Catalyst Pharmaceutical Partners Provides Updates On Progress of Phase 3 Firdapse Study In Patients With Lambert-Eaton Myasthenic Syndrome (LEMS)

Catalyst Pharmaceutical Partners, Inc. CPRX, a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today provided an update on the progress of its Phase 3 study of FirdapseTM (amifampridine phosphate) for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS). The last patient has completed the blinded portion of the study and top-line data remains on track to be reported later this quarter. A total of 38 patients completed the 3 month treatment period and were successfully randomized to either receive FirdapseTM or placebo. All patients who were randomized elected to continue in 2-year open label follow-up. If the company obtains positive results from its Phase 3 study, the company intends to submit a request to the FDA for a pre-NDA meeting and to initiate the steps necessary to seek regulatory approval of FirdapseTM. This would likely include the initiation of a rolling NDA submission to the FDA by early-2015. The FirdapseTM Phase 3 trial utilizes a randomized, double-blind, placebo-controlled, discontinuation design. The trial is being conducted at sites in the United States and Europe. Following enrollment, patients were treated with open label drug for a minimum of 91 days, and then randomized to either continue on FirdapseTM or be discontinued to placebo over a 2-week period. Following the randomization phase of the trial, patients were then eligible to receive open label FirdapseTM treatment for a two-year follow-up period, to obtain additional long term safety data. LMS-002 Patient Disposition Highlights Thirty eight (38) patients were successfully randomized into the 2-week, randomized, double-blind, placebo-controlled, discontinuation part of the study. The original target was to randomize 36 patients. All 38 randomized subjects elected to continue to receive FirdapseTM treatment in the 2-year, open label follow-up part of the trial. Patrick J. McEnany, Chief Executive Officer of Catalyst Pharmaceutical Partners, Inc., stated, "We are very pleased to be nearing completion of the Phase 3 FirdapseTM pivotal trial for patients diagnosed with LEMS, a rare neuromuscular disease, and remain on track to report data later this quarter. We were very pleased to see 100% of the patients in the randomized portion of the trial elect to continue into the open-label 2-year safety study. Assuming we receive positive data from this study, we plan to meet with the FDA in the fourth quarter to determine the fastest U.S. registration pathway. We remain committed to the LEMS patient population and are pleased to see positive feedback from our expanded access program initiated earlier this year. We hope to see FirdapseTM become the first FDA-approved, safe and effective drug for the treatment of this debilitating disease." About FirdapseTM FirdapseTM, amifampridine phosphate or 3,4-diaminopyridine (3,4-DAP) phosphate, is a potassium channel inhibitor. By blocking this ion channel, FirdapseTM increases the nerve repolarization time, which causes an increase in the influx of calcium, thereby causing more acetylcholine to be released, which restores muscle fiber contraction, thus relieving muscle weakness caused by LEMS. In addition to LEMS, other potential orphan neuromuscular indications for FirdapseTM include certain types of Myasthenia Gravis and Congenital Myasthenic Syndrome, among others. FirdapseTM has been granted orphan drug and breakthrough therapy designations by the FDA, and orphan medicinal product designation in the European Union for the treatment of LEMS. It is approved and commercialized in the E.U. by BioMarin Pharmaceutical. Upon completion of the Phase 3 trial, assuming the data from the trial is positive, Catalyst expects to begin submitting a rolling new drug application to the FDA in 2015.