OREANDA-NEWS. Cytokinetics, Inc. (Nasdaq:CYTK) today announced the completion of patient enrollment in VITALITY-ALS (Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices after Treatment for a Year in ALS), an international Phase 3 clinical trial of tirasemtiv in patients with ALS. VITALITY-ALS is designed to assess the effects of tirasemtiv versus placebo on slow vital capacity (SVC) and other measures of skeletal muscle strength in patients with ALS. VITALITY-ALS enrolled more than 700 patients in 81 centers in 11 countries. Results from VITALITY-ALS are expected in the second half of 2017. The company also announced that enrollment in the open-label extension to VITALITY-ALS is expected to begin in the fourth quarter of this year. 

“Completion of enrollment in VITALITY-ALS marks a significant milestone for Cytokinetics as well as the ALS community as we take another step toward advancing what could be the first potential new medicine for people with ALS in more than 20 years,” said Fady I. Malik, MD, PhD, Cytokinetics’ Executive Vice President, Research & Development. “We would like to express our thanks to the dedicated investigators and clinical site coordinators who are conducting the trial and also patients with ALS and their caregivers who are participating. We look forward to reporting results in the second half of 2017.”

About VITALITY-ALS

VITALITY-ALS is a multi-national, randomized, double-blind, placebo-controlled trial in patients with possible, probable or definite ALS, diagnosed within 24 months, and with percent predicted SVC at baseline ? 70 percent. The primary endpoint of the trial will assess change from baseline in SVC, to be assessed after 24 weeks of double-blind, placebo-controlled treatment. Secondary endpoints, to be assessed at 48 weeks, include time to decline from baseline in percent predicted SVC by ? 20 percentage points or the onset of respiratory insufficiency or death; time to decline from baseline in percent predicted SVC to ? 50 percent predicted or the onset of respiratory insufficiency or death; time to first occurrence of any use of assisted ventilation or death; time to decline in any of the three respiratory domains of the ALSFRS-R or death; and change in the Mega-Score of muscle strength. Patients enrolled in VITALITY-ALS receive two-weeks of open-label treatment with tirasemtiv administered at 250 mg/day. Patients are then randomized into a double-blind treatment phase to placebo or one of three target tirasemtiv dose levels (250 mg/day, 375 mg/day, 500 mg/day) in a 3:2:2:2 ratio. After 48 weeks of randomized, double-blind, placebo-controlled treatment, patients who received tirasemtiv during those 48 weeks of double-blind treatment will be randomized to continue the tirasemtiv dose at which they completed the 48 weeks of double-blind treatment or to placebo for a four-week double-blind, tirasemtiv withdrawal phase.  Patients who received placebo during the 48 weeks of double-blind treatment will continue to receive placebo during the double-blind, tirasemtiv withdrawal phase. Following their participation in VITALITY-ALS, patients are eligible to participate in an open-label extension study of tirasemtiv. 

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 the subject of a Phase 3 clinical trial program 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 develop omecamtiv 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.