Catalyst Pharmaceutical's Firdapse Phase 3 Study Published in Muscle & Nerve
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
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. Firdpase for the treatment of LEMS has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and Orphan Drug Designations for LEMS and CMS. Firdapse is the first and only drug approved in
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).
Forward-Looking Statements
This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including whether the receipt of breakthrough therapy designation for Firdapse will expedite the
development and review of Firdapse by the
Investor ContactSource:Brian Korb The Trout Group LLC (646) 378-2923 bkorb@troutgroup.com Company ContactPatrick J. McEnany Catalyst Pharmaceuticals Chief Executive Officer (305) 420-3200 pmcenany@catalystpharma.com Media ContactsDavid Schull Matt Middleman , M.D.Russo Partners (212) 845-4271 (212) 845-4272 david.schull@russopartnersllc.com matt.middleman@russopartnersllc.com
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