Roadmap To Success

Clinical Development Programs and Beyond

Since Catalyst announced its strategic collaboration with BioMarin Pharmaceuticals and obtained the North American rights to amifampridine in 2012, we have made great strides in making our corporate mission a reality. Our clinical studies involving the efficacy and safety of amifampridine for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) have produced positive, statistically significant results. To date, amifampridine is the only evidence-based, FDA-approved treatment for adult patients with Lambert-Eaton Myasthenic Syndrome (LEMS). We have great expectations for this compound.

Our clinical development program is continuing to grow—in 2018, we launched a large phase 3 trial of amifampridine in myasthenia gravis patients, with a particular focus on a subgroup, called muscle-specific receptor tyrosine kinase (MuSK-MG), after positive top-line data in a small proof-of-concept study. There is also an ongoing, phase 3 trial into the efficacy and safety of amifampridine in congenital myasthenic syndromes (CMS). Another clinical development program launching in 2018 will investigate the drug’s use in patients with spinal muscular atrophy (SMA). Other possible indications for amifampridine are to be explored.

In 2018, Catalyst received FDA approval of amifampridine for the treatment of LEMS.