Bamocaftor

Summary

Bamocaftor (CHEMBL4297849) is a phase-3 clinical-stage small molecule targeting CFTR; indicated across 1 condition including cystic fibrosis.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (CFTR)
• Indications: 1 condition
• Clinical trials: 6
• Chemistry: 591.6 Da · C28H32F3N5O4S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297849
Name	Bamocaftor
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	134393443
Molecular formula	C28H32F3N5O4S
Molecular weight	591.6
InChIKey	IGEOJNMYRZUKIK-IBGZPJMESA-N

SMILES: C[C@H]1CC(N(C1)C2=C(C=CC(=N2)N3C=CC(=N3)OCCC4(CC4)C(F)(F)F)C(=O)NS(=O)(=O)C5=CC=CC=C5)(C)C

IUPAC name: N-(benzenesulfonyl)-6-[3-[2-[1-(trifluoromethyl)cyclopropyl]ethoxy]pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide

Also known as: Bamocaftor, Vx-659, VX-659, VX659, BAMOCAFTOR

Parent form; salt/anhydrous children: CHEMBL4298159

Patent coverage: 95 distinct patent families (272 SureChEMBL compound mentions), from 1 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

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
CFTR	CFTR	Binding	7.52	0.1%	P13569

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Cystic fibrosis transmembrane conductance regulator.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 2 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CFTR	7.52	EC50	30	nM	CHEMBL_ACT_28548959
CFTR	7.52	EC50	30	nM	CHEMBL_ACT_29226293

Target pathways

Aggregated over 1 target gene(s): CFTR.

Top Reactome pathways

11 total, by targets touching each:

Pathway	Targets	Genes
ABC-family protein mediated transport	1	CFTR
RHO GTPases regulate CFTR trafficking	1	CFTR
Defective CFTR causes cystic fibrosis	1	CFTR
Ub-specific processing proteases	1	CFTR
Cargo recognition for clathrin-mediated endocytosis	1	CFTR
Clathrin-mediated endocytosis	1	CFTR
RHOQ GTPase cycle	1	CFTR
Chaperone Mediated Autophagy	1	CFTR
Late endosomal microautophagy	1	CFTR
Aggrephagy	1	CFTR
Developmental Lineage of Pancreatic Ductal Cells	1	CFTR

Dominant GO biological processes

GO term	Targets
cholesterol biosynthetic process	1
water transport	1
bicarbonate transport	1
cholesterol transport	1
response to endoplasmic reticulum stress	1
transepithelial water transport	1
sperm capacitation	1
multicellular organismal-level water homeostasis	1
intracellular pH elevation	1
establishment of localization in cell	1
transmembrane transport	1
membrane hyperpolarization	1
positive regulation of enamel mineralization	1
cellular response to cAMP	1
amelogenesis	1

Indications & clinical

Indications

1 indication (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
cystic fibrosis	3	MONDO:0009061	MONDO:0009061

Clinical trials

Total trials: 6.

Phase distribution

Phase	Trials
PHASE3	4
PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03447249	PHASE3	COMPLETED	A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
NCT03447262	PHASE3	TERMINATED	A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy
NCT03460990	PHASE3	COMPLETED	A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
NCT03633526	PHASE3	TERMINATED	Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
NCT03224351	PHASE2	COMPLETED	A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis
NCT03029455	PHASE1	COMPLETED	A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis

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

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

14 molecules share ≥1 primary target. Top 14 by shared-target count:

Molecule	Source	Status	Shared targets
IVACAFTOR	ChEMBL + PubChem	Phase 4 (approved)	CFTR
ELEXACAFTOR	ChEMBL	Phase 4 (approved)	CFTR
GLYBURIDE	ChEMBL	Phase 4 (approved)	CFTR
LUMACAFTOR	ChEMBL	Phase 4 (approved)	CFTR
TEZACAFTOR	ChEMBL	Phase 4 (approved)	CFTR
QUERCETIN	ChEMBL	Phase 3	CFTR
RUTIN	ChEMBL	Phase 3	CFTR
GALICAFTOR	ChEMBL	Phase 2	CFTR
GENISTEIN	ChEMBL	Phase 2	CFTR
GLPG-2737	ChEMBL	Phase 2	CFTR
ICENTICAFTOR	ChEMBL	Phase 2	CFTR
NAVOCAFTOR	ChEMBL	Phase 2	CFTR
RISELCAFTOR	ChEMBL	Phase 2	CFTR
Tadalafil	PubChem	Approved	CFTR