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Harmony Biosciences reveals disappointing results from the Phase III Reconnect study of ZYN002 for Fragile X Syndrome, highlighting ongoing research challenges.
The Phase III Reconnect trial studied ZYN002 in Fragile X Syndrome.
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Harmony Biosciences announced results from its Phase III registrational clinical study (the Reconnect study) of ZYN002 in Fragile X Syndrome (FXS). Results from the Reconnect study showed ZYN002 failed to reach its primary endpoint of improved social avoidance due an unexpected high placebo response rate in participants.
Kumar Budur, M.D., M.S., chief medical and scientific officer at Harmony Biosciences touched on the clinical trial results, saying, “Although the study did not achieve its primary endpoint, the findings from this study provide valuable insights into Fragile X syndrome, a rare neurobehavioral condition with significant unmet medical need and no FDA-approved therapies. 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.” Dr. Budur added, “We are grateful to the patients, families, caregivers, clinicians, and researchers who made this trial possible.”
The Reconnect Study was a Phase III randomized, double-blind, placebo-controlled, multiple-center study assessing the efficacy and safety of ZYN002. The pharmaceutically manufactured cannabidiol was administered as a transdermal gel to patients diagnosed with FXS aged 3–29 years old.1 Study participants included 215 male and female patients who were randomized 1:1 to receive ZYN002 or the placebo, spanning a 18-week treatment period, with treatment dosage being decided through weight-based metrics.1 The study’s primary endpoint aimed at improving from its baseline to week 18 in the ABC-CFXS Social Avoidance subscale score in patients with complete methylation (≥90%) in the FMR1 gene.1 Key secondary endpoints of the study included changes from baseline to week 18 in the ABC-CFXS Irritability subscale score in patients with complete methylation (≥90%) in the FMR1 gene.1
Harmony offered an at-home option for the study in an attempt to accommodate participants and their families for the struggles that FXs entails.
“This is a trial that’s taking place at multiple centers across the United States, but there’s also a unique option for families that find traveling difficult or find it easier to stay home. There is a completely at-home option that allows your family to enroll and participate in the trial entirely from the comfort of your own home.” Said Dr. Carrie Buchanan, Fragile X Program director, Greenwood Genetic Center.
ZYN002 is the first-and-only pharmaceutically manufactured synthetic cannabidiol devoid of THC and formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system.1 Production forZYN002 begins through a synthetic process in a cGMP facility without being extracted from cannabis plants, and does not contain THC, the compound causing the euphoric effect of cannabis and holds potential to be a nonscheduled product if approved.1 ZYN002’s active ingredient, Cannabidiol was granted orphan drug designation by FDA and the European Medicines Agency (EMA), as a treatment for FXS and for the treatment of 22q. Additionally, ZYN002 was granted Fast Track designation for the treatment of behavioral symptoms in patients with FXS from FDA.1
“While these results are not what we anticipated, we remain confident in our ability to bring innovative therapies to patients while creating long-term value for shareholders,” said Jeffrey M. Dayno, M.D., President and CEO of Harmony Biosciences. “We have a late-stage, catalyst-rich pipeline with multiple Phase III programs in the clinic and continue to be on track to initiate our Phase III 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.”
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