VYNE Therapeutics Announces Positive Results from Phase 1a Single & Multiple Ascending Dose Trial for Investigational Novel BET Inhibitor

VYNE Therapeutics Inc. recently announced positive results from the Phase 1a portion of its Phase 1a/b clinical trial evaluating its novel BET inhibitor, VYN201, for vitiligo.

In the Phase 1a portion of the study, single ascending and multiple ascending doses of VYN201 were applied topically once daily to 30 healthy volunteers in five dose cohorts for 2 weeks with a 1-week safety follow-up visit to evaluate the safety, tolerability, and pharmacokinetics of VYN201. Evaluated doses included VYN201 0.025%, 0.1%, 0.5%, 1.0% and 2.0% ointment strengths. The safety and tolerability results are summarized below:

• There were no serious adverse events and no dose adjustments were required.
• There were no clinically relevant treatment emergent adverse events, abnormal clinical laboratory results or electrocardiogram findings.
• No healthy volunteers withdrew from the trial for any reason.
• VYNE has selected 0.5%, 1.0%, and 2.0% ointment strengths for evaluation in the ongoing Phase 1b study evaluating VYN201 in non-segmental vitiligo patients.

In the Phase 1b portion of the study, up to 30 patients with a clinical diagnosis of non-segmental vitiligo will receive VYN201 once daily in three dose cohorts. The primary objective of the Phase 1b portion of the study will be to evaluate the safety and pharmacokinetics of VYN201. Exploratory efficacy of VYN201 in non-segmental vitiligo patients will also be evaluated, including pharmacodynamic biomarkers and photography.

“These preliminary safety results mark a significant milestone as we progress our first in-human clinical trial for VYN201 and advance the development of our BET inhibitor programs for immuno-inflammatory indications,” said David Domzalski, President and Chief Executive Officer of VYNE. “These safety data support our thesis for designing VYN201 as a locally-administered soft drug to minimize systemic exposure and maximize target engagement, and we look forward to receiving topline results from the Phase 1b portion of the study in the middle part of the year.”

VYN201 is a locally-administered pan-bromodomain BET inhibitor, designed as a “soft” drug to address diseases involving multiple, diverse inflammatory cell signaling pathways while providing low systemic exposure. To date, VYN201 has produced consistent reductions in pro-inflammatory and disease-related biomarkers, improvements in disease severity and a demonstrated local activity through several preclinical models. The company believes that these data suggest potential broad utility for VYN201 across multiple routes of administration.

Vitiligo is a chronic autoimmune depigmenting disorder of the skin, characterized by the loss of pigment producing cells known as melanocytes. Vitiligo is the most common depigmenting skin condition, with a prevalence estimated at 0.5-2% of the world population. There is currently only one FDA-approved product for the treatment of vitiligo.

BET proteins play a key role in regulating gene transcription via epigenetic interactions (“reading”), and recent research has determined a key role for these BET proteins in regulating B cell and T cell activation and subsequent inflammatory processes. As epigenetic readers, BET proteins regulate the recruitment of transcriptional factors that are key to the production of several pro-inflammatory cytokines. BET inhibitors have the potential to treat a range of immuno-inflammatory and fibrotic diseases by blocking pro-inflammatory cytokine transcription with additional potential in myeloproliferative neoplastic disorders.

VYNE’s mission is to improve the lives of patients by developing proprietary, innovative, and differentiated therapies for the treatment of immuno-inflammatory conditions. The company’s unique and proprietary bromodomain & extra-terminal (BET) domain platform includes lead programs VYN201 (pan-BETi) and VYN202 (selective-BETi), and access to a library of small molecule BET inhibitors for the potential treatment of immuno-inflammatory conditions licensed from Tay Therapeutics Limited. For more information, visit www.vynetherapeutics.com.