.

JEH has served as a consultant for AbbVie, Aclaris Therapeutics, BiologicsMD, EMD Serono, Genzyme/Sanofi, Janssen, Pfizer, Rheos Medicines, Sun Pharmaceuticals, TeVido BioDevices, The Expert Institute, 3rd Rock Ventures, and Villaris Therapeutics; has served as an investigator for Aclaris Therapeutics, Celgene, Dermira, EMD Serono, Genzyme/Sanofi, Incyte Corporation, LEO Pharma, Pfizer, Rheos Medicines, Stiefel/GlaxoSmithKline, Sun Pharmaceuticals, TeVido BioDevices, and Villaris Therapeutics; holds equity in Rheos Medicines, TeVido BioDevices, and Villaris Therapeutics; is a scientific founder of Villaris Therapeutics and NIRA Biosciences; and has patents pending for IL-15 blockade for treatment of vitiligo, JAK inhibition with light therapy for vitiligo, and CXCR3 antibody depletion for treatment of vitiligo.

SRD has received fees and/or honoraria as a consultant for Almirall, Avita, Bristol Myers Squibb, Cassiopea SpA, Dermavant Sciences, Dermira, Ferndale Laboratories, Foamix, Galderma Laboratories LP, Incyte Corporation, MC2 Therapeutics, Ortho Dermatologics, Pfizer, Scientis, Sente Labs, SkinCeuticals LLC, UCB, and Verrica Pharmaceuticals; has received stock options as a consultant for Gore Range Capital; has received honoraria as a speaker for Almirall and Ortho Dermatologics; has received grants/research funding as an investigator for AbbVie, AOBiome LLC, Atacama Therapeutics, Brickell Biotech, Dermavant Sciences, Incyte Corporation, Novan, and SkinMedica; has served as an advisory board member for the Foundation for Research & Education of Dermatology; is a stockholder of Gore Range Capital; and is a shareholder in PDP of Texas.

ML is an employee of Mount Sinai Hospital, which receives research funds from AbbVie, Amgen, Arcutis, Avotres, Boehringer Ingelheim, Dermavant, Eli Lilly, Incyte Corporation, Janssen Research & Development, LLC, Ortho Dermatologics, Regeneron, and UCB, Inc; and is a consultant for Aditum Bio, Almirall, AnaptysBio, Arcutis, Aristea, Arrive Technology, Avotres Therapeutics, BioMX, Boehringer Ingelheim, Bristol Myers Squibb, Cara Therapeutics, Castle Biosciences, Corrona, Dermavant Sciences, Dr. Reddy’s Laboratories, Evelo, Evommune, Facilitate International Dermatologic Education, Forte, Foundation for Research and Education in Dermatology, Helsinn, LEO Pharma, Meiji Seika Pharma, Mindera, Pfizer, and Verrica.

MR-M is an investigator for AbbVie, Almirall, Amgen, Bay Pharma, Dermira, Eli Lilly, Fujisawa, Galderma, GlaxoSmithKline, Incyte Corporation, Janssen, LEO Pharma, Novartis, Pierre Fabre, Sanofi, Serono, and UCB; and is an advisory board member for AbbVie, Janssen, LEO Pharma, and Novartis.

TP has received grants and/or honoraria from AbbVie, ACM Pharma, Almirall, Amgen, Astellas, Bristol Myers Squibb, Celgene, Galderma, Genzyme/Sanofi, GlaxoSmithKline, Incyte Corporation, Janssen, LEO Pharma, Eli Lilly, Novartis, Pfizer, Sun Pharma, and UCB; is the cofounder of YUKIN Therapeutics; and has patents on WNT agonists or GSK3b antagonist for repigmentation vitiligo and on the use of CXCR3B blockers in vitiligo.

JS has served as an investigator, speaker, or consultant for AbbVie, Calypso Biotech, Incyte Corporation, LEO Pharma, Novartis, Pfizer, and Pierre Fabre.

AW is a dermatologist at the Netherlands Institute for Pigment Disorders and the Department of Dermatology at the Amsterdam University Medical Center; has served as principal investigator for Avita Medical, Incyte Corporation, and Novartis; has served as an advisory board member for Incyte Corporation; has received research grants from Avita Medical and Lumenis; and has received devices from Humeca and PerfAction.

DK, KS, and KB are employees and shareholders of Incyte Corporation.

KE is a consultant for AbbVie, Incyte Corporation, La Roche-Posay, Pfizer, Pierre Fabre, Sanofi, and Viela Bio.