Zynerba goes into meltdown mode as cannabis-derived drug flops in PhII

Two years after its IPO, Devon, PA-based Zynerba Pharmaceuticals $ZYNE is grappling with the fallout from a failed Phase II study of its lead drug — a cannabis-derived therapy delivered via a skin patch.

Researchers failed to find a real benefit for epilepsy patients in the trial, missing on two doses in an attempt to reduce focal seizures over a placebo.

The biotech’s shares were blitzed by the immediate response from investors, who drove the company’s shares down 63%.

Armando Anido

CEO Armando Anido zeroed in on what positives he could find, which in this case boiled down to safety and tolerability results. As for efficacy, the high-dose treatment reduced seizures by 18.4%, the low dose by 14% and placebo by 8.7%, falling well below the range needed for statistical importance.

The focus at the wounded biotech will now shift to upcoming osteoarthritis and Fragile X data.

Zynerba’s setback occurs as GW Pharma $GWPH is pursuing an approval of Epidiolex — a liquid therapy using purified cannabadiol — which triggered a mean reduction in convulsive seizures of 39% among treatment-resistant patients with Dravet syndrome.

“We are very disappointed that the STAR 1 trial did not meet its primary endpoint in this patient population,” said Anido in a statement. “We are continuing to evaluate this study and the ongoing STAR 2 open label study to determine next steps with ZYN002 in adult epilepsy patients with focal seizures.”

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
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Atlas Venture Cambridge, MA

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