CORAL GABLES, Fla., Feb. 11, 2013 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX), a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neurological diseases and disorders, today provided an update on its research and development pipeline.
FirdapseIn October 2012, Catalyst acquired the North American rights to Firdapse, a proprietary form of amifampridine phosphate (3-4 diaminopyridine or 3-4 DAP), from BioMarin Pharmaceutical Inc. ("BioMarin"). Firdapse was approved in December 2009 by the European Medicines Agency for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), a rare and sometimes fatal autoimmune disease characterized by muscle weakness. Firdapse has been granted orphan drug designation by the U.S. Food & Drug Administration, (FDA) for the treatment of LEMS, making the product eligible to obtain seven-year marketing exclusivity, if Catalyst is the first pharmaceutical company to obtain approval of an NDA for its formulation of amifampridine. As part of its license agreement with BioMarin, Catalyst is taking over the sponsorship of their ongoing Phase III clinical trial evaluating amifampridine phosphate for the treatment of LEMS. The trial:
For further details on this trial, please go to: www.clinicaltrials.gov ; Search "amifampridine phosphate". With respect to the trial, Catalyst expects:
- is designed as a randomized double-blind, placebo-controlled discontinuation trial as recommended by FDA to BioMarin;
- has a goal to enroll approximately 30 LEMS patients (approximately one third enrolled currently);
- currently has 7 active sites (expected to be increased to approximately 25 in the near future);
- has defined as a primary endpoint-change in muscle strength during the 2-week, double-blind discontinuation period as determined using a validated questionnaire (Quantitative Myasthenia Gravis score); and
- has defined as a secondary endpoint-change in walking speed (timed 25-foot walk test) during the discontinuation period.
Assuming positive results are obtained from the trial, Catalyst hopes:
- to complete enrollment by the end of 2013; and
- to report top-line results from the double-blind portion of this clinical trial during the second quarter of 2014.
Firdapse may also be an effective treatment for other neuromuscular orphan indications:
- to file an NDA for Firdapse in the first quarter of 2015;
- to obtain approval from the FDA of such NDA by the end of 2015; and
- to commercially launch this product sometime in the first half of 2016.
Catalyst believes Firdapse can achieve peak annual revenues from sales in the United States of approximately $100 million.
- Congenital Myasthenic Syndrome; and
- Myasthenia Gravis.
CPP-115On August 27, 2009, Catalyst entered into a license agreement with Northwestern University (Northwestern), under which it acquired worldwide rights to commercialize new GABA aminotransferase inhibitors and derivatives of vigabatrin which were discovered and patented by Northwestern. Catalyst has designated the lead compound to be developed under this license as CPP-115. CPP-115 has been granted orphan drug designation by the FDA for the treatment of infantile spasms and orphan medicinal product designation in the European Union (EU) for West's syndrome (a form of infantile spasms). This means this product will be eligible to obtain the seven-year and ten-year marketing exclusivities available from the FDA and the EU, respectively, if Catalyst is the first pharmaceutical company to obtain approval of an NDA/MAA for CPP-115.