Catalyst Pharmaceuticals Inc (NASDAQ:CPRX) investors cheer the news that the biopharmaceutical company has reached an agreement with the FDA under a Special Protocol Assessment (SPA) for the protocol design, clinical endpoints, and statistical analysis approach to be taken in Catalyst’s upcoming Phase 3 study evaluating Firdapse (amifampridine phosphate) for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS).
Catalyst Pharmaceuticals shares reacted to the news, rising nearly 14% to $1.12 in pre-market trading Monday.
“Receipt of this SPA agreement is a major milestone which provides us with a clearly defined development and regulatory pathway for Firdapse in the treatment of LEMS,” said Patrick J. McEnany, Catalyst’s Chief Executive Officer. “We would like to thank the FDA for its engagement and guidance in this process.”
Catalyst intends to conduct its second Phase 3 trial (designated as LMS-003) at two clinical trial sites, one on the east coast of the United States and one on the west coast of the United States. This double-blind, placebo controlled withdrawal trial will include approximately 28 subjects, so that the trial is adequately powered, and will have the same co-primary endpoints as Catalyst’s first Phase 3 trial evaluating Firdapse for the treatment of LEMS. Further, the FDA has agreed to allow Catalyst to enroll patients from its expanded access program as study subjects in this second trial. Finally, after further discussion with the FDA in connection with the SPA request, this second trial will be a parallel design and not a cross-over design. (Original Source)
On the ratings front, Catalyst Pharmaceutical has been the subject of a number of recent research reports. In a report issued on October 13, Roth Capital analyst Scott Henry reiterated a Buy rating on CPRX, with a price target of $2.50, which represents a potential upside of 155% from where the stock is currently trading. Separately, on October 5, Piper Jaffray’s Charles Duncan upgraded the stock to Buy .
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 yearly average loss of 0.7% and 12.1% respectively. Henry has a success rate of 37% and is ranked #2992 out of 4178 analysts, while Duncan has a success rate of 30% and is ranked #3987.
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.