Drug intelligence / Profile preview

tirasemtiv

Development stage
Phase 3
Lead developer
Cytokinetics
Modality
Small Molecules
Administration
Oral
01

Overview

Tirasemtiv is a small molecule investigational drug developed for the treatment of neuromuscular diseases such as amyotrophic lateral sclerosis (ALS), myasthenia gravis, and intermittent claudication. It acts as a fast skeletal muscle troponin activator, selectively binding to the fast skeletal muscle troponin complex and sensitizing it to calcium. This mechanism amplifies muscle response to nerve input by increasing force generation at submaximal levels of nerve stimulation, which may help counteract muscle weakness in conditions with impaired neural input. Tirasemtiv was developed by Cytokinetics and reached Phase 3 clinical trials in ALS but did not meet its primary efficacy endpoints and has since been discontinued for this indication[1][3][4][5][6].

Other names
tirasemtiv5-ethynyl-3-pentan-3-yl-1H-imidazo[4,5-b]pyrazin-2-one
02

Targets

TNNC2 (Troponin C type 2)

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