Therapix Biosciences (Tel Aviv, Israel) has announced results from its investigator-initiated phase 2a study at Yale University led by Michael Bloch and James Leckman to test Therapix’s compound THX-110, which is a combination of dronabinol (delta-9-tetrahydracannabinol) and palmitoylethanolamide (PEA).
The study was a single-arm, open-label trial, in which 16 patients with refractory Tourettes’s syndrome (TS) received an oral administration of THX-110 once per day over a 12-week period. Dose of THX-110 given was titrated up to a maximum consisting of 10 mg dronabinol and 800 mg PEA. The primary endpoint was reduction in tics as measured by the Yale Global Tic Severity Scale Total Tic Score.
Of the 16 treated patients, there was an average tic reduction of 21% from baseline (P = .002), and in 6 subjects tics were reduced by more than 25%. Improvement over time was also observed using generalized linear models to analyze repeated measures data. There were no significant effects of treatment with THX-110 on comorbidities. Only 2 subjects stopped treatment early (1 due to sedation, 1 due to lack of improvement in tic symptoms) and THX-110 was generally well-tolerated with 12 of 16 patients electing to continue into a 24-week extension phase of the study.
"In general, this study enrolled 16 adults, most of (whom) had severe, refractory TS that had not responded to previous evidence-based treatment (eg, antipsychotic medications, alpha-2 agonists, behavioral therapy) and multiple investigational compounds (eg, topiramate, VMAT-inhibitors, repetitive transcranial magnetic stimulation, deep brain stimulation). A subset of these subjects experienced improvement in tic symptoms during the course of the study. The overall improvement of tics in this sample of severe, adult TS is quite encouraging and further placebo-controlled trials are needed in an attempt to demonstrate efficacy," said Dr. Michael Bloch.
Adi Zuloff-Shani, PhD, Chief Technology Officer of Therapix noted. ". . .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. Based on these study results, we intend to initiate a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability and efficacy of daily oral THX-110 in treating adults with TS."Next Story