Cytokinetics, Inc. (NASDAQ:CYTK) today announced the first patient has been enrolled in VIGOR-ALS (Ventilatory Investigations in Global Open-Label Research in ALS), an open-label extension clinical trial designed to assess the long-term safety and tolerability of tirasemtiv, in patients with ALS who have completed their participation in VITALITY-ALS, the Phase 3 clinical trial designed to assess the effects of tirasemtivversus placebo on slow vital capacity (SVC) and other measures of respiratory and skeletal muscle function.
“People living with ALS desperately need new therapies and VIGOR-ALS reflects our commitment to the patients that participated in VITALITY-ALS and who contributed enormously to the development of a potential new medicine for patients with ALS,” said Fady I. Malik, MD, PhD, Cytokinetics’ Executive Vice President, Research & Development. “This trial will provide supplemental data on the effects of long-term use of tirasemtiv.”
About VIGOR-ALS
VIGOR-ALS is an open-label extension trial of tirasemtiv in patients with ALS who completed participation in VITALITY-ALS. The primary endpoint of the trial is the incidence of adverse events in the patient population. Secondary endpoints include the time to events such as death or the first use of assisted ventilation or respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation for ³22 hours per day for ³10 consecutive days); decline from baseline in percent predicted slow vital capacity (SVC), ALS functional rating scale-revised (ALSFRS-R); and the slope of change from baseline in percent predicted SVC and ALSFRS-R. Following enrollment, patients will begin dosing of tirasemtiv 125 mg twice daily (250 mg/day) for a period of 4 weeks and will titrate to their maximum tolerated dose (based on tolerability), to no higher than 250 mg twice daily (500 mg/day). Additional information on the trial can be found at clinicaltrials.gov.
About ALS
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that afflicts approximately 30,000 people in the United States and a comparable number of patients in Europe. Approximately 6,000 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 percent 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 these patients, resulting in a high unmet need for new therapies to address functional deficits and disease progression.
About Tirasemtiv
Tirasemtiv, a novel skeletal muscle activator, selectively activates the fast skeletal muscle troponin complex by increasing its sensitivity to calcium and, in preclinical studies and early clinical trials, demonstrated increases in skeletal muscle force in response to neuronal input and delays in the onset and reductions in the degree of muscle fatigue. Tirasemtiv has been studied in clinical trials that have enrolled over 1000 people internationally. Tirasemtiv is currently the subject of VITALITY-ALS, a Phase 3 clinical trial, designed to confirm and extend findings on measures of respiratory function and muscle strength from prior studies.
About Cytokinetics
Cytokinetics is a late-stage biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators as potential treatments for debilitating diseases in which muscle performance is compromised and/or declining. As a leader in muscle biology and the mechanics of muscle performance, the company is developing small molecule drug candidates specifically engineered to increase muscle function and contractility. Cytokinetics’ lead drug candidate is tirasemtiv, a fast skeletal muscle troponin activator, for the potential treatment of ALS. Tirasemtiv 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 retains the right to develop and commercialize tirasemtiv, subject to an option held by Astellas Pharma Inc. Cytokinetics is also collaborating with Astellas to develop CK-2127107, a fast skeletal muscle activator, for the potential treatment of spinal muscular atrophy, chronic obstructive pulmonary disease and ALS. Cytokinetics is collaborating with Amgen Inc. to developomecamtiv mecarbil, a novel cardiac muscle activator, for the potential treatment of heart failure. Amgen holds an exclusive license worldwide to develop and commercialize omecamtiv mecarbil 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. For additional information about Cytokinetics, visit http://www.cytokinetics.com/.
