Zynerba (NASDAQ: ZYNE) a clinical stage specialty pharma company focused on transdermal cannabinoid treatments, today announced positive results for an open label exploratory Phase 2 trial evaluating ZYN002 cannabinidiol gel in children with Fragile X Syndrome.

The test met its primary endpoint, achieving 46% improvement in the total score of Anxiety, Depression and Mood Scale (ADAMS) at week twelve compared to baseline.

ZYN002 also achieved clinically meaningtful improvements in all measures of the Aberrant Behavior Checklist for Fragile X, which addresses key symptoms including social avoidance, temper tantrums, repetitive movements, and hyperactivity.

The data is “very exciting and demonstrate that ZYN002 may have a profound effect on improving many of the disabling symptoms of Fragile X, such as anxiety and difficult behaviors,” said Steven Siegal, MD, PhD and Chair, at Keck School of Medicine at USC. “ Many families struggle with a lack of approved drugs…” for this disease.

Zynerba expects to move on to Phase 2/3 with adolescent patients in 2018. The FDA has granted Zynerba Orphan Drug designation, which gives it exclusive marketing rights for drugs with less than 200,000 patients for 7 years.

Zynerba stock shot up 52% on the news, on volume of 13 million shares, to close at $9.44. The company’s market cap is $125.15 million.

Source: Zynerba press release

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