Scienza e Tecnologia
Cytokinetics Joins Global Movement to Raise Awareness for Rare Diseases
Cytokinetics, Incorporated (Nasdaq: CYTK) announced today that it is joining forces on February 28 with patients and health care advocates in the United States and globally to raise awareness for Rare Disease Day . Rare Disease Day is dedicated to elevating public understanding of rare diseases and calling attention to the special challenges faced by patients with rare diseases and the support community around them.
"Cytokinetics is proud to stand alongside patients and caregivers representing the rare disease community," stated Robert I. Blum, Cytokinetics' President & Chief Executive Officer. "We applaud the tireless work of NORD and EURORDIS, two outstanding organizations that perform extraordinary work every day in support of education, advocacy, research and the care of patients suffering from rare diseases. Cytokinetics is dedicated to the discovery and development of novel therapeutics that modulate muscle function for the treatment of severe illnesses and conditions, including rare neuromuscular diseases. "
Rare Disease Day, which takes place every year on the last day of February, was established in Europe in 2008 by the European Organisation for Rare Diseases (EURORDIS), the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. In the United States, Rare Disease Day is sponsored by the National Organization for Rare Disorders (NORD), a leading independent, non-profit organization committed to the identification, treatment, and cure of rare diseases. According to EURORDIS, a disease or disorder is defined as rare in Europe when it affects less than 1 in 2000. Rare diseases may affect 30 million European Union citizens. According to the National Institutes of Health (NIH), a disease is rare if it affects fewer than 200,000 people. Nearly 1 in 10 Americans live with a rare disease-affecting 30 million people - and two-thirds of these patients are children. There are more than 7,000 rare diseases and only approximately 450 FDA-approved medical treatments. For more information about Rare Disease Day in the U.S., go to www.rarediseaseday.us. For information about global activities, go to www.rarediseaseday.org. To search for information about rare diseases, visit NORD's website, www.rarediseases.org.
Cytokinetics is developing two drug candidates for the potential treatment of rare diseases. The company is developing , a fast skeletal troponin muscle activator, as a potential treatment for patients with amyotrophic lateral sclerosis (ALS). In addition, the company is collaborating with Astellas Pharma Inc. to develop CK-2127107, a structurally distinct fast skeletal troponin muscle activator, as a potential treatment for patients with spinal muscular atrophy (SMA).
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that afflicts approximately 25,000 people in the United States and a comparable number of patients in Europe. Approximately 5,600 new cases of ALS are diagnosed each year in the United States. The average life expectancy of an ALS patient is approximately three to five years after diagnosis and only 10% of patients survive for more than 10 years. Death is usually due to respiratory failure because of diminished strength in the skeletal muscles responsible for breathing. Few treatment options exist for patients with ALS, resulting in a high unmet need for new therapeutic options to address the symptoms and modify disease progression.
Spinal Muscular Atrophy (SMA) is a severe neuromuscular disease that occurs in 1 in every 6000 to 10,000 live births each year and is one of the most common fatal genetic disorders. Spinal muscular atrophy manifests in various degrees of severity as progressive muscle weakness resulting in respiratory and mobility impairment. There are four types of SMA based on time of initial onset of muscle weakness and related symptoms: Type I (Infantile), Type II (Intermediate), Type III (Juvenile) and Type IV (Adult Onset). Life expectancy and disease severity varies from Type I patients, who have the worst prognosis and a life expectancy of generally less than 2 years from birth to Type IV patients, who have a normal life span but with gradual weakness in proximal muscles of the extremities resulting in mobility issues. Few treatment options exist for these patients, resulting in a high unmet need for new therapeutic options to address symptoms and modify disease progression.
Cytokinetics is a clinical-stage biopharmaceutical company focused on the discovery and development of novel small molecule therapeutics that modulate muscle function for the potential treatment of serious diseases and medical conditions. Cytokinetics is developing , a fast skeletal muscle activator, as a potential treatment for amyotrophic lateral sclerosis (ALS). has been granted orphan drug designation and fast track status by the U.S. Food and Drug Administration and orphan medicinal product designation by the European Medicines Agency for the potential treatment of ALS. Cytokinetics is collaborating with Amgen Inc. to develop , a cardiac muscle activator, for the potential treatment of heart failure. Cytokinetics is collaborating with Astellas Pharma Inc. to develop CK-2127107, a skeletal muscle activator, for the potential treatment of spinal muscular atrophy. Amgen holds an exclusive license worldwide to develop and commercialize and Astellas holds an exclusive license worldwide to develop and commercialize CK-2127107. Both licenses are subject to Cytokinetics' specified development and commercialization participation rights. All of these drug candidates have arisen from Cytokinetics' muscle biology focused research activities and are directed towards the cytoskeleton. The cytoskeleton is a complex biological infrastructure that plays a fundamental role within every human cell. Additional information about Cytokinetics can be obtained at http://www.cytokinetics.com/ .
Contact:
Joanna L. Goldstein
Cytokinetics
Manager, Investor Relations & Corporate Communications
(650) 624-3000
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