Catalyst Pharmaceuticals Inc (NASDAQ:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced top-line results in an open-label, proof-of-concept trial of CPP-109 (vigabatrin) used to treat patients suffering from Tourette’s Disorder (TD) that were refractory to all other previous treatments. One of four patients demonstrated a very clear, clinically significant reduction in tics, and two others showed about a 25% reduction in tics, but without subjective clinical improvement.

Patrick J. McEnany, Catalyst’s Chief Executive Officer, stated, “We see these data as promising despite the small number of subjects, single center, open-label design trial.” Mr. McEnany went on to say, “These top-line results demonstrate an encouraging signal of activity in adult treatment-refractory patients with Tourette’s Disorder. We believe that CPP-109’s mechanism of action validates the potential for CPP-115 to be a candidate for the treatment of Tourette’s Disorder. We are evaluating our options and will be meeting with the Co-Principal Investigators of the trial, as well as other key opinion leaders, to determine the next steps in development.”

This proof-of-concept trial of CPP-109 (Vigabatrin) for Treatment-Refractory Tourette’s Disorder was conducted by Barbara Coffey M.D., M.S., Co-Principal Investigator and Chief of the Tics and Tourette’s Clinical and Research Program at the Icahn School of Medicine at Mount Sinai in New York, in collaboration with Dr. Jonathan D. Brodie at the NYU Langone Medical Center, the Co-Principal Investigator and lead inventor of this use of GABA-AT inhibitors. The trial was designed to obtain preliminary data regarding clinical effect, dosage, and tolerability of vigabatrin in subjects at least 18 years of age with a DSM-IV-TR diagnosis of Tourette’s Disorder, who exhibited unsatisfactory response to all prior treatments.

Dr. Coffey said, “The clinical signal from this small open label trial, despite its limitations, is encouraging and warrants further investigation. The only formally approved medications to treat Tourette’s Disorder are first generation antipsychotic drugs which block D2 dopamine receptors; however, they are infrequently used in clinical practice because of their severe and unacceptable side effects.”  Dr. Coffey continued, “Of particular note is that these patients were refractory to all previous treatments; one of the subjects has elected to continue treatment, and has remained on vigabatrin for two years. Drugs in the GABA-aminotransferase inhibitor class may offer new hope to treatment-refractory TD patients who have no other acceptable options.” Dr. Brodie added, “The positive long term response generated by even a single refractory patient opens the possibility that appropriately monitored GABA-AT inhibitors may offer an additional clinical alternative in the care of younger patients who may require longer term treatment.”

The 8-week clinical trial was designed as an open label trial to evaluate the potential effect of GABA-aminotransferase inhibition as a mechanism for reducing tics in patients with treatment-refractory Tourette’s Disorder. Vigabatrin was used as a “research surrogate” to demonstrate the utility of GABA-aminotransferase (GABA-AT) blockade, with the expectation that upon successfully demonstrating the utility of this mechanism, further development activities would focus on the potentially safer, more potent GABA-AT inhibitor, CPP-115.

During the trial, each subject’s Yale Global Tic Severity Score (YGTSS), Clinical Global impression for Tics (CGI-Tics: the doctor’s assessment of motor and vocal tic symptom severity), and Global Assessment of Functioning score (GAF: an assessment of psychological, social, and occupational impairment) were collected at baseline (prior to treatment) and at various points during treatment. There was a statistically significant change between baseline and week six of treatment (the last point at which subjects were on the maximum dose) for all the tic assessments, and a clinically significant change for all of the assessments in one of the four subjects. This subject was featured as a treatment success on Mount Sinai Hospital’s Patient Stories for the Tics and Tourette’s Program web page. Two other subjects also showed a measurable change in tic assessments, but without subjective improvement. Visual field testing was conducted before and after treatment, and no significant alterations to vision were detected. One of the subjects has remained on vigabatrin for two years, and periodic visual field testing has shown no alteration in the subject’s field of vision.

In recent years, GABA-ergic drugs have become an active and promising area of research to identify potential Tourette’s Disorder treatments. Recent research at the University of Nottingham in the United Kingdom suggests that increased GABA in the supplementary motor area of the brain down-regulates tics associated with Tourette’s Disorder. GABA-AT inhibitors, like vigabatrin and its potentially safer, more potent analog, CPP-115, would increase GABA, effectively increasing the gain of this down-regulating pathway. (Original Source)

Shares of Catalyst Pharmaceutical Partners closed yesterday at $3.99. CPRX has a 1-year high of $5.10 and a 1-year low of $2.13. The stock’s 50-day moving average is $3.81 and its 200-day moving average is $3.67.

Catalyst Pharmaceuticals Inc is abiopharmaceutical company. The Company is engaged in the development and commercialization of prescription drugs targeting rare (orphan) neurological diseases and disorders.