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Catalyst Pharmaceuticals Inc (NASDAQ:CPRX) investors have a smile on their faces Thursday morning, after the small biotech company 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. Genetically-confirmed 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 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.” . (Original Source)

On the ratings front, CPRX stock has been the subject of a number of recent research reports. In a report issued on November 14, H.C. Wainwright analyst Andrew Fein reiterated a Buy rating on CPRX. Separately, on November 10, Roth Capital’s Scott Henry reiterated a Buy rating on the stock and has a price target of $2.50.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Andrew Fein and Scott Henry have a yearly average return of 3.1% and a loss of 2.7% respectively. Fein has a success rate of 41% and is ranked #1183 out of 4271 analysts, while Henry has a success rate of 36% and is ranked #3805.

Catalyst Pharmaceuticals, Inc. is a biopharmaceutical company. It focuses on development and commercialization of prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette’s disorder.