Cytokinetics Joins Global Initiative to Recognize International Rare Disease Day
Cytokinetics (Nasdaq: CYTK) is participating in Rare Disease Day on February 28, 2023, alongside EURORDIS and NORD to raise awareness for over 300 million individuals affected by rare diseases. CEO Robert I. Blum emphasized the company's commitment to enhancing access to diagnosis, treatment, and care for these patients. Cytokinetics is developing aficamten, a cardiac myosin inhibitor for hypertrophic cardiomyopathy (HCM), currently in a Phase 3 trial, SEQUOIA-HCM. Additionally, reldesemtiv, aimed at treating amyotrophic lateral sclerosis (ALS), is under evaluation in COURAGE-ALS, also a Phase 3 trial. Both drug candidates have received Orphan Drug Designation from the FDA.
- Cytokinetics is actively engaged in raising awareness for rare diseases, aligning with EURORDIS and NORD.
- Aficamten is in Phase 3 clinical trial SEQUOIA-HCM, focusing on HCM treatment.
- Reldesemtiv is also in Phase 3 clinical trial COURAGE-ALS, targeting ALS.
- Both candidates have received Orphan Drug Designation from the FDA.
- None.
SOUTH SAN FRANCISCO, Calif., Feb. 22, 2023 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that it is joining the European Organisation for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD) to recognize Rare Disease Day® on February 28, 2023. Rare Disease Day® is an international campaign elevating the awareness and public understanding of rare diseases. The initiative spotlights the more than 300 million people worldwide living with a rare disease, and the awareness efforts focused on bringing them more equitable access to diagnosis, treatment, care and social opportunity.
“We take great pride in joining forces with EURORDIS, NORD and millions worldwide for Rare Disease Day, an opportunity to recognize and honor the courage and resilience of those living with rare diseases, while we advocate for improved access to diagnosis, treatment and care,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “As we continue to work toward making a meaningful difference in the lives of those living with HCM and ALS, we remain committed to partnering with patients, caregivers and advocacy organizations to elevate their voices.”
Cytokinetics is developing aficamten, a next-generation cardiac myosin inhibitor, for the potential treatment of hypertrophic cardiomyopathy (HCM). Cytokinetics is currently conducting SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), a Phase 3 clinical trial of aficamten in patients with symptomatic obstructive HCM. Aficamten is also being evaluated in non-obstructive HCM in Cohort 4 of the Phase 2 clinical trial, REDWOOD-HCM (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM). Aficamten has been granted Orphan Drug Designation, as well as Breakthrough Therapy Designation for the treatment of symptomatic HCM by the U.S. Food and Drug Administration (FDA).
Cytokinetics is also developing reldesemtiv, a fast skeletal muscle troponin activator, for the potential treatment of amyotrophic lateral sclerosis (ALS), currently the subject of COURAGE-ALS (Clinical Outcomes Using Reldesemtiv on ALSFRS-R in a Global Evaluation in ALS), a Phase 3 clinical trial in patients with ALS. Reldesemtiv has been granted Orphan Drug Designation for the treatment of ALS by the FDA and by the European Medicines Agency (EMA).
About Rare Disease Day
Rare Disease Day, which takes place every year on the last day in February, was established in Europe in 2008 by the European Organisation for Rare Diseases (EURORDIS) 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 the National Institutes of Health (NIH), in the US, a rare disease is defined as one that affects fewer than 200,000 people. With over 6,000 rare diseases, 25 million Americans are living with a rare disease, but only 5 percent of these diseases have a treatment.
About Hypertrophic Cardiomyopathy
Hypertrophic cardiomyopathy (HCM) is a disease in which the heart muscle (myocardium) becomes abnormally thick (hypertrophied). The thickening of cardiac muscle leads to the inside of the left ventricle becoming smaller and stiffer, and thus the ventricle becomes less able to relax and fill with blood. This ultimately limits the heart’s pumping function, resulting in symptoms including chest pain, dizziness, shortness of breath, or fainting during physical activity. A subset of patients with HCM are at high risk of progressive disease which can lead to atrial fibrillation, stroke and death due to arrhythmias.
About Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that afflicts approximately 27,000 people in the United States and a comparable number of patients in Europe. Approximately 6,300 new cases of ALS are diagnosed each year in the United States. The average life expectancy of a person with ALS is approximately three to five years after diagnosis and only approximately 10 percent of people with ALS 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 these patients, resulting in a high unmet need for new therapies to address functional deficits and disease progression.
About Cytokinetics
Cytokinetics is a late-stage biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators and next-in-class muscle inhibitors as potential treatments for debilitating diseases in which muscle performance is compromised. As a leader in muscle biology and the mechanics of muscle performance, the company is developing small molecule drug candidates specifically engineered to impact muscle function and contractility. Cytokinetics is readying for the potential commercialization of omecamtiv mecarbil, its cardiac muscle activator, following positive results from GALACTIC-HF, a large, international Phase 3 clinical trial in patients with heart failure. Cytokinetics is also developing aficamten, a next-in-class cardiac myosin inhibitor, currently the subject of SEQUOIA-HCM, the Phase 3 clinical trial of aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Aficamten is also being evaluated in non-obstructive HCM in Cohort 4 of the Phase 2 clinical trial, REDWOOD-HCM. Cytokinetics is also developing reldesemtiv, an investigational fast skeletal muscle troponin activator, currently the subject of COURAGE-ALS, a Phase 3 clinical trial in patients with amyotrophic lateral sclerosis (ALS). In 2023, Cytokinetics is celebrating its 25-year history of pioneering innovation in muscle biology and related pharmacology focused to diseases of muscle dysfunction and conditions of muscle weakness.
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