CORAL GABLES, FL, USA I February 08, 2016 I Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced the initiation of an investigator-sponsored, adequate and well-controlled clinical trial evaluating safety, tolerability and efficacy of Firdapse® (amifampridine phosphate) as a symptomatic treatment for patients with MuSK-antibody positive myasthenia gravis (MuSK-MG).
The study will be conducted by a team of researchers led by Renato Mantegazza, MD, Director, Department of Neuroimmunology and Neuromuscular Diseases, Fondazione Istituto Neurologico Carlo Besta in Milan, Italy, a major referral center for MuSK-MG patients. The study is designed as a randomized (1:1), double-blind, placebo-controlled, crossover, outpatient study to evaluate the safety, tolerability and potential efficacy of amifampridine in patients diagnosed with MuSK‑MG. The study is planned to include approximately 20 male and female patients, and Catalyst anticipates reporting top-line results from the study in about a year. Catalyst is providing study drug and financial support for the study.
Patrick J. McEnany, Chief Executive Officer of Catalyst, said, “We are very pleased to provide support to Dr. Mantegazza and his team at Istituto Neurologico Carlo Besta for this study of Firdapse in patients diagnosed with MuSK-antibody positive myasthenia gravis. Unapproved or off-label treatments for patients with this disease have limited efficacy and significant side effects. If the results from this trial support the safety and efficacy of Firdapse as a treatment for MuSK-MG, we intend to submit an application for orphan drug designation and pursue approval of this product for this indication.”
The rationale for this trial comes from published positive results obtained with 3,4-DAP (amifampridine) in two preclinical studies conducted in animal models of MuSK antibody positive myasthenia gravis by research teams in Australia (Morsch et al., 20131) and Japan (Mori et al., 20122).
1 Morsch M, Reddel SW, Ghazanfari N, et al. Pyridostigime, but not 3,4-diaminopyridine exacerbates ACh receptor loss and myasthenia induced in mice by muscle-specific kinase autoantibody. J Physiol 591[10], 2747-2762. 2013.
2 Mori S, Kishi M, Kubo S, et al. 3,4-diaminopyridine improves neuromuscular transmission in a MuSK antibody-induced mouse model of myasthenia gravis. J Neuroimmunol 245, 75‑78. 2012.
About MuSK Antibody-Positive Myasthenia Gravis
MuSK-MG is a rare disease that is estimated to inflict 5-8% of all myasthenia gravis patients (equating to an estimate of approximately 4,500 patients in the United States). MuSK antibodies identify a clinically distinguishable, more severe form of MG. The disease is characterized by a predominance in females, prominent bulbar involvement, more severe clinical condition and resistance to treatment. Although many patients with MuSK‑MG are presently treated with anticholinesterase inhibitors or immunosuppressants, such patients do not generally respond adequately to these treatments.
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. Catalyst’s lead candidate, Firdapse® for the treatment of LEMS, has completed testing in a global, multi-center, double-blinded randomized pivotal Phase 3 trial resulting in positive top-line data and Catalyst has recently filed an NDA for this product seeking approval for its use as a treatment of LEMS and CMS. Firdapse for the treatment of LEMS has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and orphan drug designation for LEMS and CMS. Firdapse is the first and only European approved drug for symptomatic treatment in adults with LEMS.
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).
SOURCE: Catalyst Pharmaceuticals
Post Views: 136
CORAL GABLES, FL, USA I February 08, 2016 I Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced the initiation of an investigator-sponsored, adequate and well-controlled clinical trial evaluating safety, tolerability and efficacy of Firdapse® (amifampridine phosphate) as a symptomatic treatment for patients with MuSK-antibody positive myasthenia gravis (MuSK-MG).
The study will be conducted by a team of researchers led by Renato Mantegazza, MD, Director, Department of Neuroimmunology and Neuromuscular Diseases, Fondazione Istituto Neurologico Carlo Besta in Milan, Italy, a major referral center for MuSK-MG patients. The study is designed as a randomized (1:1), double-blind, placebo-controlled, crossover, outpatient study to evaluate the safety, tolerability and potential efficacy of amifampridine in patients diagnosed with MuSK‑MG. The study is planned to include approximately 20 male and female patients, and Catalyst anticipates reporting top-line results from the study in about a year. Catalyst is providing study drug and financial support for the study.
Patrick J. McEnany, Chief Executive Officer of Catalyst, said, “We are very pleased to provide support to Dr. Mantegazza and his team at Istituto Neurologico Carlo Besta for this study of Firdapse in patients diagnosed with MuSK-antibody positive myasthenia gravis. Unapproved or off-label treatments for patients with this disease have limited efficacy and significant side effects. If the results from this trial support the safety and efficacy of Firdapse as a treatment for MuSK-MG, we intend to submit an application for orphan drug designation and pursue approval of this product for this indication.”
The rationale for this trial comes from published positive results obtained with 3,4-DAP (amifampridine) in two preclinical studies conducted in animal models of MuSK antibody positive myasthenia gravis by research teams in Australia (Morsch et al., 20131) and Japan (Mori et al., 20122).
1 Morsch M, Reddel SW, Ghazanfari N, et al. Pyridostigime, but not 3,4-diaminopyridine exacerbates ACh receptor loss and myasthenia induced in mice by muscle-specific kinase autoantibody. J Physiol 591[10], 2747-2762. 2013.
2 Mori S, Kishi M, Kubo S, et al. 3,4-diaminopyridine improves neuromuscular transmission in a MuSK antibody-induced mouse model of myasthenia gravis. J Neuroimmunol 245, 75‑78. 2012.
About MuSK Antibody-Positive Myasthenia Gravis
MuSK-MG is a rare disease that is estimated to inflict 5-8% of all myasthenia gravis patients (equating to an estimate of approximately 4,500 patients in the United States). MuSK antibodies identify a clinically distinguishable, more severe form of MG. The disease is characterized by a predominance in females, prominent bulbar involvement, more severe clinical condition and resistance to treatment. Although many patients with MuSK‑MG are presently treated with anticholinesterase inhibitors or immunosuppressants, such patients do not generally respond adequately to these treatments.
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. Catalyst’s lead candidate, Firdapse® for the treatment of LEMS, has completed testing in a global, multi-center, double-blinded randomized pivotal Phase 3 trial resulting in positive top-line data and Catalyst has recently filed an NDA for this product seeking approval for its use as a treatment of LEMS and CMS. Firdapse for the treatment of LEMS has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and orphan drug designation for LEMS and CMS. Firdapse is the first and only European approved drug for symptomatic treatment in adults with LEMS.
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).
SOURCE: Catalyst Pharmaceuticals
Post Views: 136
