Emixustat

Summary

Emixustat (CHEMBL2107821) is a phase-3 clinical-stage small molecule; indicated across 3 conditions including stargardt disease and age-related macular degeneration.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 3 conditions
• Clinical trials: 7
• Chemistry: 263.37 Da · C16H25NO2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2107821
Name	Emixustat
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	25221720
Molecular formula	C16H25NO2
Molecular weight	263.37
InChIKey	WJIGGYYSZBWCGC-MRXNPFEDSA-N

SMILES: C1CCC(CC1)COC2=CC=CC(=C2)[C@@H](CCN)O

IUPAC name: (1R)-3-amino-1-[3-(cyclohexylmethoxy)phenyl]propan-1-ol

Also known as: ACU-4429, Acu-4429, Emixustat, EMIXUSTAT

Parent form; salt/anhydrous children: CHEMBL2107829

Patent coverage: 85 distinct patent families (256 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 220 (86%) 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

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

3 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
Stargardt disease	3	MONDO:0019353	MONDO:0019353
age-related macular degeneration	2	MONDO:0005150	EFO:0001365

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 7.

Phase distribution

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

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01802866	PHASE2/PHASE3	COMPLETED	Safety and Efficacy Assessment Treatment Trials of Emixustat Hydrochloride
NCT03772665	PHASE3	COMPLETED	Safety and Efficacy of Emixustat in Stargardt Disease
NCT01002950	PHASE2	COMPLETED	Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ACU-4429 in Subjects With Geographic Atrophy
NCT03033108	PHASE2	COMPLETED	Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
NCT00703183	PHASE1	COMPLETED	Safety and Tolerability of ACU-4429
NCT00942240	PHASE1	COMPLETED	Safety and Tolerability of Repeat Doses of ACU-4429 in Healthy Subjects
NCT02130531	PHASE1	COMPLETED	Pharmacokinetic and Pharmacodynamic Study of Emixustat in Subjects With Geographic Atrophy Associated With Dry Age-Related Macular Degeneration

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).