Aficamten

Summary

Aficamten (CHEMBL4847050) is a phase-3 clinical-stage small molecule; indicated across 2 conditions including hypertrophic cardiomyopathy and cardiomyopathy.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 2 conditions
• Clinical trials: 9
• Chemistry: 337.4 Da · C18H19N5O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4847050
Name	Aficamten
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	139331495
Molecular formula	C18H19N5O2
Molecular weight	337.4
InChIKey	IOVAZWDIRCRMTM-OAHLLOKOSA-N

SMILES: CCC1=NC(=NO1)C2=CC3=C(C=C2)[C@@H](CC3)NC(=O)C4=CN(N=C4)C

IUPAC name: N-[(1R)-5-(5-ethyl-1,2,4-oxadiazol-3-yl)-2,3-dihydro-1H-inden-1-yl]-1-methylpyrazole-4-carboxamide

Also known as: Aficamten, CK-3773274, AFICAMTEN

Patent coverage: 37 distinct patent families (122 SureChEMBL compound mentions), from 2 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

No target linkage available.

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

2 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
hypertrophic cardiomyopathy	3	MONDO:0005045	EFO:0000538
cardiomyopathy	1	MONDO:0004994	EFO:0000318

Clinical trials

Total trials: 9.

Phase distribution

Phase	Trials
PHASE3	3
PHASE2/PHASE3	2
PHASE1	2
PHASE4	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07600177	PHASE4	RECRUITING	Mavacamten to Aficamten Transition in Patients With Obstructive Hypertrophic Cardiomyopathy
NCT04848506	PHASE2/PHASE3	ENROLLING_BY_INVITATION	Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults With HCM
NCT06081894	PHASE3	ACTIVE_NOT_RECRUITING	Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic nHCM
NCT06412666	PHASE2/PHASE3	RECRUITING	A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
NCT05186818	PHASE3	COMPLETED	Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM
NCT06116968	PHASE3	COMPLETED	An Open-Label Study of Aficamten for Chinese Patients With Symptomatic oHCM
NCT04219826	PHASE2	COMPLETED	Dose-finding Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CK-3773274 in Adults With Hypertrophic Cardiomyopathy
NCT04783766	PHASE1	COMPLETED	Safety, Tolerability and Pharmacokinetics Study of CK-3773274
NCT05924815	PHASE1	COMPLETED	Study to Evaluate the Effect of Aficamten Administration on QT/QTc Interval

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

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