PLYMOUTH MEETING, PA — Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) unveiled promising results from its Open-Label Extension (OLE) study evaluating ZYN002, a potential treatment for Fragile X syndrome (FXS). The data, presented at the American Academy of Neurology (AAN) 2025 Annual Meeting, demonstrated meaningful reductions in irritability-related symptoms in FXS patients, a neurological condition with no existing FDA-approved therapies.
“The acceptance of our Fragile X syndrome OLE data as a podium presentation at AAN underlines the significant unmet need in FXS, and the potential for ZYN002 to become the first-and-only approved treatment for this condition,” said Dr. Kumar Budur, Chief Medical and Scientific Officer at Harmony Biosciences.
Participants in the OLE trial, drawn from earlier Phase 2/3 and Phase 1/2 studies, showed measurable behavioral improvements. Over 60% achieved at least a 9-point reduction in irritability scores, and 73.3% of long-term ZYN002 users maintained clinically significant progress. Caregivers also reported improved behaviors in 40.6% of patients receiving active treatment.
The study assessed 240 patients over several years, with ZYN002 administered at varying doses based on weight. Adverse events were minimal, with serious cases occurring in just 4.6% of patients.
ZYN002 is now under investigation in a pivotal Phase 3 trial, RECONNECT, with topline results expected in the third quarter of 2025. With an estimated 80,000 individuals affected by FXS in the U.S., Harmony remains focused on advancing this innovative treatment to address a critical therapeutic gap.
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