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Catalyst Pharmaceuticals Announces Initiation of Investigator-Sponsored Study of Firdapse in Patients With MuSK-Antibody Positive Myasthenia Gravis

CORAL GABLES, Fla., Feb. 08, 2016 (GLOBE NEWSWIRE) — Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced the initiation of an investigator-sponsored, adequate and well-controlled clinical trial evaluating safety, tolerability and efficacy of Firdapse® (amifampridine phosphate) as a symptomatic treatment for patients with MuSK-antibody positive myasthenia gravis (MuSK-MG).

The study will be conducted by a team of researchers led by Renato Mantegazza, MD, Director, Department of Neuroimmunology and Neuromuscular Diseases, Fondazione Istituto Neurologico Carlo Besta in Milan, Italy, a major referral center for MuSK-MG patients. The study is designed as a randomized (1:1), double-blind, placebo-controlled, crossover, outpatient study to evaluate the safety, tolerability and potential efficacy of amifampridine in patients diagnosed with MuSK‑MG. The study is planned to include approximately 20 male and female patients, and Catalyst anticipates reporting top-line results from the study in about a year. Catalyst is providing study drug and financial support for the study.

Patrick J. McEnany, Chief Executive Officer of Catalyst, said, “We are very pleased to provide support to Dr. Mantegazza and his team at Istituto Neurologico Carlo Besta for this study of Firdapse in patients diagnosed with MuSK-antibody positive myasthenia gravis. Unapproved or off-label treatments for patients with this disease have limited efficacy and significant side effects. If the results from this trial support the safety and efficacy of Firdapse as a treatment for MuSK-MG, we intend to submit an application for orphan drug designation and pursue approval of this product for this indication.”

The rationale for this trial comes from published positive results obtained with 3,4-DAP (amifampridine) in two preclinical studies conducted in animal models of MuSK antibody positive myasthenia gravis by research teams in Australia (Morsch et al., 20131) and Japan (Mori et al., 20122).