Last month, Cytokinetics announced the failure of its candidate therapy for ALS, tirasemtiv, to meet primary endpoints in the BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv in ALS) Phase IIb clinical trial (see April 2014 Drug News). Tirasemtiv enhances troponin function in skeletal muscles, leading to increased muscle force in response to input from motor neurons. Although the drug did not improve motor function based on the ALS functional rating scare (ALS-FRS), tirasemtiv appeared to have beneficial effects on respiratory muscle function. Cytokinetics is now addressing some questions raised by the outcome of BENEFIT-ALS in three publications describing preclinical and clinical studies conducted in support of the tirasemtiv as a candidate therapy for ALS. The papers appear in PLOS ONE, Muscle & Nerve, and the American Journal of Respiratory and Critical Care Medicine.
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