Therapix Biosciences reported topline data from a phase 2a study showing that its cannabinoid-based treatment for Tourette syndrome met its primary endpoint: improving symptoms in adults with the disorder.
The Tel Aviv-based company is working on repurposing Dronanbinol, an FDA-approved synthetic cannabinoid, for a number of indications, including pain management, traumatic brain injury and obstructive sleep apnea, as well as Tourette.
In the single-arm, open-label trial, 16 patients received a daily oral dose of THX-110, a combination of dronabinol and palmitoylethanolamide (PEA), and were followed up for 12 weeks. Most of the patients had severe, refractory Tourette syndrome that had not responded to other treatments, including antipsychotic drugs and behavioral therapy as well as investigational treatments, such as topiramate, VMAT-inhibitors and deep-brain stimulation.
The primary endpoint was measured by the Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS). The data showed an average tic reduction of 21% across all patients, while six of the 16 (about one-third) of the patients had a decrease in YGTSS-TSS that was greater than 25%. Twelve of the 16 patients continued into a 24-week extension of the trial, which is coming to a close. The company will present full data at the annual meeting of the European Society for the Study of Tourette syndrome in June.
"The successful completion of this study is a key milestone in our clinical development plan of our proprietary drug candidate THX-110 for the treatment of Tourette syndrome for which current available treatments are frequently inadequate or unsafe," said Adi Zuloff-Shani, Ph.D., chief technology officer at Therapix. "Moreover, these results are of particular interest as the pharmacology of THX-110 appears to be distinct from existing medications for TS and may offer a unique option for treating these patients."
Next up, the company will launch a randomized, double-blind, placebo-controlled trial of THX-110 in adults with Tourette.
Therapix filed for a $12 million Nasdaq IPO in November 2016, joining a number of other companies developing cannabinoid-based treatments. These include Arena Pharmaceuticals, Zynerba and the U.K.'s GW Pharmaceuticals. Arena reported some positive phase 1 data for its pain drug in April 2016, while GW, one of the segment's biggest players, posted a phase 2 failure earlier this year in a study of patients with focal seizures.