Clinical Trials
Clinical Trials

Catalyst has an extensive clinical development program at various stages of progression. The core mission of the Catalyst development program is to advance the treatment landscape for patients with rare, debilitating neuromuscular and neurological diseases.

Therapeutic areas of concentration

Lambert-Eaton Myasthenic Syndrome (LEMS)

Catalyst has completed two, phase 3 trials for amifampridine phosphate in the treatment of Lambert-Eaton myasthenic syndrome (LEMS). Both randomized trials resulted in positive, statistically significant data and in 2018 Catalyst will file a NDA for amifampridine phosphate for the treatment of LEMS. The FDA has assigned amifampridine phosphate as a “Breakthrough Therapy Designation” and an “Orphan Drug Designation” for LEMS.

For more information about the 1st LEMS trial, click here or go to clinicaltrials.gov.

For more information about the 2nd  LEMS trial, click here or go to clinicaltrials.gov.

Congenital Myasthenic Syndromes (CMS)

Catalyst has an ongoing, phase 3 trial evaluating the efficacy and safety of amifampridine phosphate in the treatment of certain genetic subtypes of CMS.  This randomized, double-blind, placebo controlled, crossover study has an estimated completion date of July 2018 and Catalyst will also be filing a NDA. The FDA has already assigned amifampridine phosphate an “Orphan Drug Designation” in CMS.

For more information about the CMS trial, click here or go to clinicaltrials.gov.

Muscle-Specific Receptor Tyrosine Kinase Myasthenia Gravis (MuSK-MG)

In 2018, Catalyst is recruiting patients for a phase 3 trial to evaluate the efficacy and safety of amifampridine phosphate in the treatment of MuSK-MG. The primary efficacy endpoint of THE MuSK Trial will be the degree to which amifampridine phosphate may help improve the daily activities in patients with MuSK-MG, as measured by improvement in MG-ADL score compared with placebo. The FDA has assigned amifampridine phosphate an orphan drug designation in MuSK-MG. For more information about the MuSK-MG trial, click here. To view eligibility requirements or for more information about the trial itself go to clinicaltrials.gov.

MG-ADL=myasthenia gravis activities of daily living.