VYNE Therapeutics Announces Topline Results from Phase 2b Trial with Repibresib Gel in Nonsegmental Vitiligo

Core Insights - VYNE Therapeutics announced that its Phase 2b trial for Repibresib gel in nonsegmental vitiligo did not meet its primary endpoint of achieving at least a 50% improvement in the Facial Vitiligo Area Scoring Index (F-VASI50) at week 24 compared to the vehicle group [1][2][4] - Despite missing the primary and key secondary endpoints, nominally statistically significant effects were observed in some exploratory endpoints, indicating potential efficacy at the highest dose [2][4] - The company plans to terminate the ongoing extension phase of the trial and seek an external partner for the continued development of Repibresib [1][4] Trial Details - The Phase 2b trial involved 177 subjects and was a randomized, double-blind, vehicle-controlled study assessing the safety and efficacy of Repibresib at concentrations of 1%, 2%, and 3% [3] - The trial was conducted across 45 sites in North America and evaluated multiple efficacy endpoints, including the primary endpoint of F-VASI50 and key secondary endpoints [3] Efficacy Results - The trial did not meet the primary endpoint, with only 19.5% of subjects in the 3% Repibresib group achieving F-VASI50 compared to 23.4% in the vehicle group [5] - For the key secondary endpoint of F-VASI75, 9.8% of subjects in the 3% group achieved this compared to 6.4% in the vehicle group [6] - The percent change from baseline in F-VASI score at week 24 showed a mean reduction of -43.6% for the 3% Repibresib group versus -25.6% for the vehicle group, indicating a statistically significant treatment effect [7] - The percent change from baseline in T-VASI score at week 24 also showed a significant reduction of -28.3% for the 3% Repibresib group compared to -16.2% for the vehicle group [8] Safety and Tolerability - The trial reported a higher rate of treatment-emergent adverse events (TEAEs) in subjects receiving Repibresib compared to the vehicle group, with application site pain being the most common adverse event [9] - Eight subjects discontinued due to adverse events in the Repibresib group, while none in the vehicle group experienced this [9] Financial Position - As of June 30, 2025, VYNE expects to report approximately $39.6 million in cash, cash equivalents, and investments [4]