VYNE Therapeutics (VYNE) H.C. Wainwright 3rd Annual Autoimmune & Inflammatory Disease Virtual Conference summary

Platform overview and innovation

• Focused on developing BET inhibitors for autoimmune and inflammatory diseases, diverging from traditional oncology uses.

• Two main assets: Repibresib (topical, tissue-specific pan-BD BET inhibitor) and VYN202 (oral, highly selective BD2 BET inhibitor).

• Repibresib targets non-segmental vitiligo in Phase IIb; VYN202 is in Phase Ib for moderate to severe plaque psoriasis.

• BET inhibitors historically faced safety issues; new approaches aim to mitigate GI toxicity and thrombocytopenia.

• VYN202 designed for high BD2 selectivity, minimizing BD1-related side effects and improving safety.

Clinical development and trial progress

• Repibresib Phase IIb in vitiligo is fully enrolled with 180 patients across four arms, including three active doses.

• Top-line readout for the double-blind, vehicle-controlled portion expected mid-year; study includes a 12-month treatment window.

• Primary endpoint is F-VASI50 (50% improvement in facial vitiligo lesions), with secondary endpoints including F-VASI75 and T-VASI.

• VYN202 completed Phase Ia SAD-MAD with strong safety and PK results; Phase Ib proof of concept in psoriasis underway.

• VYN202 Phase Ib is a 12-week study to assess safety, exposure, and exploratory efficacy, with results expected by year-end.

Differentiation, market opportunity, and future plans

• Repibresib’s topical delivery and once-daily dosing offer advantages over systemic JAK inhibitors, especially for localized vitiligo.

• Only one approved drug for vitiligo; Repibresib could become first-line or adjuvant therapy if data holds.

• Both assets have potential for broader indications beyond current targets, with preclinical data supporting expansion.

• Careful trial design, including longer treatment windows and biomarker analysis, aims to de-risk development.

• Key catalysts include mid-year Phase IIb readout for Repibresib and year-end Phase Ib data for VYN202.