Emixustat hydrochloride

• CAT Number: I000533
• CAS Number: 1141934-97-5
• Molecular Formula: C16H26ClNO2
• Molecular Weight: 299.839
• Purity: ≥95%
• Synonyms: ACU-4429; ACU 4429; ACU4429; Emixustat; Emixustat HCl; Emixustat hydrochloride.;(1R)-3-Amino-1-(3-(cyclohexylmethoxy)phenyl)propan-1-ol hydrochloride
• Target: visual cycle modulator
• Solubility: Soluble in DMSO, not in water
• Storage: 0 - 4°C for short term or -20 °C for long term
• Related CAS: 1141777-14-1 (free base).
• InChI: InChI=1S/C16H25NO2.ClH/c17-10-9-16(18)14-7-4-8-15(11-14)19-12-13-5-2-1-3-6-13;/h4,7-8,11,13,16,18H,1-3,5-6,9-10,12,17H2;1H/t16-;/m1./s1
• InChIKey: BPZWRYOUJMDQSY-PKLMIRHRSA-N
• SMILES: O[C@@H](C1=CC=CC(OCC2CCCCC2)=C1)CCN.[H]Cl

Emixustat hydrochloride (Cat No.: I000533) is an orally active inhibitor of retinal pigment epithelium (RPE) 65 isomerase, a key enzyme in the visual cycle. It reduces the production of toxic retinoid byproducts, making it a promising therapeutic candidate for retinal diseases such as Stargardt disease and age-related macular degeneration (AMD). By modulating retinoid metabolism, Emixustat helps protect photoreceptor cells from degeneration. Its high specificity and bioavailability ensure reliable outcomes in ophthalmic research, supporting the development of novel treatments for retinal disorders.

Reference

1: Dugel PU, Novack RL, Csaky KG, Richmond PP, Birch DG, Kubota R. Phase ii,
randomized, placebo-controlled, 90-day study of emixustat hydrochloride in
geographic atrophy associated with dry age-related macular degeneration. Retina.
2015 Jun;35(6):1173-83. doi: 10.1097/IAE.0000000000000606. PubMed PMID: 25932553;
PubMed Central PMCID: PMC4452434.

2: Kubota R, Al-Fayoumi S, Mallikaarjun S, Patil S, Bavik C, Chandler JW. Phase
1, dose-ranging study of emixustat hydrochloride (ACU-4429), a novel visual cycle
modulator, in healthy volunteers. Retina. 2014 Mar;34(3):603-9. doi:
10.1097/01.iae.0000434565.80060.f8. PubMed PMID: 24056528.