Tirasemtiv

Summary

Tirasemtiv (CHEMBL3039529) is a phase-3 clinical-stage small molecule; indicated across 4 conditions including amyotrophic lateral sclerosis and myasthenia gravis.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 4 conditions
• Clinical trials: 7
• Chemistry: 230.27 Da · C12H14N4O

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3039529
Name	Tirasemtiv
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	23729157
Molecular formula	C12H14N4O
Molecular weight	230.27
InChIKey	RSQGZEAXODVTOL-UHFFFAOYSA-N

SMILES: CCC(CC)N1C2=NC(=CN=C2NC1=O)C#C

IUPAC name: 5-ethynyl-3-pentan-3-yl-1H-imidazo[4,5-b]pyrazin-2-one

Also known as: CK-2017357, CK2017357, Tirasemtiv, TIRASEMTIV

Patent coverage: 58 distinct patent families (197 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 158 (80%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Cytochrome P450 1A2.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

4 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
amyotrophic lateral sclerosis	3	MONDO:0004976	MONDO:0004976
myasthenia gravis	2	MONDO:0009688	EFO:0004991
intermittent vascular claudication	2	MONDO:0005295	EFO:0003876
peripheral arterial disease	2	MONDO:0005386	EFO:0004265

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
PHASE2	5
PHASE3	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02496767	PHASE3	COMPLETED	Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year
NCT02936635	PHASE3	COMPLETED	A Study for Patients Who Completed VITALITY-ALS (CY 4031)
NCT01089010	PHASE2	COMPLETED	A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
NCT01131013	PHASE2	COMPLETED	A Study of CK-2017357 in Patients With Peripheral Artery Disease and Symptomatic Claudication
NCT01268280	PHASE2	TERMINATED	Pharmacodynamic Study of CK-2017357 in Patients With Generalized Myasthenia Gravis
NCT01486849	PHASE2	COMPLETED	Dose Titration Study to Test Safety and Effects of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
NCT01709149	PHASE2	COMPLETED	Study of Safety, Tolerability & Efficacy of CK-2017357 in Amyotrophic Lateral Sclerosis (ALS)

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).