Harmony Biosciences Stock Slumps After Rare Genetic Disorder Drug Fails Late-Stage Study

Harmony Biosciences Holdings (HRMY) on Wednesday said that its late-stage study of ZYN002 in Fragile X syndrome did not meet the primary endpoint of improvement in social avoidance.

Fragile X syndrome (FXS) is a rare genetic disorder that negatively affects synaptic function, plasticity, and neuronal connections. It results in a spectrum of intellectual disabilities and behavioral symptoms, such as social avoidance and irritability. According to the company, the disease affects upwards of 80,000 patients in the U.S. and 121,000 patients in the European Union and the U.K. The disease is more common in males than females, but there are no FDA-approved treatments for the disorder.

The company said on Wednesday that its drug did not meet the primary endpoint of the trial primarily due to a higher-than-expected placebo response rate.

Shares of HRMY traded 15% lower at the time of writing. On Stocktwits, retail sentiment around HRMY stock rose from ‘neutral’ to ‘bullish’ territory over the past 24 hours, while message volume rose from ‘normal’ to ‘high’ levels.

Chief Medical Officer Kumar Budur stated that, although the study's findings did not meet the primary endpoint, they provide valuable insights into Fragile X syndrome. “We will conduct a comprehensive analysis of the full dataset to better understand the results as part of our continued commitment to the Fragile X community,” Budur said.

The trial, which enrolled 215 patients with FXS aged 3 to under 30 years, aimed to assess the efficacy and safety of ZYN002, a pharmaceutically manufactured synthetic cannabidiol.

CEO Jeffrey M. Dayno stated that the company has additional candidates in its pipeline. “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,” he said.

HRMY stock is down by 21% this year and by 28% over the past 12 months. 

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