CORAL GABLES, FL, USA I October 15, 2013 I Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX), a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases and disorders, announced today that, at a recently held meeting, the independent Data Monitoring Committee (DMC) overseeing the Company’s ongoing pivotal Phase III clinical trial in the United States and Europe evaluating Firdapse™ for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) recommended that the Company continue the trial as planned based on the committee’s review of safety and clinical data from the trial.
The DMC is a group of experts responsible for the independent review of accumulated, unblinded clinical safety and efficacy data obtained in the Company’s clinical trial, in order to safeguard the interests and safety of participants and future patients. The DMC considers study-specific data, as well as relevant background knowledge about the disease, test agent or patient population under study.
The Firdapse™ Phase III clinical trial is designed as a randomized, double-blind, placebo-controlled, discontinuation trial enrolling 36 patients diagnosed with LEMS at sites in the U.S. and Europe. To accomplish this, the Company, in addition to the 7 active sites at the time of acquisition, has initiated 12 additional sites and expects to initiate shortly an additional 6 sites in the U.S., Europe, Canada and South America. The Company expects to complete enrollment in the trial about the end of the fourth quarter of 2013 and to announce top line data from the trial during the second quarter of 2014.
About LEMS
Lambert-Eaton Myasthenic Syndrome, or LEMS, is a rare autoimmune disorder characterized by muscle weakness of the limbs. The disease is caused by an autoimmune reaction where antibodies are formed against the connection between nerves and the muscles they supply. Often, LEMS is associated with an underlying malignancy, most commonly small-cell lung cancer, and in some individuals, LEMS is the first symptom of such malignancy. LEMS generally affects the extremities, especially the legs. As the disease most affects the parts of limbs closest to the trunk, difficulties with climbing stairs or rising from a sitting position are commonly noted. Physical exercise and high temperatures tend to worsen the symptoms. Other symptoms occasionally seen include weakness of the muscles of the mouth, throat, and eyes. Individuals affected with LEMS also may have a disruption of the autonomic nervous system, including dry mouth, constipation, blurred vision, impaired sweating, and/or hypotension.
About Catalyst Pharmaceutical Partners
Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette Syndrome. Catalyst’s lead candidate, Firdapse™ for the treatment of LEMS, is currently undergoing testing in a global, multi-center, pivotal phase III trial and recently received “Breakthrough Therapy Designation” from the U.S. Food and Drug Administration (FDA). Catalyst is also developing a potentially safer and more potent vigabatrin analog (designated CPP-115) to treat infantile spasms, and epilepsy, as well as other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette Syndrome.
SOURCE: Catalyst Pharmaceutical Partners
Post Views: 148
CORAL GABLES, FL, USA I October 15, 2013 I Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX), a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases and disorders, announced today that, at a recently held meeting, the independent Data Monitoring Committee (DMC) overseeing the Company’s ongoing pivotal Phase III clinical trial in the United States and Europe evaluating Firdapse™ for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) recommended that the Company continue the trial as planned based on the committee’s review of safety and clinical data from the trial.
The DMC is a group of experts responsible for the independent review of accumulated, unblinded clinical safety and efficacy data obtained in the Company’s clinical trial, in order to safeguard the interests and safety of participants and future patients. The DMC considers study-specific data, as well as relevant background knowledge about the disease, test agent or patient population under study.
The Firdapse™ Phase III clinical trial is designed as a randomized, double-blind, placebo-controlled, discontinuation trial enrolling 36 patients diagnosed with LEMS at sites in the U.S. and Europe. To accomplish this, the Company, in addition to the 7 active sites at the time of acquisition, has initiated 12 additional sites and expects to initiate shortly an additional 6 sites in the U.S., Europe, Canada and South America. The Company expects to complete enrollment in the trial about the end of the fourth quarter of 2013 and to announce top line data from the trial during the second quarter of 2014.
About LEMS
Lambert-Eaton Myasthenic Syndrome, or LEMS, is a rare autoimmune disorder characterized by muscle weakness of the limbs. The disease is caused by an autoimmune reaction where antibodies are formed against the connection between nerves and the muscles they supply. Often, LEMS is associated with an underlying malignancy, most commonly small-cell lung cancer, and in some individuals, LEMS is the first symptom of such malignancy. LEMS generally affects the extremities, especially the legs. As the disease most affects the parts of limbs closest to the trunk, difficulties with climbing stairs or rising from a sitting position are commonly noted. Physical exercise and high temperatures tend to worsen the symptoms. Other symptoms occasionally seen include weakness of the muscles of the mouth, throat, and eyes. Individuals affected with LEMS also may have a disruption of the autonomic nervous system, including dry mouth, constipation, blurred vision, impaired sweating, and/or hypotension.
About Catalyst Pharmaceutical Partners
Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette Syndrome. Catalyst’s lead candidate, Firdapse™ for the treatment of LEMS, is currently undergoing testing in a global, multi-center, pivotal phase III trial and recently received “Breakthrough Therapy Designation” from the U.S. Food and Drug Administration (FDA). Catalyst is also developing a potentially safer and more potent vigabatrin analog (designated CPP-115) to treat infantile spasms, and epilepsy, as well as other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette Syndrome.
SOURCE: Catalyst Pharmaceutical Partners
Post Views: 148
