Acoramidis

Summary

Acoramidis (CHEMBL3940890) is an approved small molecule (ATC C01EB25) targeting TTR; indicated across 3 conditions including familial amyloid neuropathy and cardiovascular disorder.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: C01EB25
• Targets: 1 (TTR)
• Indications: 3 conditions
• Clinical trials: 13
• Chemistry: 292.3 Da · C15H17FN2O3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3940890
Name	Acoramidis
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	71464713
ATC	C01EB25
Molecular formula	C15H17FN2O3
Molecular weight	292.3
InChIKey	WBFUHHBPNXWNCC-UHFFFAOYSA-N

SMILES: CC1=C(C(=NN1)C)CCCOC2=C(C=CC(=C2)C(=O)O)F

IUPAC name: 3-[3-(3,5-dimethyl-1H-pyrazol-4-yl)propoxy]-4-fluorobenzoic acid

Also known as: Acoramidis, AG-10, AG10, ACORAMIDIS

Parent form; salt/anhydrous children: CHEMBL4650226

Patent coverage: 307 distinct patent families (576 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 505 (88%) 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

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
TTR	transthyretin	Binding	8.32	0%	P02766

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Transthyretin, Albumin.

Bioactivity

ChEMBL activities: 6 potent at pChembl ≥ 5 of 6 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
TTR	8.32	Kd	4.8	nM	CHEMBL_ACT_16761938
TTR	8.32	Kd	4.8	nM	CHEMBL_ACT_18557073
TTR	8.14	Kd	7.3	nM	CHEMBL_ACT_24869652
TTR	6.8	IC50	160	nM	CHEMBL_ACT_24355277
TTR	6.5	Kd	314	nM	CHEMBL_ACT_16761937
ALB	5.1	Kd	8000	nM	CHEMBL_ACT_18557081

Target pathways

Aggregated over 1 target gene(s): TTR.

Top Reactome pathways

6 total, by targets touching each:

Pathway	Targets	Genes
The canonical retinoid cycle in rods (twilight vision)	1	TTR
Non-integrin membrane-ECM interactions	1	TTR
Neutrophil degranulation	1	TTR
Retinoid metabolism and transport	1	TTR
Amyloid fiber formation	1	TTR
Defective visual phototransduction due to STRA6 loss of function	1	TTR

Dominant GO biological processes

GO term	Targets
retinoid metabolic process	1
negative regulation of glomerular filtration	1
purine nucleobase metabolic process	1
phototransduction, visible light	1
signal transduction	1

Indications & clinical

Indications

3 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
familial amyloid neuropathy	3	MONDO:0007100	EFO:0004129
cardiovascular disorder	3	MONDO:0004995	EFO:0000319
amyloidosis	1	MONDO:0019065	EFO:1001875

Clinical trials

Total trials: 13.

Phase distribution

Phase	Trials
PHASE3	6
PHASE1	3
PHASE4	2
PHASE2	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07298044	PHASE4	RECRUITING	A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to Acoramidis
NCT07306949	PHASE4	RECRUITING	Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers
NCT04988386	PHASE3	ACTIVE_NOT_RECRUITING	Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants
NCT06563895	PHASE3	RECRUITING	Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
NCT07116473	PHASE3	NOT_YET_RECRUITING	To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)
NCT03860935	PHASE3	COMPLETED	Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy
NCT04418024	PHASE3	WITHDRAWN	Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
NCT04882735	PHASE3	WITHDRAWN	Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)
NCT03536767	PHASE2	ACTIVE_NOT_RECRUITING	Open-Label Study of AG10 in Patients with Cardiomyopathy
NCT03458130	PHASE2	COMPLETED	Study of AG10 in Amyloid Cardiomyopathy
NCT03294707	PHASE1	COMPLETED	Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10
NCT04769479	PHASE1	COMPLETED	A Single Dose Study to Evaluate the Pharmacokinetics of Acoramidis Modified Release Formulations in Healthy Subjects
NCT04958135	PHASE1	COMPLETED	Study to Assess the Effect of Food on a Single Dose of Acoramidis in Healthy Adult Participants

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.

37 molecules share ≥1 primary target. Top 37 by shared-target count:

Molecule	Source	Status	Shared targets
TAFAMIDIS	ChEMBL + PubChem	Phase 4 (approved)	TTR
AMLEXANOX	ChEMBL	Phase 4 (approved)	TTR
BENZBROMARONE	ChEMBL	Phase 4 (approved)	TTR
BENZIODARONE	ChEMBL	Phase 4 (approved)	TTR
BITHIONOL	ChEMBL	Phase 4 (approved)	TTR
DASATINIB	ChEMBL	Phase 4 (approved)	TTR
DEXTROTHYROXINE	ChEMBL	Phase 4 (approved)	TTR
DICLOFENAC	ChEMBL	Phase 4 (approved)	TTR
DIFLUNISAL	ChEMBL	Phase 4 (approved)	TTR
GEMFIBROZIL	ChEMBL	Phase 4 (approved)	TTR
LEVOTHYROXINE	ChEMBL	Phase 4 (approved)	TTR
MECLOFENAMIC ACID	ChEMBL	Phase 4 (approved)	TTR
TOLCAPONE	ChEMBL	Phase 4 (approved)	TTR
TRICLABENDAZOLE	ChEMBL	Phase 4 (approved)	TTR
TRICLOSAN	ChEMBL	Phase 4 (approved)	TTR
CAFFEIC ACID	ChEMBL	Phase 3	TTR
DIACEREIN	ChEMBL	Phase 3	TTR
EPIGALOCATECHIN GALLATE	ChEMBL	Phase 3	TTR
RESVERATROL	ChEMBL	Phase 3	TTR
ACECLOFENAC	ChEMBL	Phase 2	TTR
DAIDZEIN	ChEMBL	Phase 2	TTR
FLUFENAMIC ACID	ChEMBL	Phase 2	TTR
GENISTEIN	ChEMBL	Phase 2	TTR
LUTEOLIN	ChEMBL	Phase 2	TTR
NIFLUMIC ACID	ChEMBL	Phase 2	TTR
PTEROSTILBENE	ChEMBL	Phase 2	TTR
TOLFENAMIC ACID	ChEMBL	Phase 2	TTR
XANTHOHUMOL	ChEMBL	Phase 2	TTR
albuterol	PubChem	Approved	TTR
Amiodarone	PubChem	Approved	TTR
Bromhexine	PubChem	Approved	TTR
Capsaicin	PubChem	Approved	TTR
Carbinoxamine	PubChem	Approved	TTR
Chlorogenic Acid	PubChem	Approved	TTR
Crizotinib	PubChem	Approved	TTR
Nitisinone	PubChem	Approved	TTR
Tafamidis Meglumine	PubChem	Approved	TTR