Catalyst Pharma Announces Positive Top-Line Phase 3 Data From Firdapse Trial

(RTTNews) - Catalyst Pharmaceutical Partners, Inc. (CPRX) on Monday announced positive top-line results from the pivotal Phase 3 clinical trial of Firdapse for the symptomatic treatment of Lambert-Eaton myasthenic syndrome.

According to the company, both co-primary endpoints, quantitative myasthenia gravis score and subject global impression, showed that Firdapse was significantly superior to placebo, as did a secondary endpoint for the physician's clinical global impression of improvement.

The clinical trial was designed as a double blind, randomized, "withdrawal trial" in which all patients were initially treated with Firdapse during a 91 day run-in period followed by treatment with either Firdapse or placebo during a 2 week randomization period. A total of 38 patients completed the 3 month run-in period and subsequent 2 week randomization period. In a trial of this design, the clinically significant findings, when present, are worsening of symptoms in the placebo group.

Patrick McEnany, Chief Executive Officer of Catalyst Pharmaceuticals, said, "These results show that Firdapse provides an important benefit to LEMS patients and continues to demonstrate a favorable safety profile. We plan to meet with the FDA in the near future to determine the fastest way to get an NDA for Firdapse approved. We are committed to the LEMS patient community and are pleased that we will be launching our expanded access program next month. This program will provide Firdapse at no charge to patients who meet the inclusion/exclusion requirements."