After getting crushed by bad data, Zynerba says its cannabis-based drug worked on Fragile X symptoms

Less than two months since a pair of mid-stage setbacks crushed its stock price, researchers at Zynerba $ZYNE say they had better results from a small Phase II study that tested their cannabis-based therapy on symptoms of Fragile X syndrome. And the news quickly sparked a big rally for its battered stock.

Armando Anido

Often compared to GW Pharmaceuticals, which has had some striking successes in the field, Zynerba set out to see what impact their cannabidiol therapy would have tamping down on a range of common symptoms for Fragile X, including anxiety and depression. And with 20 patients in the single-arm study, the investigators say they hit their primary with a 46% drop in their ADAMS score, tracking Anxiety, Depression, and Mood Scale.

Fragile X is an autism spectrum disorder, one tough disease to treat at the source. It’s significant that Zynerba didn’t go after the disease itself, but rather symptoms that can make life miserable for the patients and their families. That’s an easier target, especially when you have a small, uncontrolled study to use — which is far from ideal.

Now, the biotech plans to steer this drug into a pivotal Phase II/III, news that revived their share price — which immediately soared 90% this morning in pre-market trading.

Zynerba was forced to concede last month that its drug failed two back-to-back mid-stage studies for epilepsy as well as osteoarthritis, with their cannabinoid unable to significantly reduce knee pain.

“The clinically meaningful improvements in Fragile X symptoms and the excellent tolerability seen in the FAB-C trial are compelling,” exclaimed Zynerba CEO Armando Anido in a statement. “These data will allow us to discuss the pathway to approval in a meeting with the FDA, which we expect to take place during the first half of 2018.”

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