Zynerba shares up on rare disease trial success
- Zynerba Pharmaceuticals' shares shot up by 52.5% after news on Thursday that its lead drug, ZYN002 cannabidiol gel met its primary endpoint in its FAB-C Phase 2 trial in children and adolescents with Fragile X syndrome.
- In the open label study of the orphan drug, patients had a 46% improvement from baseline on the Anxiety, Depression and Mood Scale (p<0.0001), and clinically meaningful improvements in all measures of the Aberrant Behavior Checklist.
- The next step will be a meeting with the Food and Drug Administration in the first half of 2018, and a Phase 2/3 trial in pediatric and adolescent patients by the end of next year.
"These data are extremely promising, particularly the improvements in anxiety, social avoidance and irritability as measured by scales including ADAMS, ABC-FXS, and PARS-R. Tolerability is essential in these patients, so I'm very pleased to see that ZYN002 was well tolerated in Fragile X patients," said Honey Heussler, associate professor at Children's Health Queensland, medical director Child Development and lead investigator in the FAB-C study.
As well as meeting a statistically significant primary endpoint, the drug was able to improve a number of quality of life issues for patients that were measured as secondary endpoints.
During the 12-week study, two patients discontinued due to worsening eczema and four patients experienced adverse events, although none were considered severe. Thirteen of the 18 patients who completed the study moved on to the open-label extension study.
Cannabidiol is a non-psychoactive cannabinoid and ZYN002 is in development for a diverse set of conditions, including refractory epilepsy, osteoarthritis of the knee and Fragile X syndrome. These results in Fragile X syndrome will have come as very good news for Zynerba, after its shares tanked last month on news of the drug's failure in the Phase 2 STAR1 trial in adult epilepsy and focal seizures, failing almost 60% from the beginning of the year.
Results are still awaited from the mid-stage STOP trial in osteoarthritis. Zynerba is also developing ZYN001, a prodrug of the psychoactive cannabinoid tetrahydrocannabinol (THC), in Phase 1 for the treatment for fibromyalgia and peripheral neuropathic pain. Shares in Zynerba's key competitor in the cannabis field, GW Pharmaceuticals, also rose slightly this week.
Fragile X syndrome is a rare autism spectrum disorder causing intellectual disability, and affecting around 71,000 people in the U.S. There are no approved therapeutics, so ZYN002, described by Zynerba Pharmaceuticals as the first and only synthetic cannabidiol formulated as a permeation-enhanced gel for transdermal delivery, could meet a currently unmet need.
Other products in development for Fragile X syndrome include Ovid Therapeutics' OV101 (gaboxadol), Bellus Health's AMO-01, and Neurotrope BioScience's bryostatin-1, all in Phase 1, and Neuren Pharmaceuticals' NNZ-2556, in Phase 2 development.
- Zynerba Pharmaceuticals Statement
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