Discovery of Tirasemtiv, the First Direct Fast Skeletal Muscle Troponin Activator

ACS Med Chem Lett. 2018 Feb 13;9(4):354-358. doi: 10.1021/acsmedchemlett.7b00546. eCollection 2018 Apr 12.

Abstract

The identification and optimization of the first activators of fast skeletal muscle are reported. Compound 1 was identified from high-throughput screening (HTS) and subsequently found to improve muscle function via interaction with the troponin complex. Optimization of 1 for potency, metabolic stability, and physical properties led to the discovery of tirasemtiv (25), which has been extensively characterized in clinical trials for the treatment of amyotrophic lateral sclerosis.