Catalyst Pharmaceuticals Inc (NASDAQ: CPRX) rose Monday, after the company disclosed 'positive' top-line results from second Phase 3 clinical trial of Firdapse in patients with Lambert-Eaton myasthenic syndrome.
The biopharmaceutical company out of Coral Gables, Florida focuses on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases.
Catalyst announced positive top-line results from a second Phase 3 clinical trial (LMS-003) of Firdapse® (amifampridine phosphate tablets equivalent to 10 mg amifampridine) for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS).
Firdapse received Breakthrough Therapy designation from the Food and Drug Administration (FDA) in August 2013 and this clinical trial was conducted using a protocol agreed to by the FDA through the Special Protocol Assessment (SPA) process.
Said Catalyst CEO Patrick McEnany, “We are extremely pleased with the top-line efficacy and safety results from this second Phase 3 trial, which reinforces the potential of Firdapse to be an important treatment for patients suffering from LEMS.
“We look forward to presenting further data in future publications and at medical conferences. We remain on track to submit our NDA to the FDA in the first quarter of 2018. Catalyst remains committed to the development of therapies that will improve the function and lives of people with rare neurodegenerative diseases.”
A total of 26 patients completed the randomized treatment. In a trial of this design, the clinically significant findings, when present, are worsening of symptoms in the placebo group.
Shares in the company climbed 12 cents, or 3.4%, to $3.62.
