Catalyst Pharmaceuticals Inc (NASDAQ:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today provided an update on the content of the planned resubmission of the New Drug Application (“NDA”) for Firdapse® (amifampridine phosphate), which currently has Breakthrough Therapy and Orphan Drug designations for Lambert-Eaton myasthenic syndrome (LEMS). Catalyst recently met with the Food and Drug Administration (“FDA”) to obtain greater clarity regarding what will be required by the FDA to accept the Firdapse NDA for filing.
The FDA has stated that in addition to the results of the submitted multi-center, randomized, placebo-controlled, Phase 3 LMS-002 trial, the Company will need to submit positive results from an additional adequate and well-controlled study in patients with LEMS. The FDA has stated that it is open to discuss a study design that could efficiently accomplish the requirement with a small, short-term study. Additionally, there is a requirement for several more short-term toxicology studies, which are expected to start soon.
“While we are very disappointed by this delay, Catalyst and our employees remain committed to working with the FDA and bringing Firdapse to market for patients suffering with LEMS and congenital myasthenic syndromes (CMS). We are surprised with the FDA’s request for an additional clinical study for Firdapse, but are encouraged that the agency is open to an efficient, small short-term study design. We are currently in discussions with the FDA, and our clinical experts regarding the protocol and logistics for this confirmatory study,” stated Patrick J, McEnany, Chief Executive Officer of Catalyst. “As always, we remain committed to LEMS and CMS patients with our continued research and our expanded access program, which continues to enroll new patients and provide Firdapse at no charge to eligible patients.”
Catalyst is also continuing the development of Firdapse for additional indications and has recently initiated an investigator-sponsored study of Firdapse in patients with MuSK-Antibody Positive Myasthenia Gravis. This year the company also expects to complete a clinical trial of Firdapse for pediatric patients with CMS.
As of March 31, 2016 the company had approximately $52 million in cash and investments. The Company believes that it has the cash resources required to complete the additional studies, as well as funding operations. Catalyst will announce first quarter financial results and provide further updates by conference call on May 10, 2016. (Original Source)
Shares of Catalyst Pharmaceutical closed yesterday at $1.24, up $0.03 or 2.48%. CPRX has a 1-year high of $5.80 and a 1-year low of $0.93. The stock’s 50-day moving average is $1.19 and its 200-day moving average is $2.06.
On the ratings front, Catalyst Pharmaceutical has been the subject of a number of recent research reports. In a report issued on March 22, Roth Capital analyst Scott Henry reiterated a Buy rating on CPRX, with a price target of $3.25, which represents a potential upside of 162.1% from where the stock is currently trading. Separately, on February 17, Piper Jaffray’s Charles Duncan reiterated a Buy rating on the stock and has a price target of $6.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Scott Henry and Charles Duncan have a total average return of 2.1% and -5.5% respectively. Henry has a success rate of 39.0% and is ranked #1189 out of 3829 analysts, while Duncan has a success rate of 42.5% and is ranked #3554.
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. The company was founded by McEnany J. Patrick and Huckel E. Hubert in January 2002 and is headquartered in Coral Gables, FL.