Harmony Biosciences (NASDAQ: HRMY) presented updated data from its Open-Label Extension (OLE) study of ZYN002 for Fragile X Syndrome (FXS) treatment. The study demonstrated clinically meaningful improvements in irritability-related symptoms in patients.
Key findings include:
Over 60% of participants achieved clinically meaningful improvement of ≥9 points on ABC-CFXS Irritability scores
After three years, ~73% of patients experienced clinically meaningful improvements
Treatment-related adverse events occurred in 12.9% of patients, with application site pain being most common (6.7%)
The study included 240 patients aged 3-17 years, with data collected from September 2018 to January 2024. The company expects topline data from its Phase 3 RECONNECT study in Q3 2025. Currently, there are no FDA-approved treatments for FXS, which affects approximately 80,000 individuals in the U.S.
Positive
Strong efficacy data with >60% of participants showing meaningful improvement in irritability scores
73% of patients showed sustained clinical improvements after 3 years
Low serious adverse event rate at 4.6% with minimal discontinuation (3.3%)
Potential first-mover advantage in untapped market of 80,000 FXS patients
Negative
FDA approval still pending with Phase 3 results not expected until Q3 2025
12.9% of patients experienced treatment-related adverse events
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