Catalyst Pharmaceutical Partners has developed a very clear market strategy as a specialty biopharma focusing on the commercialization of orphan prescription drugs in the area of neurological disorder and disease. North American rights to the company's lead product candidate, Firdapse™, were acquired for this proprietary form of amifampridine phosphate from BioMarin Pharmaceutical back in October of 2012, and the drug has seen prior approval by the European Medicines Agency (2009, product launched in EU in 2010) for the potentially fatal, muscle weakness-characterized autoimmune disease, Lambert-Eaton Myasthenic Syndrome (LEMS). The company is in a joint development agreement (JDA) with BioMarin to split costs down the middle on future studies and will be advancing Firdapse aggressively to treat neuromuscular diseases, including LEMS.
An ongoing Phase III study in LEMS started by BioMarin is now being sponsored by CRPX and the additional applications of Firdapse in congenital myasthenic syndrome and myasthenia gravis (neuromuscular disorders characterized by muscle weakness) are a tantalizing possibility. CRPX is looking to complete enrollment of some 30 patients by later this year and anticipates reporting top-line results from the randomized double-blind, placebo-controlled discontinuation trial (as recommended by FDA) by Q2 of 2014. The study has identified solid end points and positive results should tip off an NDA filing as early as the start of 2015, with a commercial launch of Firdapse in the following year.
Back in late 2009, CRPX entered into a license agreement with Northwestern University, who originally discovered and patented several GABA aminotransferase (GABA-AT) inhibitors and derivatives of vigabatrin (an established antiepileptic sold as Sabril). The company now stands poised to capitalize mightily as they believe they control all extant IP for drugs whose primary method of action relates to GABA-AT. This opens another broad territory (besides Firdapse) for central nervous system-focused indications, and CRPX has already devised some revolutionary developments for infantile spasms, including the more severe and pronounced form called West's syndrome, via a more potent vigabatrin analog designated CPP-115.
The company has obtained FDA orphan drug designation for CPP-115 to treat infantile spasms and across the pond, similar traction with EU regulators has seen CPP-115 fitted with an orphan medicinal product designation for West's. As per the company's recent (Feb 11) R&D pipeline update, these developments put CRPX in the pole-position to obtain the respective seven-year and ten-year marketing exclusivities if they beat everyone to the punch on the NDA/MAA. Make no mistake, Catalyst is pushing hard to achieve just that and has been in constant engagement with both regulatory bodies, especially considering the excellent pre-clinical work done in CPP-115 that indicates CRPX has a real winner on their hands here (the product much more potent and has fewer side effects than vigabatrin). CPP-115 has even shown promising potential in complex partial seizures, multiple sclerosis, and dyskinesia (severe movement disorder) associated with Parkinson's, meaning that the long-term commercial horizon is vast.
The May 22, 2012, results from the company's double-blind, placebo-controlled, Phase I(a) human clinical trial of CPP-115 (IND was filed in Q4 2011 and covers through Phase II) evaluating the safety, tolerability, and pharmacokinetic profile, saw well-tolerated receptivity to all six doses administered with no significant adverse events (including respiratory and cardiovascular) and the product was absorbed quite rapidly as well (30 minutes to peak blood concentration levels achieved). The company is bucking to get further human clinical trials rolling here and obviously smells a big success in CPP-115 commercialization, so the company is currently looking for strategic partnership opportunities (as with BioMarin who has a 16% investment along with the JDA) and pursing other funding methods like NIH/foundation grant money.
The company's work developing CPP-109 originally for addictive drugs showed informative results in treating adult cocaine addiction, but this venue was discontinued due to disappointing Phase II(b) results. Luckily, the broader GABA-AT inhibitor potential of the technology here (both CPP-109 and CPP-115) in treating Tourette's, for which CRPX recently filed a provisional patent application with the USPTO (as a co-inventor alongside New York University and the Feinstein Institute for Medical Research), will most likely salvage the vector. The company is currently providing funding and CPP-109 to a small Phase I/II trial at Mt. Sinai School of Medicine in New York and the hope is to see a marked reduction in the number or frequency of tics, which would lead to development of either CPP-109 or CPP-115 for this indication (which should easily qualify for an FDA orphan drug designation).
The company has a potent two-pronged market attack strategy here with the potassium channel blocker technology (Firdapse) on the one hand and the exciting GABA-AT inhibitors on the other (CPP-109 and CPP-115), both of which show tremendous potential in treating neurological problems that desperately cry out for an answer. CPP-115 has shown potency as great as 200 times that of CPP-109 in both in vitro and animal model, with the additional massive benefit of not showing the visual field defects associated with chronic administration of vigabatrin. Given the absence of any other FDA-approved therapy for LEMS in the U.S., Firdapse could be a real mover and shaker as it addresses a significant, un-served demand.
For more information on Catalyst Pharmaceutical Partner, visit www.CatalystPharma.com
Sign up for "The Mission Report" at www.MissionIR.com