Trial Did Not Meet Primary Endpoint or Key Secondary Endpoint of F-VASI50 and F-VASI75
Nominally Statistically Significant Effects Observed in Key Secondary and Exploratory Endpoints of Change from Baseline in F-VASI and T-VASI at 3% Concentration
Company Will Terminate Extension Phase of Trial and Seek External Partner for Continued Development of Repibresib
BRIDGEWATER, N.J., July 30, 2025 (GLOBE NEWSWIRE) — VYNE Therapeutics Inc. (Nasdaq: VYNE) (“VYNE” or the “Company”), a clinical-stage biopharmaceutical company focused on developing differentiated therapies to treat chronic inflammatory and immune-mediated conditions with high unmet need, today announced topline results from its Phase 2b trial evaluating Repibresib gel in nonsegmental vitiligo.
The trial, which evaluated 177 subjects, did not meet its primary endpoint of the proportion of subjects achieving an improvement in Facial Vitiligo Area Scoring Index of at least 50% from baseline (F-VASI50) at week 24 compared to vehicle. The trial also missed a key secondary endpoint of F-VASI75. However, the trial showed a nominally statistically significant treatment effect for the highest dose cohort in a key secondary endpoint, percent change-from-baseline (CFB) in F-VASI score at week 24 compared to vehicle (Repibresib 3%: -43.6% v. Vehicle: -25.6%), and an exploratory endpoint of percent CFB in T-VASI score at week 24 compared to vehicle (Repibresib 3%: -28.3% v. Vehicle: -16.2%). In evaluating the results of the trial, the Company believes the results were impacted by an unusually high vehicle effect and further impacted by a higher-than-expected dropout rate in the active arms of the trial relative to vehicle (Repibresib 3%: 36.6%; Repibresib 2%: 30.2%; Repibresib 1%: 26.1% and vehicle: 10.6%). Based on these data, the company will discontinue treatment in the ongoing extension phase of the trial and will terminate the trial.
The Phase 2b trial was a randomized double-blind, vehicle-controlled, multi-center trial that evaluated the safety and efficacy of Repibresib dosed once daily (QD) at 1%, 2% and 3% concentrations, versus vehicle in 177 subjects (mITT population) with nonsegmental vitiligo. The trial was conducted at 45 sites in North America and assessed various efficacy endpoints, including the primary endpoint of the proportion of subjects achieving F-VASI50 at Week 24 versus vehicle, as well as key secondary endpoints, which included the proportion of subjects achieving F-VASI75 at Week 24, percent CFB in F-VASI score at week 24 and exploratory endpoints including percent CFB in T-VASI score at week 24.
“We are disappointed with the results of our Phase 2b trial, which were impacted by an unexpectedly high treatment effect in the vehicle arm and a high discontinuation rate in the active arms,” said David Domzalski, President and Chief Executive Officer of VYNE. “Although we missed our F-VASI50 and F-VASI75 endpoints, we did see a meaningful reduction in the percent change from baseline in both F-VASI and T-VASI for our highest dose and are conducting a thorough evaluation of the full dataset to analyze any other data that may inform the results and our strategic next steps. Vitiligo remains an area of significant unmet need and we are grateful to the patients, investigators, and clinical staff who participated in the trial. Despite this outcome, we remain confident in the potential of our InhiBET™ BET inhibitor platform as a promising and innovative mechanistic approach for the treatment of a broad range of serious immune-mediated diseases. We intend to seek a development and commercialization partner for Repibresib and we will provide an update on our plans, including those for our oral BET inhibitor VYN202, in the coming weeks.”
While the Company has not finalized its full financial results for the quarter ended June 30, 2025, the Company expects to report that it had cash, cash equivalents and investments of approximately $39.6 million (unaudited) as of June 30, 2025.
About Repibresib
Repibresib is a pan-bromodomain BET inhibitor designed to be locally administered as a “soft” drug to address diseases involving multiple, diverse inflammatory cell signaling pathways, while providing low systemic exposure. Repibresib has produced consistent reductions in pro-inflammatory and disease-related biomarkers and improvements in disease severity in several preclinical models (using several different routes of administration).
About VYN202
VYN202 is an innovative, oral small molecule BET inhibitor that has potential class-leading selectivity and potency for BD2 vs. BD1. By maximizing BD2 selectivity, VYNE believes VYN202 has the potential to be a differentiated, more conveniently administered, non-biologic treatment option for both acute control and chronic management of immuno-inflammatory indications, in which the damaging effects of unrestricted inflammatory signaling activity are common.
About VYNE Therapeutics Inc.
VYNE is a clinical-stage biopharmaceutical company focused on developing differentiated therapies to treat chronic inflammatory and immune-mediated conditions with high unmet need. VYNE’s unique and proprietary BET inhibitors, which comprise its InhiBET™ platform, are designed to overcome limitations of early generation BET inhibitors by leveraging alternative routes of administration and enhanced selectivity.