An Israeli biotech developing cannabinoid-based drugs has found preliminary evidence that its lead product can reduce symptoms in adults suffering from Tourette syndrome.
In a single-arm, open-label Phase IIa trial designed primarily to confirm safety, tolerability and feasibility, six of 16 TS patients experienced a response to Therapix’s treatment, as defined by a reduction in the Yale Global Tic Severity Scale Total Tic Score of greater than 25%, after 12 weeks. The investigators from Yale also reported a mean difference of 7.9 in score (from 38.4 to 30.5). That translates to an average tic reduction of 21% — referring to the repeated involuntary movements and sounds that characterizes the disease — though the lack of a control arm makes it difficult to access just how significant it is.
According to Michael Bloch, the principal investigator, the results are encouraging given that these are patients who have not responded to previous treatments (like antipsychotic medications and behavioral therapy) or experimental therapies (like topiramate or deep brain stimulation).
Therapix shares $TRPX shot up 32% in premarket trading.
The drug, named THX-110, is a combination of dronabinol (a synthetic cannabinoid) and palmitoylethanolamide. The company now plans to take it into a randomized, double-blind, placebo controlled study to evaluate its efficacy, in addition to a 24-week extension phase of the trial read out today. Twelve of the study subjects have agreed to take part in the extension.
“We believe that the efficacy demonstrated in this study further reinforce the potential role of cannabinoids in the field of CNS and movement disorders and suggests that Therapix Biosciences may have a proprietary cannabinoid product candidate in the treatment of Tourette syndrome,” said Ascher Shmulewitz, Therapix’s chairman and interim CEO, in a statement.
THX-110 is also being studied for obstructive sleep apnea and for the treatment of pain.
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