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Positive Biomarker Data Shared Following Phase 1b Trial of VYN201 in Vitiligo


Researchers said this data is demonstrative of VYN201's positive effects on multiple vitiligo-related biomarkers.

Drobot Dean/Adobe Stock
Drobot Dean/Adobe Stock

VYNE Therapeutics announced today positive biomarker data stemming from its successful phase 1b trial of BET inhibitor VYN201 for vitiligo.

Positive results from the phase 1b trial of VYN201 were reported in October 2023. The trial aimed to assess safety, tolerability, pharmacokinetics, and efficacy of once-daily topical VYN201.

“We are encouraged by these new biomarker data from the Phase 1b trial for VYN201, building on our recently announced positive clinical results,” said David Domzalski, president and CEO of VYNE, in a press release. “We’ve made considerable progress in advancing this program, and we look forward to initiating a Phase 2b trial evaluating VYN201 in patients with both active and stable nonsegmental vitiligo in the second quarter of this year.”

The 16-week open-label study involved 29 patients with active nonsegmental vitiligo across three dose cohorts (0.5%, 1.0%, and 2.0%). Skin biopsies from selected vitiligo lesions were taken before and after 8 weeks of treatment, revealing promising outcomes.

In the 2.0% dose cohort, VYN201 demonstrated significant biological activity, particularly in modulating key biomarkers associated with vitiligo severity and progression.

Matrix metalloproteinase-9 (MMP-9), an inflammatory biomarker linked to melanocyte loss in vitiligo, was downregulated by 40.8% at week 8 compared to baseline.

Additionally, VYN201 induced the upregulation of melanocyte-related transcription factors such as SOX10, LEF1, β-Catenin, and MITF, indicating potential benefits for melanocyte proliferation, melanogenesis recovery, and skin re-pigmentation.

The 2.0% dose cohort exhibited more pronounced biomarker modulation, suggesting a dose-response relationship.

“VYN201’s potential to reduce melanocyte detachment by downregulating MMP-9 and by upregulating the expression of proteins relevant to melanocyte proliferation and melanogenesis is promising, and I look forward to the continued advancement of VYN201 as a potential differentiated therapy for vitiligo,” said Amit Pandya, MD, former President of the Global Vitiligo Foundation and dermatologist in the Department of Dermatology of Palo Alto Foundation Medical Group, in a press release.


VYNE Therapeutics. VYNE Therapeutics announces positive biomarker data from successful phase 1b trial of VYN201 for the treatment of vitiligo. BioSpace. January 10, 2024. Accessed January 10, 2024. https://www.biospace.com/article/releases/vyne-therapeutics-announces-positive-biomarker-data-from-successful-phase-1b-trial-of-vyn201-for-the-treatment-of-vitiligo/#:~:text=The%20data%20show%20that%20BET,were%20announced%20in%20October%202023

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