- Allowing adult CMS patients to participate
- Number of patients increased to approximately 20
- Additional site added
CORAL GABLES, Fla., Dec. 8, 2016 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today provided an update on its clinical trial with amifampridine phosphate in patients with congenital myasthenic syndromes (CMS). After discussions with the U.S. Food and Drug Administration (FDA) the study has been expanded beyond pediatric patients to include adult CMS patients and the enrollment size has been increased to approximately 20 patients.
Further, there are now a total of five sites participating in the study:
? Children’s Healthcare of Atlanta
? Johns Hopkins Pediatric Neurology
? Boston Children’s Hospital
? Nationwide Children’s Hospital
? University of California, Los Angeles, Department of Neurology
Amifampridine phosphate, Firdapse®, has received Breakthrough Therapy Designation from the FDA for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), as well as orphan drug designations for LEMS, CMS, and Myasthenia Gravis.
Patrick J. McEnany, Catalyst’s Chief Executive Officer said, “We are pleased to enhance and expand our clinical study to include adults with CMS, as we evaluate the efficacy and safety of amifampridine phosphate in patients of all ages diagnosed with certain genetic mutations of CMS. Assuming the data from our study is positive, we will work towards including data and information on the benefits of amifampridine phosphate for CMS in our new drug application that we plan to submit for Firdapse. Additionally, we also expect to include in a new submission the positive results seen to date in children with CMS who are currently being treated with amifampridine phosphate under an investigator treatment IND. We continue to believe that we will report top-line results from this study in the second half of 2017.”
Dr. Gary Ingenito, Ph.D., Catalyst’s Chief Medical Officer said, “Though CMS is primarily diagnosed in infancy and childhood, adult patients who have been diagnosed with other neuromuscular diseases have been found to have CMS; thus, we are pleased to add this population to our clinical study. Geneticallyconfirmed CMS patients older than two years of age diagnosed with acetylcholine receptor defect, Rapsyn deficiency, MuSK deficiency, Dok-7 deficiency, SYT2 mutations, SNAP25B deficiency, and fast channel 2 syndrome may be eligible for this study. Genetic testing will be provided to potential participants whose CMS has not been genetically-confirmed within an eligible diagnosis.”
Additional information about this trial (NCT02562066) can be found on www.clinicaltrials.gov.
About Congenital Myasthenic Syndromes
Congenital myasthenic syndromes, or CMS, are rare neuromuscular disorders comprising a spectrum of genetic defects and is characterized by fatigable weakness of skeletal muscles with usual onset at or shortly after birth or early childhood; in rare cases symptoms may not manifest themselves until later in childhood or adulthood. The severity and course of the disease are variable, ranging from minor symptoms to progressive disabling weakness; symptoms may be mild, but sudden severe exacerbations of weakness or even sudden episodes of respiratory insufficiency also occur.
Congenital myasthenic syndromes are rare, estimated at one-tenth that of myasthenia gravis, which in itself is rare. Based on currently available information, Catalyst estimates that there are between 1,000 and 1,500 CMS patients in the United States.
About Catalyst Pharmaceuticals
Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, including Lambert-Eaton myasthenic syndrome (LEMS), congenital myasthenic syndromes (CMS), infantile spasms, and Tourette’s Disorder. Firdapse for the treatment of LEMS has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and orphan drug designation for LEMS, CMS and Myasthenia Gravis. Firdapse is the first and only approved drug in Europe for symptomatic treatment in adults with LEMS. Catalyst is also developing CPP-115 to treat infantile spasms, epilepsy and other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette’s Disorder. CPP-115 has been granted U.S. orphan drug designation for the treatment of infantile spasms by the FDA and has been granted E.U. orphan medicinal product designation for the treatment of West Syndrome by the European Commission. In addition, Catalyst is developing a generic version of Sabril® (vigabatrin).