Harmony Biosciences Holdings, Inc. has released topline results from its Phase 3 RECONNECT Study evaluating ZYN002, a transdermal cannabidiol gel, in patients with Fragile X syndrome (FXS). The study did not meet its primary endpoint of improving social avoidance symptoms, with the company citing an unexpectedly high placebo response rate as a key factor.
Despite the outcome, Harmony emphasized the importance of the trial in contributing to the understanding of Fragile X, a rare neurobehavioral disorder that currently has no FDA-approved treatments. Dr. Kumar Budur, Chief Medical and Scientific Officer at Harmony Biosciences, stated that while the trial did not achieve its main goal, the data provides valuable insights. He reaffirmed the company’s commitment to the Fragile X community and noted that a full analysis of the data will be conducted to better understand the results. Dr. Budur also expressed appreciation for the patients, families, and clinicians who participated in the study.
The RECONNECT Study was a randomized, double-blind, placebo-controlled, multi-center trial designed to assess the safety and efficacy of ZYN002 in individuals aged 3 to under 30 with Fragile X syndrome. ZYN002 is a pharmaceutically manufactured cannabidiol gel administered through the skin, offering a non-oral route of delivery.
Commenting on the results, Harmony’s President and CEO Dr. Jeffrey M. Dayno acknowledged the disappointment but maintained confidence in the company’s overall development pipeline. He noted that Harmony has several Phase 3 programs in progress and is preparing to launch trials for pitolisant HD in both narcolepsy and idiopathic hypersomnia in the fourth quarter of this year. Dr. Dayno highlighted the company’s strong financial position and self-funding business model as key advantages that will support continued growth and innovation.
While the outcome of the RECONNECT Study represents a setback in the development of ZYN002, Harmony Biosciences remains committed to advancing treatments for patients with rare and underserved neurological disorders.