Cure SMA Announces Grant to Cytokinetics for Preclinical Development of Tirasemtiv for Spinal Muscular Atrophy

Originally published on April 4, 2013.

Award to support testing of Tirasemtiv in mouse models of Spinal Muscular Atrophy.

Cytokinetics, Incorporated (Nasdaq: CYTK) and Cure SMA announced the award of a grant from Cure SMA to Cytokinetics to support preclinical research on muscle function in a mouse model of spinal muscular atrophy (SMA) to be conducted with the company’s fast skeletal muscle troponin activator, tirasemtiv. Financial details of the grant were not disclosed. 

Tirasemtiv, the lead drug candidate from Cytokinetics’ skeletal muscle contractility program, selectively activates the fast skeletal muscle troponin complex by increasing its sensitivity to calcium, thereby increasing skeletal muscle force in response to neuronal input and delaying the onset and reducing the degree of muscle fatigue. Cytokinetics is evaluating tirasemtiv as a potential treatment for amyotrophic lateral sclerosis (ALS) in BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv in ALS), an international Phase IIb clinical trial that is now enrolling patients.  Tirasemtiv (formerly CK-2017357) is currently being evaluated in BENEFIT-ALS, an international, double-blind, randomized, placebo-controlled, Phase IIb clinical trial designed to evaluate the safety, tolerability and potential efficacy of this novel drug candidate in patients with ALS.  BENEFIT-ALS is designed to enroll approximately 400 patients who will first complete one week of treatment with open-label tirasemtiv at 125 mg twice daily.

The objective of this new funded preclinical research is to examine whether tirasemtiv can improve muscle function in mouse models of SMA. Cytokinetics will examine the effects of tirasemtiv on leg and respiratory muscle function and the effects of tirasemtiv to reduce fatigue and improve muscle strength during exercise.

“There remains a significant unmet medical need for a novel therapy that can improve muscle function, including respiratory muscle function, in patients with SMA,” stated Jill Jarecki, Ph.D., Research Director of Cure SMA. “If tirasemtiv can improve muscle function in mouse models of SMA, it may also ameliorate muscle weakness in patients with SMA and thereby has the potential to improve quality of life for patients affected by this disease.”

“We are pleased to be the recipient of this grant funding from Cure SMA which will enable us to investigate the potential of tirasemtiv to address some of the unmet needs of this grievous disease,” stated Jeffrey Jasper, Ph.D., Cytokinetics’ Head of Pharmacology. “We look forward to working with Cure SMA on this important project which may inform Cytokinetics’ plans for future clinical development activities of tirasemtiv.” 

About Cytokinetics

Cytokinetics is a clinical-stage biopharmaceutical company focused on the discovery and development of novel small molecule therapeutics that modulate muscle function for the potential treatment of serious diseases and medical conditions. Cytokinetics’ lead drug candidate from its cardiac muscle contractility program, omecamtiv mecarbil, is in Phase II clinical development for the potential treatment of heart failure. Amgen Inc. holds an exclusive license worldwide (excluding Japan) to develop and commercialize omecamtiv mecarbil and related compounds, subject to Cytokinetics’ specified development and commercialization participation rights. Cytokinetics is independently developing tirasemtiv and CK-2127107, both fast skeletal muscle activators, as potential treatments for diseases and medical conditions associated with aging, muscle wasting or neuromuscular dysfunction. Tirasemtiv is currently the subject of a Phase II clinical trials program and 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 amyotrophic lateral sclerosis, a debilitating disease of neuromuscular impairment in which treatment with tirasemtiv produced potentially clinically relevant pharmacodynamic effects in Phase II trials. All of these drug candidates have arisen from Cytokinetics’ muscle biology focused research activities and are directed towards the cytoskeleton. The cytoskeleton is a complex biological infrastructure that plays a fundamental role within every human cell. Additional information about Cytokinetics can be obtained at

Funding for New Drug Discovery Programs

In late 2012, Cure SMA’s Translational Advisory Committee (TAC) met to evaluate research funding for new drug discovery projects for SMA. Cure SMA is planning to award three new drug discovery projects over the next few months. The grant to Cytokinetics is the second of four new grants awarded by the Cure SMA TAC. 

Cure SMA has been investing in and advancing drug research since 2000. The goal of drug discovery funding at Cure SMA is to build a large and diverse therapeutic pipeline to maximize the chances for success in finding a treatment for SMA. Cure SMA has been involved in funding half of all the ongoing SMA drug programs to date. New awards in the current round will focus on providing critical funding for early-exploratory projects assessing novel therapeutic approaches for SMA. The Cure SMA research funding philosophy is based on expert and independent oversight of research projects. This approach ensures that Cure SMA funds only the most promising research, and that funded projects are run in a professional and efficient manner under the guidance of world-class experts. Cure SMA committed $2.5 Million towards SMA Research in 2012.

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