Catalyst Pharmaceutical Partners Initiates Phase 1(b) Safety and Tolerance Study for CPP-115

Catalyst Pharmaceutical Partners, Inc.

, a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced the initiation of its second Phase 1 safety and tolerance study for CPP-115, a novel GABA aminotransferase (GABA-AT) inhibitor. "We are excited to be advancing CPP-115 in the clinic," said Steven Miller, Ph.D., Chief Operating Officer/Chief Scientific Officer. "Our preclinical experience with CPP-115 has demonstrated its potential for certain epilepsy indications, infantile spasms and other neurological diseases. We expect to report the results of this study at the end of the first quarter or early in the second quarter of 2015." The Phase 1(b) trial is designed in 2 parts to evaluate the safety and tolerability of single and multiple doses of CPP-115, including CNS side effects, and respiratory and cardiovascular safety. In addition: Part 1 of the trial is an open label, single descending dose study in up to 3 cohorts of 2 normal healthy, male volunteers, intended to verify that the drug crosses the blood-brain barrier at levels sufficient to significantly raise brain GABA levels as measured by Magnetic Resonance Spectroscopy (MRS). Catalyst believes increases in brain GABA, an inhibitory neurotransmitter, to be a biomarker for the potential efficacy of CPP-115 as a treatment for Tourette Syndrome, Post-Traumatic Stress Disorder (PTSD) and infantile spasms. If the first dose shows a significant increase, lower single doses will be evaluated to determine a minimal effective dose to be able to set the doses to be evaluated in the multiple dose part of the trial. Part 2 of the study is designed as a randomized, double-blind, multiple ascending dose study in 4 cohorts of eight normal healthy, male volunteers. In addition to studying basic human safety and tolerability, the effect of multiple doses of CPP-115 on brain GABA levels will also be followed by MRS.