Tourette's Syndrome

April 16, 2018

TEL AVIV announced topline results from its investigator-initiated Phase IIa study, 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 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 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 percent across the entire sample. 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.

December 4, 2017

Abide Therapeutics reported results of a completed, exploratory phase Ib study in Tourette Syndrome (TS) with ABX-1431. This study in adults was a randomized, blinded, placebo-controlled crossover, single dose design. Change from baseline was measured in the Yale Global Tic Severity Scale (YGTSS), Adult Tic Questionnaire (ATQ), Premonitory Urge for Tics Scale (PUTS) and Modified Rush Video Scale (MRVS). A statistically significant reduction of 10% (placebo-adjusted, p=0.0384) in the Total Tic Score of the YGTSS was observed at eight hours. Furthermore, statistically significant effects were seen in the PUTS, a scale measuring the intensity of the premonitory urge to tic, which is an uncomfortable sensation experienced immediately prior to tics, and in the ATQ, a self-assessment of tics, where tic intensity was reduced an average of 30%. Standardized video assessment (the MRVS) also showed improvement, but did not reach statistical significance. ABX-1431 was generally safe and well-tolerated. There were no serious adverse events, and all adverse events (AE) resolved. Headache, somnolence and fatigue were the most common AE.

January 30, 2017

Psyadon Pharmaceuticals released results of a phase IIb study of ecopipam for treatment of total tic severity scores in children (7-17 years) with Tourette’s syndrome. The study was a double-blind, randomized, placebo-controlled crossover study. Patients were instructed to take the study medication (either ecopipam or placebo) each evening before bedtime over a four-week treatment period. Patients were evaluated in the clinic every other week with telephone contacts on the alternate weeks. The primary efficacy endpoint was the change in the Yale Global Tic Severity Score–Total Tic Score (YGTSS-TTS), a well-validated rating scale typically used in Tourette’s syndrome trials. The study was fully enrolled at 40 subjects. The study’s primary endpoint was a statistically significant reduction in the mean Yale Global Tic Severity Score Total Tic Score (YGTSS-TTS). This is a well-validated and standard measure of the clinical efficacy of drugs treating Tourette’s syndrome. Ecopipam caused significant reductions in the YGTSS-TTS at both two- and four weeks after initiating treatment. Furthermore, the side effects seen in the subjects taking ecopipam were comparable to those seen in the placebo-treated subjects, including no clinically significant changes in standard clinical lab tests or ECGs, and no increases in body weights.

July 30, 2012

Psyadon Pharmaceuticals issued results from a phase II trial of ecopipam for the treatment of Tourette’s syndrome. This eight-week, multi-center, open-label, non-randomized study enrolled 15 patients with moderate to severe Tourette’s syndrome. Subjects received ecopipam 50mg/day for the first two weeks and then 100mg/day for the remaining six weeks. The results showed a statistically significant (p<0.001) reduction in the total tic severity scores of the Yale Global Tic Severity Score, a well-validated rating scale typically used in Tourette’s trials. This effect was seen in the analysis of both patients who completed all visits (completer analysis; n=12) and all enrolled patients (intent-to-treat analysis; n=15). The study was stopped early when a planned interim analysis revealed a statistically significant reduction in the severity of the patients’ tic symptoms. Ecopipam was well tolerated. The most frequent adverse events were fatigue, nausea, sedation, headache, restlessness and sleeplessness. Psyadon is eager to move ecopipam into phase III trials.