- Shares of Zynerba Pharmaceuticals Inc. tanked on Monday after the Pennsylvania biotech announced its lead candidate failed in a mid-stage study for patients with adult epilepsy and focal seizures.
- Zynerba’s stock dropped 59% in early morning trading to linger just above $6 per share after the company revealed the disappointing topline results from its Phase 2 STAR1 trial.
- ZYN002, a synthetic transdermal cannabidiol gel, failed to demonstrate a statistically significant reduction of focal seizures with either the high or low dose compared to placebo.
Zynerba CEO Armando Anido expressed his disappointment in the Phase 2 results in an early morning statement to investors.
"We are continuing to evaluate this study and the ongoing STAR 2 open label study to determine next steps with ZYN002 in adult epilepsy patients with focal seizures," Anido said further.
The 188-patient study randomized patients to receive either the 195 mg dose of ZYN002 4.2% CBD gel every 12 hours, the 95 mg dose of the drug or placebo. Patients included in the study ranged from ages 18 to 71 and had an average of 10.6 seizures a month. Patients were on an average of 2.5 other anti-eptileptic medications. The primary endpoint of the study was reduction in seizures after 12 weeks compared to an 8-week baseline period.
Patients taking the low dose showed an 18.4% reduction in seizures, while those on the high dose showed a 14.0% reduction, compared with an 8.7% reduction in the placebo group.
ZYN002 is the company's lead product candidate and is also being studied in Fragile X syndrome and osteoarthritis. Results from mid-stage studies of both drugs are expected in coming months.
"We are excited that we will present top-line data from our ZYN002 STOP trial in osteoarthritis soon, followed by top-line data from our FAB-C study in Fragile X syndrome by the end of September," added Anido.
ZYN002 is a synthetic derivative of the active chemicals found in marijuana that is non-psychoactive. There is a growing portion of the pharmaceutical industry focused on using the plant to create treatments for pain. Even though this area of science is getting more attention, clinical success has been spotty and only a handful of competitors are moving forward in the space.
Competitor GW Pharmaceuticals is expected to file a marketing application in the U.S for its own cannabis-based drug. The failure of ZYN002 improves the competitive landscape for GW and shares in the company were up slightly in morning trading, increasing to $119.10 apiece.