Catalyst Pharmaceuticals Reveals Data on Neurological Drug

Catalyst Pharmaceuticals, Inc. CPRX announced encouraging top-line results from a phase I study on pipeline candidate, CPP-115.

The double-blind, placebo-controlled, safety and tolerance study was conducted among healthy volunteers. The primary endpoint of the study was to obtain safety and tolerance data for CPP-115 administered over 14 days. The study also measures brain gamma-aminobutyric acid (GABA) levels as a surrogate marker of potential efficacy, since CPP-115 is a second-generation GABA aminotransferase inhibitor.

Catalyst Pharma is evaluating CPP-115 for the treatment of infantile spasms, epilepsy and other neurological conditions that are associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette’s Disorder.

Data from the study showed that CPP-115 significantly raises brain GABA, which can treat epilepsy, infantile spams and potentially Tourette's disorder. It was also observed that CPP-115 was more potent than anticipated. Hence, a lower dose of CPP-115 can potentially effectively treat these diseases, which in turn, may reduce or eliminate the risk of visual field defects, a side effect associated with Sabril.

We note that the candidate enjoys Orphan Drug designation in the U.S. for the treatment of infantile spasms. CPP-115 also enjoys orphan medicinal product designation in the EU for the treatment of West Syndrome.

Catalyst Pharma expects full results from the study in early 2016. The company will thereafter decide on a phase I dose-ranging study evaluating CPP-115 at lower doses and steps required to make CPP-115 ready for phase II.

Meanwhile, Catalyst Pharma’s lead candidate, Firdapse, is being evaluated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS). The drug is already approved in Europe for symptomatic treatment in adults with LEMS.

Catalyst Pharma carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Corcept Therapeutics CORT, Mylan MYL and Baxalta Inc. BXLT. All three sport a Zacks Rank #1 (Strong Buy).

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