Cytokinetics Inc. announced that additional results from BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv in ALS) were presented during the Joint Congress of European Neurology at the International Congress Center in Istanbul, Turkey. The new data from BENEFIT-ALS were presented Sunday by Jeremy Shefner, professor and chair, Department of Neurology at the Upstate Medical University, State University of New York and the lead investigator for BENEFIT-ALS.
In BENEFIT-ALS, patients with amyotrophic lateral sclerosis (ALS) were randomized 1:1 to double-blind treatment with tirasemtiv or placebo for 12 weeks. The primary outcome measure, the ALS Functional Rating Scale in its revised form (ALSFRS-R), and secondary outcomes measures of respiratory performance and other measures of skeletal muscle function and fatigability were assessed after four, eight, and 12 weeks of double-blind treatment, and again at one and four weeks after the last dose of double-blind treatment. The results from double-blind treatment during BENEFIT-ALS were presented at the Annual Meeting of the American Academy of Neurology. Presented Sunday were outcome measures obtained at one and four weeks after the last dose of double-blind study medication.
“We now have evidence that differences in Slow Vital Capacity between tirasemtiv and placebo observed during double-blind treatment in BENEFIT-ALS were maintained up to four weeks after discontinuation of treatment,” stated Shefner. “These data suggest that tirasemtiv may have a durable impact on this measure of pulmonary function in patients with ALS which has been shown to predict disease progression in prior clinical trials. These effects, if persistent over longer periods of time, suggest mechanisms by which tirasemtiv could preserve critical functionality in patients with ALS.”
Differences detected during double-blind treatment in the percentage changes from baseline in Muscle Strength Mega-Score between tirasemtiv and placebo were not sustained during the follow-up period. No differences in Maximum Voluntary Ventilation and Hand Grip Fatigue were observed on tirasemtiv versus placebo during double-blind treatment nor during the period through 4 weeks after the last double-blind dose.
“These data suggest that tirasemtiv may have effects on the respiratory muscles involved in performing the Slow Vital Capacity assessment that extend beyond its immediate pharmacologic action,” said Andrew Wolff, Cytokinetics’ chief medical officer and senior vice president of clinical research and development. “The absence of a difference between tirasemtiv and placebo on muscle strength and other endpoints at four weeks after double-blind treatment is consistent with diminishing pharmacologic effects of tirasemtiv following its discontinuation. We are encouraged by these results and are continuing our analyses of data from BENEFIT-ALS to inform the potential further development of tirasemtiv in patients with ALS.”
Date: June 2, 2014
Source: Cytokinetics
