PLYMOUTH MEETING, PA — Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) reported topline results from its Phase 3 RECONNECT study of ZYN002 in Fragile X syndrome (FXS), announcing that the trial did not meet its primary endpoint due to an unexpectedly high placebo response rate.
The 18-week randomized, double-blind, placebo-controlled study evaluated ZYN002, a pharmaceutically manufactured cannabidiol transdermal gel, in 215 patients ages 3 to under 30. The primary measure was improvement in social avoidance among patients with complete methylation of the FMR1 gene, with secondary measures assessing irritability.
“Although the study did not achieve its primary endpoint, the findings provide valuable insights into Fragile X syndrome, a rare condition with significant unmet medical need and no FDA-approved therapies,” said Kumar Budur, M.D., M.S., Chief Medical and Scientific Officer at Harmony. “We will conduct a comprehensive analysis of the dataset as part of our continued commitment to the Fragile X community.”
Fragile X syndrome is the leading inherited cause of intellectual disability and autism spectrum disorder, affecting an estimated 80,000 people in the U.S. Current treatment options remain limited to symptom management.
ZYN002, which has received orphan drug designation from both the FDA and EMA, as well as FDA Fast Track designation, is a synthetic cannabidiol formulated for transdermal delivery and does not contain THC.
Harmony’s President and CEO Jeffrey M. Dayno, M.D., said the company remains focused on advancing its late-stage pipeline, with Phase 3 trials of pitolisant HD for narcolepsy and idiopathic hypersomnia expected to begin in the fourth quarter. “We remain confident in our ability to bring innovative therapies to patients while creating long-term value for shareholders,” Dayno said.
Despite the setback, Harmony underscored its commitment to further analysis of the trial data, which it believes will inform the next steps in addressing the considerable treatment gap for Fragile X patients.
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