Catalyst Pharmaceuticals Completes Enrolment for a Study of Amifampridine Phosphate in Ambulatory Patients who have SMA Type 3

03 August 2020

Catalyst, a biopharmaceutical company focused on developing innovative therapies for people with rare debilitating neuromuscular and neurological diseases, is evaluating amifampridine phosphate, a neuronal potassium channel blocker, for the treatment of SMA.

About the drug

Amifampridine has been approved by the European Medicines Agency for the treatment of adult patients with Lambert-Eaton Myasthenic Syndrome (LEMS). LEMS is a rare autoimmune condition that affects the specialised connections between lower motor neurons and muscles known as neuromuscular junctions.

The medicine works by increasing the amount of a chemical “message” called acetylcholine that is released into the neuromuscular junction. It is through acetylcholine that nerve cells communicate with muscle cells and control their movement. Current evidence demonstrates some improvement in LEMS patient muscle function from treatment with amifampridine.

About the study

This is a randomised, double-blind, placebo-controlled outpatient study, designed to evaluate the safety, tolerability and efficacy of amifampridine phosphate in ambulatory, nusinersen-naïve patients diagnosed with SMA Type 3.

The study has enrolled 12 SMA Type 3 patients (both male and female patients), aged between 6 and 50 years in sites in Italy and Serbia. Patients will only be taking the assigned investigational product (amifampridine phosphate 10 mg tablets or matching placebo tablets), and no new therapies are permitted during the study. Having met its target patient number, this study is currently closed for any new enrolment. 

More information

About the trial
About Catalyst Pharmaceuticals