VYNE Therapeutics Announces Positive Data from Phase 1b Trial for Novel BET Inhibitor VYN201 in Patients with Nonsegmental Vitiligo - Seite 2

“Vitiligo patients with active disease face an uphill battle compared to those with stable vitiligo and may require aggressive treatment in order to avoid potentially permanent loss of pigmentation,” said Amit Pandya, M.D., former President of the Global Vitiligo Foundation and Staff Dermatologist, Department of Dermatology, Palo Alto Foundation Medical Group. “VYN201’s ability to halt the progression of depigmentation and demonstrate repigmentation in these difficult-to-treat patients as observed in this trial is very promising. These data merit the continued investigation of VYN201’s potential as a differentiated therapy for vitiligo.”

Key results include:

• Proof-of-concept achieved:
  • Significant clinical improvement observed in 1% and 2% cohorts with rapid onset of action and a dose-dependent response. Mean percentage reduction in F-VASI score from baseline after 16 weeks of treatment was:
    • 7.5% for the 0.5% cohort (n=10), with 20% of patients achieving a ≥25% improvement in F-VASI score from baseline (“F-VASI25”)
    • 30.3% for the 1.0% cohort (n=10), with 50% of patients achieving F-VASI25 and 30% of patients achieving a ≥50% improvement in F-VASI score from baseline (“F-VASI50”)
    • 39.0% for the 2.0% cohort (n=9), with 67% of patients achieving F-VASI25 and 33% of patients achieving F-VASI50, including one patient who achieved a ≥75% improvement in F-VASI score from baseline
    • The mean percentage reduction in F-VASI score from baseline for each of the 1.0% and 2.0% cohorts was statistically significant (p = 0.0137 and p = 0.0077¹, respectively) compared to the 0.5% cohort at week 16, indicative of a dose response
• Safety, Tolerability and Pharmacokinetics: Topical VYN201 was generally well-tolerated with a favorable safety profile in vitiligo patients at all dose levels with no serious adverse events reported and no abnormal laboratory values suggestive of low or reducing platelet counts. All treatment emergent adverse events have been classified as mild or moderate with no dose-dependent trends observed to date. All mean local skin tolerability assessment scores were less than 0.30 on a severity scale 0 (“None”) to 3 (“Severe”).