William Sumner
Zynerba Pharmaceuticals Inc. (ZYNE), a clinical-stage biopharmaceutical company dedicated to developing cannabinoid-based medicines, announced positive results from a meeting with the United States Food and Drug Administration (FDA). The stock initially popped on the news moving almost 5% higher to $10.50 before pulling back to $10.08 in midday trading.
The meeting was in regards to the development strategy for ZYN002 in treating Fragile X syndrome (FXS). FXS is a genetic disability that has been known to cause autism spectrum disorder and other intellectual disabilities in patients. The company currently has U.S. Orphan Drug designation for the use of cannabidiol as a treatment of FXS.
“We believe that ZYN002 may address core behavioral symptoms of FXS and improve the quality of life for patients and their families,” said Dr. Liza Squires, Zynerba Chief Medical Officer, in a statement. “We believe we have designed an efficient pivotal program that includes endpoints that measure clinically relevant and observable behaviors in patients with FXS, and if successful, positions us to bring the FXS community its first targeted treatment designed with patients’ symptoms in mind.”
Based off of its meeting with the FDA, the company expects to launch a mid-year clinical study to help assist a New Drug Application (NDA) for ZYN002 in treating FXS. The study will be a 14-week randomized, double-blind, placebo-controlled clinical trial, with approximately 200 pediatric and adolescent patients from New Zealand, the United States, and Australia.
Additionally, once the study is completed, patients will be able to enroll in a 12-month open-label extension.
If the study is successful, it could be the turning point the company has been looking for. Over the last year, Zynerba’s has taken a beating, due in no small part to a failed clinical study for ZYN002 in the treatment of patients suffering from epilepsy.
The fallout from the failed study caused the company’s stock to fall precipitously from $15 down to approximately $6, losing roughly 60% of its value. Currently trading a roughly $10 a share, the company has recovered somewhat from the dip, but positive results from its planned mid-year study could go a long way in restoring or even exceeding the value they have lost.
