Zynerba's Zygel Fails in Phase III Fragile X Syndrome Study
Zynerba Pharmaceuticals, Inc. ZYNE announced that the phase III study — CONNECT-FX — evaluating its pipeline candidate, Zygel, failed to achieve statistical significance in primary as well as secondary endpoints. The study evaluated Zygel cannabidiol gel as a treatment for behavioral symptoms of Fragile X syndrome (“FXS”), a genetic condition causing intellectual disability.
Shares of Zynerba plunged 48.5% on Jun 30 following the study data readout. In fact, the company’s shares have declined 44.2% so far this year compared with the industry’s decrease of 5.4%.
Data from the 14-week CONNECT-FX study showed that treatment with Zygel did not achieve statistical significance improvement in the Social Avoidance subscale of the Aberrant Behavior Checklist (“ABC”) – Community FXS (“CFXS”), the study’s primary endpoint, compared to placebo. The study also failed to meet statistical significance in secondary endpoints of Irritability subscale score of the ABC-CFXS, the Socially Unresponsive/Lethargic subscale score of the ABC-CFXS and Improvement in Clinical Global Impression, as measured by change from baseline to the end of the treatment period.
However, the company stated that a pre-planned ad hoc analysis showed that patients treated with Zygel achieved statistical significance in the primary endpoint of improvement at 12 weeks of treatment. The analysis included most severely impacted patients in the study, as defined by patients having at least 90% methylation (full methylation) of the impacted FMR1 gene, representing 80% of the total study population. The company believes that full methylation occurs in approximately 60% of total FXS patients in the market.
The company plans to meet with the FDA, as soon as possible, to discuss a regulatory path forward for Zygel as a treatment for FXS patients based on the results from the ad hoc analysis.
Apart from FXS, Zynerba is also evaluating Zygel in other neurological indications namely — developmental and epileptic encephalopathies ("DEE"), autism spectrum disorder ("ASD") and 2 deletion syndrome (22q).
The company had announced positive top-line data from the phase II study evaluating Zygel in DEE patients in September 2019. It anticipates to provide a clinical path forward in this indication in the third quarter. The company announced positive top-line data from the phase II study evaluating Zygel in ASD patients in May. It intends to meet with the FDA to discuss the results and clinical path forward in ASD indication in the second half of 2020. Top-line data from the phase II study evaluating the candidate in 22q patients is expected in the fourth quarter of 2020.
Please note that cannabidiol is a key compound of Zygel. The legalization of medical marijuana in multiple states in the United States suggests that the availability of cannabis-based products like cannabidiol is likely to increase.
We note that a few companies have cannabidiol drugs in their portfolio. The market is expected to reach $20 billion by 2024 per a Forbes report. GW Pharmaceuticals’ GWPH cannabidiol drug, Epidiolex was the first medicine made from Cannabis sativa plant species to win approval from the FDA. The drug is primarily used to treat rare forms of epilepsy.
Zynerba Pharmaceuticals, Inc. Price
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Zacks Rank & Stocks to Consider
Zynerba currently carries a Zacks Rank #3 (Hold). A couple of stocks to consider from the pharma/biotech sector include Avenue Therapeutics, Inc. ATXI and Aldeyra Therapeutics, Inc. ALDX, both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Avenue Therapeutics’ loss per share estimates have narrowed 21.3% for 2020 and 5.5% for 2021 over the past 60 days. The stock has rallied 12.2% year to date.
Aldeyra Therapeutics’ loss per share estimates have narrowed 24% for 2020 and 24.3% for 2021 over the past 60 days.
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