CORAL GABLES, FL, USA I December 16, 2015 I Catalyst Pharmaceuticals, Inc.(Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced top line results from its Phase 1(b) double-blind, placebo controlled safety and tolerance study of CPP-115 in normal healthy volunteers. The results showed significant increases in brain levels of the surrogate marker for potential efficacy, gamma-aminobutyric acid (GABA). The main adverse effect of prolonged elevated brain GABA, somnolence, was also observed.
While the primary objective of this study was to obtain safety and tolerance data for CPP-115 administered over 14 days, brain GABA levels were measured as a surrogate marker of potential efficacy, since CPP-115 is a second generation GABA aminotransferase inhibitor. Specifically, this study examined GABA levels in both the POC (Parietal-Occipital Cortex) a grey matter rich region thought to be associated with epilepsy, and which was previously studied for vigabatrin, and the SMA (Supplementary Motor Area), which is thought to be associated with Tourette’s Disorder. The maximum brain GABA increases, in both brain regions, ranged from about 150% to over 200% of baseline levels, as measured by magnetic resonance spectroscopy (MRS).
Patrick J. McEnany, Catalyst’s Chief Executive Officer stated, “These data are promising, because they provide evidence that CPP-115 significantly raises brain GABA, a mechanism known to effectively treat epilepsy, infantile spams, and potentially Tourette’s disorder. CPP-115 was also found to be even more potent than anticipated, which may indicate that a lower dose of CPP-115 has the potential to effectively treat these diseases. A low drug dose may reduce or eliminate the risk of visual field defects, a side effect associated with vigabatrin.”
Steven Miller, Ph.D., Catalyst’s Chief Operating Officer and Chief Scientific Officer stated, “We expect to receive the full results of this study early next year, including additional data from laboratory safety tests, pharmacokinetic modeling, and brain levels of CPP-115 as determined from MRS data of the patients in this study. Once we obtain the full results of this study, we intend to determine the steps required to make CPP-115 “Phase 2 ready,” including a Phase 1 dose ranging study evaluating CPP-115 at lower doses.”
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, recently completed testing in a global, multi-center, double-blinded randomized pivotal Phase 3 trial resulting in positive top-line data on both co-primary endpoints. Firdapse 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 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: 19
CORAL GABLES, FL, USA I December 16, 2015 I Catalyst Pharmaceuticals, Inc.(Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced top line results from its Phase 1(b) double-blind, placebo controlled safety and tolerance study of CPP-115 in normal healthy volunteers. The results showed significant increases in brain levels of the surrogate marker for potential efficacy, gamma-aminobutyric acid (GABA). The main adverse effect of prolonged elevated brain GABA, somnolence, was also observed.
While the primary objective of this study was to obtain safety and tolerance data for CPP-115 administered over 14 days, brain GABA levels were measured as a surrogate marker of potential efficacy, since CPP-115 is a second generation GABA aminotransferase inhibitor. Specifically, this study examined GABA levels in both the POC (Parietal-Occipital Cortex) a grey matter rich region thought to be associated with epilepsy, and which was previously studied for vigabatrin, and the SMA (Supplementary Motor Area), which is thought to be associated with Tourette’s Disorder. The maximum brain GABA increases, in both brain regions, ranged from about 150% to over 200% of baseline levels, as measured by magnetic resonance spectroscopy (MRS).
Patrick J. McEnany, Catalyst’s Chief Executive Officer stated, “These data are promising, because they provide evidence that CPP-115 significantly raises brain GABA, a mechanism known to effectively treat epilepsy, infantile spams, and potentially Tourette’s disorder. CPP-115 was also found to be even more potent than anticipated, which may indicate that a lower dose of CPP-115 has the potential to effectively treat these diseases. A low drug dose may reduce or eliminate the risk of visual field defects, a side effect associated with vigabatrin.”
Steven Miller, Ph.D., Catalyst’s Chief Operating Officer and Chief Scientific Officer stated, “We expect to receive the full results of this study early next year, including additional data from laboratory safety tests, pharmacokinetic modeling, and brain levels of CPP-115 as determined from MRS data of the patients in this study. Once we obtain the full results of this study, we intend to determine the steps required to make CPP-115 “Phase 2 ready,” including a Phase 1 dose ranging study evaluating CPP-115 at lower doses.”
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, recently completed testing in a global, multi-center, double-blinded randomized pivotal Phase 3 trial resulting in positive top-line data on both co-primary endpoints. Firdapse 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 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: 19
