VYNE Therapeutics (VYNE) TD Cowen 45th Annual Healthcare Conference summary

Platform and pipeline overview

• Focus on novel BET inhibitors for autoimmune diseases, with two main assets: topical repibresib (VYN201) and oral VYN202.

• Repibresib is in a phase II-B trial for non-segmental vitiligo; VYN202 is in phase I-B for moderate to severe psoriasis.

• BET inhibitors have historically been used in oncology, but these programs target autoimmune and inflammatory conditions.

• VYN202 is a highly selective BD2 BET inhibitor, aiming for improved safety and efficacy.

Clinical trial updates and results

• Phase I-B for repibresib in vitiligo showed clear dose response and strong results at higher doses, supporting phase II-B progression.

• Biomarker analysis indicated upregulation of Wnt beta-catenin pathway genes in responders, suggesting skin recovery.

• Repibresib demonstrated minimal systemic exposure and no evidence of thrombocytopenia, addressing key safety concerns.

• Phase II-B for vitiligo uses a gel formulation, includes both active and stable disease patients, and runs for up to a year.

• Enrollment for phase II-B completed in January; results expected mid-2025.

Efficacy and safety benchmarks

• Primary endpoint for phase II-B is FASI 50 (50% improvement in facial vitiligo severity); FASI 75 is a key secondary endpoint.

• Study design closely mirrors that of Opzelura, the current standard topical treatment.

• Once-daily dosing is a key differentiator versus twice-daily Opzelura, potentially improving compliance.

• Safety profile aims to avoid black box warnings associated with JAK inhibitors.