Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) released topline results from its Phase 3 registrational clinical trial (the RECONNECT Study) of ZYN002 in Fragile X syndrome (FXS).

Fragile X syndrome is a rare genetic disorder that is the leading known cause of both inherited intellectual disability and autism spectrum disorder.

The disorder negatively affects synaptic function, plasticity and neuronal connections, and results in a spectrum of intellectual disabilities and behavioral symptoms, such as social avoidance and irritability. 

Also Read: Harmony Biosciences Stock Falls On FDA Setback For Pitolisant In Sleeping Disorder

There is a significant unmet medical need in patients living with FXS as there are currently no FDA-approved treatments for this disorder.

The RECONNECT Study did not meet the primary endpoint of improvement in social avoidance primarily due to a higher-than-expected placebo response rate.

Kumar Budur, Chief Medical and Scientific Officer at Harmony Biosciences, said, “We will conduct a comprehensive analysis of the full dataset to better understand the results…”

The RECONNECT Study was a Phase 3 randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of ZYN002, a pharmaceutically manufactured cannabidiol administered as a transdermal gel to patients with FXS ages 3 to under 30 years old.

“We have a late-stage, catalyst-rich pipeline with multiple Phase 3 programs in the clinic and continue to be on track to initiate our Phase 3 trials for pitolisant HD in narcolepsy and idiopathic hypersomnia in the fourth quarter of this year. Harmony’s unique profile as a profitable self-funding biotech company with strong cash-generation positions us well to drive future growth,” said Jeffrey Dayno, President and CEO of Harmony Biosciences.

Price Action: HRMY stock was trading lower by 14.55% to $27.41 at last check Wednesday.

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