LB1056 Phase 1b: Translational Evaluation of VYN201, a Pan-BD BET Inhibitor, for the Treatment of Non-Segmental Vitiligo

The Phase 1b study evaluated VYN201, a topical pan-BD BET inhibitor, for treating non-segmental vitiligo in 29 patients over 16 weeks. The study assessed safety, tolerability, pharmacokinetics, and exploratory efficacy across three dose cohorts (0.5%, 1%, and 2%). Most treatment-emergent adverse events were mild or moderate, with only one severe case of application site pain in the 2% cohort. Efficacy was dose-related, with the 2% cohort showing a mean F-VASI improvement of -39.004% and significant upregulation of melanocyte-related transcription factors, indicating potential for re-pigmentation. VYN201 demonstrated a promising safety profile and potential as a differentiated treatment for active non-segmental vitiligo.