TEL AVIV, Israel, April 9, 2018 Therapix Ltd. (NASDAQ: TRPX) ("Therapix" or "the Company"), a biopharmaceutical company focused on discovering, developing, and commercializing novel therapeutics from its proprietary cannabinoid product platform THX-110, announced today topline results from its investigator-initiated Phase IIa study at Yale University, suggesting that THX-110 [which is a combination of dronabinol (Delta-9-tetrahydracannabinol) and palmitoylethanolamide (PEA)] significantly improved symptoms over time in adult Subjects with Tourette syndrome.
"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. This study was designed primarily to confirm safety, tolerability and feasibility in this challenging patient population and is encouraging that we obtained positive data that suggests that the combination of dronabinol (Delta-9-tetrahydracannabinol) and palmitoylethanolamide (PEA) (THX-110) should be pursued as a treatment for TS patients," continued Dr. Zuloff-Shani. "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 Tourette syndrome."
The study was a single-arm, open-label trial, in which each subject both received one daily treatment of the drug via oral administration and was followed-up for a period of 12 weeks. Sixteen subjects participated in the study and received THX-110 at the Yale University Child Study Center at Yale University, USA. The primary endpoint of the study was to assess the performance of THX-110 in the treatment of adult patients suffering from symptoms of Tourette syndrome, as measured by the Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS), the gold-standard and customary index for assessing symptom severity. Treatment was given in a dose titration regimen with a maximum dose of THX-110 consisting of 10mg Dronabinol and 800mg PEA.
The study was investigator-initiated and led by Michael H. Bloch, M.D., Associate Professor at the Yale University Child Study Center, and James F. Leckman, M.D., Ph.D., of the Neison Harris Professor in the Child Study Center and Professor of Pediatrics Child Psychiatry at Yale University.
The study showed that these 16 subjects with medication-refractory TS had a reduction of tic symptoms (paired t-test: YGTSS-TTS mean difference (mean +/- SD) =7.9+/-8.4, t= 3.7, df=15, p=0.002) from baseline (YGTSS-TTS: 38.4 +/- 8.3) to endpoint (YGTSS-TTS: 30.5 +/- 10.9). This resulted in an average tic reduction of 21% across the entire sample.
Six of the 16 medication-refractory TS subjects experienced a response to treatment as defined by a reduction in YGTSS-TTS of greater than 25%. Improvement over time with treatment was also observed when generalized linear models were used to analyze repeated measures data on the YGTSS-TTS. THX-110 demonstrated no significant effects on comorbidity. The medication was generally well-tolerated by subjects with only two subjects stopping treatment early (one due to sedation and another due to lack of improvement in tic symptoms). Twelve of the 16 subjects elected to continue into a 24-week extension phase of the trial, which is nearing completion.
"In general, this study enrolled 16 adults, most of which had severe, refractory Tourette syndrome that had not responded to previous evidence-based treatment (e.g. antipsychotic medications, alpha-2 agonists, behavioral therapy) and multiple investigational compounds (e.g. 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 ef...
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