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 from its Phase 1(b) double-blind, placebo controlled safety and tolerance study of CPP-115 in normal healthy volunteers.  The results showed significant increases in brain levels of the surrogate marker for potential efficacy, gamma-aminobutyric acid (GABA).  The main adverse effect of prolonged elevated brain GABA, somnolence, was also observed.

While the primary objective of this study was to obtain safety and tolerance data for CPP-115 administered over 14 days, brain GABA levels were measured as a surrogate marker of potential efficacy, since CPP-115 is a second generation GABA aminotransferase inhibitor.  Specifically, this study examined GABA levels in both the POC (Parietal-Occipital Cortex) a grey matter rich region thought to be associated with epilepsy, and which was previously studied for vigabatrin, and the SMA (Supplementary Motor Area), which is thought to be associated with Tourette’s Disorder.  The maximum brain GABA increases, in both brain regions, ranged from about 150% to over 200% of baseline levels, as measured by magnetic resonance spectroscopy (MRS).

Patrick J. McEnany, Catalyst’s Chief Executive Officer stated, “These data are promising, because they provide evidence that CPP-115 significantly raises brain GABA, a mechanism known to effectively treat epilepsy, infantile spams, and potentially Tourette’s disorder.  CPP-115 was also found to be even more potent than anticipated, which may indicate that a lower dose of CPP-115 has the potential to effectively treat these diseases.  A low drug dose may reduce or eliminate the risk of visual field defects, a side effect associated with vigabatrin.”

Steven Miller, Ph.D., Catalyst’s Chief Operating Officer and Chief Scientific Officer stated, “We expect to receive the full results of this study early next year, including additional data from laboratory safety tests, pharmacokinetic modeling, and brain levels of CPP-115 as determined from MRS data of the patients in this study. Once we obtain the full results of this study, we intend to determine the steps required to make CPP-115 “Phase 2 ready,” including a Phase 1 dose ranging study evaluating CPP-115 at lower doses.” (Original Source)

Shares of Catalyst Pharmaceutical Partners closed yesterday at $2.48, up $0.08 or 3.33%. CPRX has a 1-year high of $5.80 and a 1-year low of $2.27. The stock’s 50-day moving average is $2.82 and its 200-day moving average is $3.75.

On the ratings front, Catalyst Pharmaceutical has been the subject of a number of recent research reports. In a report issued on September 22, Piper Jaffray analyst Charles Duncan reiterated a Buy rating on CPRX, with a price target of $7, which implies an upside of 182.3% from current levels. Separately, on September 10, Roth Capital’s Scott Henry maintained a Buy rating on the stock and has a price target of $7.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Charles Duncan and Scott Henry have a total average return of 4.3% and 9.6% respectively. Duncan has a success rate of 41.3% and is ranked #867 out of 3632 analysts, while Henry has a success rate of 41.4% and is ranked #320.

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