Global InformationEmixustat information
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| Formula | C16H25NO2 |
| Molar mass | 263.381 g·mol−1 |
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Emixustat is a small molecule notable for its establishment of a new class of compounds known as visual cycle modulators (VCMs). Formulated as the hydrochloride salt, emixustat hydrochloride, it is the first synthetic medicinal compound shown to affect retinal disease processes when taken by mouth.[1][2] Emixustat was invented by the British-American chemist, Ian L. Scott, and is currently in Phase 3 trials for dry, age-related macular degeneration (AMD).[3][4][5]
The compound is also being investigated as a potential therapy for proliferative diabetic retinopathy, diabetic macular edema and Stargardt disease.[6] In 2008, Acucela Inc. partnered with Otsuka Pharmaceutical Company for the development of emixustat (ACU-4429) in an agreement totaling $263 million in cash and milestone payments.[7]
Emixustat hydrochloride is a non-retinoid small molecule inhibitor of RPE65 (retinal pigment epithelium-specific 65 kDa protein, also known as retinoid isomerohydrolase). It is thought to reduce visual chromophore biosynthesis and prevent the accumulation of toxic retinal byproducts.[1]
- ^ a b Bavik C, Henry SH, Zhang Y, Mitts K, McGinn T, Budzynski E, et al. (2015). "Visual Cycle Modulation as an Approach toward Preservation of Retinal Integrity". PLOS ONE. 10 (5): e0124940. Bibcode:2015PLoSO..1024940B. doi:10.1371/journal.pone.0124940. PMC 4430241. PMID 25970164.
- ^ Kubota R, Boman NL, David R, Mallikaarjun S, Patil S, Birch D (January 2012). "Safety and effect on rod function of ACU-4429, a novel small-molecule visual cycle modulator". Retina. 32 (1): 183–188. doi:10.1097/IAE.0b013e318217369e. PMID 21519291. S2CID 8680462.
- ^ "Ryo Kubota - Acucela Inc". The Wall Street Transcript. March 20, 2006.
- ^ US 7982071, Scott IL, Kuksa VA, Orme MW, Little T, Gall A, Hong F, "Alcoxy Compounds for Disease Treatment", issued 19 July 2011, assigned to Acucela Inc
- ^ Clinical trial number NCT01802866 for "Safety and Efficacy Assessment Treatment Trials of Emixustat Hydrochloride" at ClinicalTrials.gov
- ^ Dugel PU, Novack RL, Csaky KG, Richmond PP, Birch DG, Kubota R (June 2015). "Phase ii, randomized, placebo-controlled, 90-day study of emixustat hydrochloride in geographic atrophy associated with dry age-related macular degeneration". Retina. 35 (6): 1173–1183. doi:10.1097/IAE.0000000000000606. PMC 4452434. PMID 25932553.
- ^ Carroll J. "Acucela inks development deal for two compounds". Fierce Biotech. Fierce Markets. Retrieved 30 March 2016.