There’s been a lot of hype in recent years – and often times rightfully so – regarding the prowess of the cannabis plant to provide a meaningful benefit to help epilepsy patients with respect to fewer seizures. Zynerba Pharmaceuticals Inc. (NASDAQ: ZYNE) was surely hoping to be the next in line to release positive news on an experimental new cannabis-constituent drug, but such was not the case, sending shares hurtling lower at Monday’s opening bell.
The Devon, Pennsylvania-based company said that its phase 2 study of ZYN002, dubbed STAR 1 (Synthetic Transdermal Cannabidiol for the Treatment of Epilepsy), failed in meeting its primary endpoint in providing a reduction in focal, or partial, seizures in adult epilepsy patients compared to placebo.
ZYN002 is a synthetic cannabidiol (CBD), one of the active cannabinoids in cannabis, formulated as a clear gel for absorption through the skin (transdermal administration). Zynerba is in the clinical stage of developing the gel for multiple indications, including refractory epilepsy, osteoarthritis (phase 2) and Fragile X syndrome (phase 2). One of the benefits of transdermal delivery, according to the company, is avoiding degradation of the active ingredient in stomach acid.
In the STAR 1 study, 188 patients across clinical sites in Australia and New Zealand were randomized to receive the high dose of ZYN002 (195 mg) or low dose of ZYN002 (97.5 mg) or placebo every 12 hours for 12 weeks. Enrolled patients had a median monthly seizure frequency of 10.6 and were on an average of 2-1/2 anti-epileptic drugs.
Patients on the low dose of ZYN002 showed a 18.4% median reduction in focal seizures, while those on the high dose saw less benefit at only a 14.0% reduction during the treatment period versus baseline. Patients in the placebo group demonstrated an 8.7% reduction.
Further, no secondary endpoints, which looked at patients who saw their seizure frequency at least halved, showed any statistical significance between the treatment and placebo groups.
On the bright side, the gel was found to be safe and very well tolerated.
Zynerba still has an open label STAR 2 trial ongoing in adult epilepsy patients with focal seizures and will continue to analyze all the data in this patient population to determine the best clinical pathway going forward.
Shares of ZYNE are taking a lumping on the trial failure, plunging 58% to $6.35 from Friday’s close at $15.06 at the noon hour Eastern Time.
