Cytokinetics' ALS Drug Fails In Phase IIB Trial

Cytokinetics reported that its Phase IIB trial BENEFIT-ALS assessing investigational drug tirasemtiv in amyotrophic lateral sclerosis (ALS) failed to meet its primary efficacy endpoint.

BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv in ALS) did not meet its primary efficacy endpoint of mean change from baseline in the ALS Functional Rating Scale in its revised form (ALSFRS-R) on tirasemtiv versus placebo. The company also reported that secondary efficacy analyses focusing specifically on respiratory function and skeletal muscle function were inconclusive.

Robert I. Blum, Cytokinetics’ President and CEO, said, “Patients with ALS desperately need new therapeutic alternatives to slow the course of their disease and loss of function. We stand with the ALS community in our disappointment that BENEFIT-ALS did not achieve its primary efficacy endpoint. The results from BENEFIT-ALS are just now becoming available to our team at Cytokinetics and will be shared in more detail with the broader scientific and medical community focused to research in ALS in the next few days. Understanding these results will require significant further review. Once we have fully evaluated the data from BENEFIT-ALS, we expect to determine whether there is a potential development path forward for tirasemtiv for the potential treatment of ALS and what may be the appropriate next steps.”

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects an estimated 25,000 people in the U.S. alone. About 5,600 new ALS cases are diagnosed every year in the U.S. Average life expectancy of patients is approximately three to five years after diagnosis. Only 10 percent of patients with ALS survive for more than ten years.

Tirasemtiv is a novel skeletal muscle activator and the lead drug candidate of the company’s skeletal muscle contractility program. The drug increases the calcium sensitivity of the fast skeletal muscle troponin complex. Tirasemtiv increased skeletal muscle force in response to neuronal input and reduced muscle fatigue in early clinical trials and preclinical studies.

The company said it will present detailed results of the BENEFIT-ALS trial during the 66th Annual Meeting of the American Academy of Neurology (AAN) to be held in Philadelphia, PA this month.