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 phosphate in 2012, we have made great strides in making our corporate mission a reality. To date, our clinical studies involving the efficacy and safety of amifampridine phosphate for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) have produced positive, statistically significant results. We have great expectations for this compound.

Our clinical development program is continuing to grow—in 2018, we are launching a large phase 3 trial of amifampridine phosphate 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 phosphate 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 phosphate are to be explored.

In 2018, Catalyst plans to file an NDA for amifampridine phosphate for the treatment of LEMS and CMS.

Additionally, with our strategic collaboration with Northwestern University and in-licensed worldwide rights to a group of next-generation GABA-aminotransferase (GABA-AT) inhibitors, we are exploring potential indications in underserved patients.

CPP-115 is one of these next-generation GABA-AT inhibitors, and we believe it shows tremendous promise in the treatment of infantile spasms.

CPP-115 has received orphan drug designation in the United States for infantile spasms and in the EU for West syndrome, a severe type of infantile spasms. Catalyst has begun the clinical development of CPP-115 by completing a randomized, double-blind, single ascending dose phase 1a study in healthy volunteers, and early results indicate that CPP-115 is well tolerated. CPP-115 has progressed to the point where phase 2 studies may begin.