Catalyst Pharmaceuticals Announces Phase 2 Study of Firdapse® in Ambulatory Patients SMA Type 3

CORAL GABLES, Fla., Nov. 21, 2017 (GLOBE NEWSWIRE) — Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today announced the initiation of a company-sponsored, adequate and well-controlled, proof-of-concept clinical trial evaluating safety, tolerability and efficacy of Firdapse® (amifampridine phosphate) as a symptomatic treatment for patients with Spinal Muscular Atrophy (SMA) Type 3.

The study will be conducted by a team of researchers led by Lorenzo Maggi, MD, and Giovanni Baranello, MD, of the Fondazione Istituto Neurologico Carlo Besta in Milan, Italy, a major referral center for SMA patients. The study is designed as a randomized (1:1), double-blind, 2-period, 2-treatment, crossover, outpatient proof-of-concept study to evaluate the safety, tolerability and potential efficacy of amifampridine in ambulatory patients diagnosed with SMA Type 3.

The study is planned to include approximately 12 patients, and Catalyst anticipates reporting top-line results from the study in the first half of 2019.