Zynerba Pharmaceuticals (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, today announced that the U.S. Patent and Trademark Office has issued Patent No. 11,458,110, titled “Treatment of Fragile X Syndrome With Cannabidiol,” which includes claims directed to methods of treating Fragile X syndrome with cannabidiol.
This new patent, which expires in 2038, is part of an expanding international intellectual property portfolio covering Zynerba’s transdermal cannabidiol product candidate, Zygel. There are currently four corresponding U.S. patents and one corresponding EU patent related to methods of treating Fragile X syndrome. Last year Green Market Report reported that Zynerba
published the results from its open-label Phase 2 BELIEVE (Open-Label Study to Assess the Safety and Efficacy of Zygel (ZYN002) Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy) study in the Journal of the American Medical Association (JAMA) Network Open.
The study demonstrated a 58% median monthly reduction in seizures at month 5, and a 43.5% reduction over the 6.5 month study period (the primary efficacy endpoint). In addition, the parents/caregivers noted improvements in social or interpersonal engagement and irritability (77%); alertness, energy, and sleep (53%); and cognition or concentration (47%).
48 patients with a mean age of 10.5 years were enrolled in BELIEVE and included in the safety analysis. 60% had at least one treatment-related adverse event (AE) over the 6.5 month trial period and 96% of these AE’s were mild or moderate. During the treatment period, 10 patients (21%) reported serious adverse events (SAEs). Two SAEs were considered to be possible treatment-related: nonconvulsive status epilepticus and lower respiratory tract infection, in separate patients. All SAEs were resolved, and none resulted in an alteration of the study medication. The study warned that there was no control group and so it is impossible to know how much was due to the placebo effect and the desire for cannabis products to work.