Catalyst Pharmaceuticals Inc (NASDAQ:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today reported on the publication of detailed results from the LMS-002 Phase 3 study of amifampridine phosphate (Firdapse®) in patients with Lambert-Eaton myasthenic syndrome (LEMS). The study results were published in the May 2016 issue of Muscle & Nerve (volume 53, number 5), authored by Dr. Shin Oh, et al.

As previously reported by Catalyst, its LMS-002 Phase 3 study was a multi-center, randomized, double blind, placebo-controlled “withdrawal trial” in which all patients were initially treated with Firdapse followed by treatment with either Firdapse or placebo during a 2-week randomization period. The study assessed the safety and efficacy of amifampridine phosphate in patients with LEMS, an ultra-rare autoimmune disease resulting in debilitating muscle weakness and other, possibly severe, symptoms. A total of 38 patients completed the trial. In this trial design, the clinically significant findings, when present, are worsening of symptoms in the placebo group.

Summary of Clinical Trial Results for Firdapse

  • Co-primary endpoints
    • The primary endpoint of change in quantitative myasthenia gravis score, or QMG, at day 14 reached statistical significance (p=0.0452).
    • The primary endpoint of change in subject global impression, or SGI, at day 14 was also statistically significant (p=0.0028).
  • Secondary endpoints
    • The secondary endpoint for the physician’s clinical global impression of improvement, or CGI-I, reached statistical significance (p=0.0267).
    • The secondary endpoint of change in walking speed at day 14 showed a worsening of 9.67 ft/min in the placebo group. As expected, this was a quantitative worsening in walking speed in the placebo group, but the magnitude of the change relative to the variance inherent in this test prevented reaching statistical significance for this endpoint with this small sample size.

“Publication in Muscle & Nerve, a leading medical publication devoted to neuromuscular disorders and treatments, further validates the strength of the Phase 3 data that showed a significant benefit for patients taking Firdapse,” stated Gary Ingenito, M.D., Ph.D., Chief Medical Officer of Catalyst. “I would also like to reiterate our appreciation to all the patients and physicians who participated in LMS-002 as we continue to work towards gaining FDAapproval of Firdapse.”

Shin J. Oh, M.D., Distinguished Professor Emeritus, University of Alabama at Birmingham, noted, “These findings demonstrate that amifampridine phosphate is safe and effective in the symptomatic treatment of LEMS.  This is an important milestone in the development of Firdapse towards an FDAapproval and access for all patients with LEMS who may benefit.” (Original Source)

Shares of Catalyst Pharmaceutical closed yesterday at $0.5551, down $-0.04 or -7.28%. CPRX has a 1-year high of $5.80 and a 1-year low of $0.51. The stock’s 50-day moving average is $0.93 and its 200-day moving average is $1.75.

On the ratings front, Catalyst has been the subject of a number of recent research reports. In a report issued on April 26, Piper Jaffray analyst Charles Duncan downgraded CPRX to Hold, with a price target of $1, which implies an upside of 80.1% from current levels. Separately, on March 22, Roth Capital’s Scott Henry reiterated a Buy rating on the stock and has a price target of $3.25.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Charles Duncan and Scott Henry have a total average return of -11.9% and -2.5% respectively. Duncan has a success rate of 32.7% and is ranked #3739 out of 3833 analysts, while Henry has a success rate of 35.5% and is ranked #3291.

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.