Zynerba (ZYNE) has been issued a US Patent titled “Treatment of Fragile X Syndrome with Cannabidiol”, notes biotechnology specialist John McCamant, editor of The Medical Technology Stock Letter.
The patent includes claims directed to a method of treating Fragile X syndrome, comprising transdermally administering 250 mg or 500 mg of cannabidiol (CBD) daily via a gel or cream.
This new patent, which expires in 2038, is part of an expanding intellectual property portfolio covering the company’s transdermal CBD product candidate, Zygel (ZYN002 CBD gel). CBD itself is not patentable, thus adding to the significance of strong IP for topical delivery.
In our view, Zygel’s enhanced topical permeation which allows for CBD delivery into the blood stream differentiates it from most topical CBDs which just reach part of the dermis and not the blood stream.
Additionally, we believe that topical CBD delivery is superior to oral CBD delivery because it avoids two very real side effects, raised liver enzymes and somnolence.
Zygel is currently being studied in CONNECT-FX, a pivotal, multi-national, randomized, double blind, placebo-controlled study evaluating the efficacy and safety of Zygel in 204 FXS children ranging in age from 3-17 years old with full mutation of the FMR1 gene.
The primary endpoint is the change from baseline to the end of the treatment period in the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale.
Patients who have completed the double-blind phase are now enrolling into the 12-month open label phase. The company expects to report top line data in H1:20. FXS is a significant market opportunity with an estimated 71,000 patients in the U.S. and a like amount in the EU.
ZYNE’s stock has been under tax loss selling pressure and at current prices could be a big winner as soon as next year if the FXS data is positive. In our view, the FXS data will be positive and will significantly de-risk the stock. We rate the stock a buy under $18.
