emixustat profile

emixustat: a visual cycle modulator that inhibits retinal pigment epithelium 65 protein [Medical Subject Headings (MeSH), National Library of Medicine, extracted Dec-2023]

ID Source	ID
PubMed CID	25221720
CHEMBL ID	2107821
SCHEMBL ID	966515
MeSH ID	M000599381

Synonym
CHEMBL2107821
acu-4429
emixustat
emixustat (usan)
D10348
02dz1hbf0m ,
unii-02dz1hbf0m
1141777-14-1
emixustat [usan:inn]
acu 4229
emixustat [who-dd]
emixustat [usan]
emixustat [inn]
(r)-(+)-3-amino-1-(3-(cyclohexylmethoxy)phenyl)propan-1-ol
(1r)-3-amino-1-[3-(cyclohexylmethoxy)phenyl]propan-1-ol
benzenemethanol, .alpha.-(2-aminoethyl)-3-(cyclohexylmethoxy)-, (.alpha.r)-
acu02
SCHEMBL966515
a3v ,
DTXSID50150665
HY-19720
CS-6177
3-amino-1-[3-(cyclohexylmethoxy)phenyl]propan-1(r)-ol hydro -chloride
DB12608
AKOS032945088
Q25323930
WJIGGYYSZBWCGC-MRXNPFEDSA-N
(r)-3-amino-1-(3-(cyclohexylmethoxy)phenyl)propan-1-ol
us10188615, example 28
bdbm323419
F85505
MS-23717

Research Excerpts

Overview

Emixustat is a visual cycle modulator that has entered clinical trials as a treatment for age-related macular degeneration (AMD) It reduces chromophore levels and, therefore, may mimic light conditions.

Excerpt	Reference	Relevance
"Emixustat is a visual cycle modulator (VCM) that reduces chromophore levels and, therefore, may mimic light conditions."	( Emixustat Reduces Metabolic Demand of Dark Activity in the Retina. Calkins, DJ; Henry, SH; Kubota, R; Linsenmeier, RA, 2019)	2.68
"Emixustat is a visual cycle modulator that has entered clinical trials as a treatment for age-related macular degeneration (AMD). "	( Molecular pharmacodynamics of emixustat in protection against retinal degeneration. Badiee, M; Dong, Z; Golczak, M; Kiser, PD; Palczewska, G; Palczewski, K; Tochtrop, GP; Zhang, J, 2015)	2.15

Treatment

Excerpt	Reference	Relevance
"Emixustat treatment reduced cation channel activity; activity was comparable to vehicle-treated controls in light conditions."	( Emixustat Reduces Metabolic Demand of Dark Activity in the Retina. Calkins, DJ; Henry, SH; Kubota, R; Linsenmeier, RA, 2019)	2.68

Toxicity

Excerpt	Reference	Relevance
" Safety assessments included adverse events, vital signs, clinical laboratory assays, electrocardiograms, and ophthalmologic examination."	( Safety and effect on rod function of ACU-4429, a novel small-molecule visual cycle modulator. Birch, D; Boman, NL; David, R; Kubota, R; Mallikaarjun, S; Patil, S, 2012)	0.38
" Adverse events were mild and visual in nature (dyschromatopsia and alteration in dark adaptation), transient, and resolved within a few days."	( Safety and effect on rod function of ACU-4429, a novel small-molecule visual cycle modulator. Birch, D; Boman, NL; David, R; Kubota, R; Mallikaarjun, S; Patil, S, 2012)	0.38

Bioavailability

Excerpt	Reference	Relevance
" Other attributes of emixustat, such as oral bioavailability and target specificity make it an attractive candidate for clinical development in the treatment of retinal disease."	( Visual Cycle Modulation as an Approach toward Preservation of Retinal Integrity. Bavik, C; Blumberg, B; Budzynski, E; Fawzi, A; Henry, SH; Kubota, R; Kuksa, V; Lieu, KL; McGinn, T; Mitts, K; Orme, M; Pashko, A; Scott, I; Zhang, Y; Zhong, S, 2015)	0.74

[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Timeframe	Studies, This Drug (%)	All Drugs %
pre-1990	0 (0.00)	18.7374
1990's	0 (0.00)	18.2507
2000's	0 (0.00)	29.6817
2010's	16 (66.67)	24.3611
2020's	8 (33.33)	2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Publication Type	This drug (%)	All Drugs (%)
Trials	5 (20.83%)	5.53%
Reviews	6 (25.00%)	6.00%
Case Studies	0 (0.00%)	4.05%
Observational	0 (0.00%)	0.25%
Other	13 (54.17%)	84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Substance	Relationship Strength	Studies	Classes	Roles
carbamylhydrazine carbamylhydrazine: RN given refers to parent cpd	2.21	1	carbohydrazide; monocarboxylic acid amide; one-carbon compound; ureas
sodium fluoride [no description available]	2.11	1	fluoride salt	mutagen
benzylamine aminotoluene : Any member of the class of toluenes carrying one or more amino groups.	2.21	1	aralkylamine; primary amine	allergen; EC 3.5.5.1 (nitrilase) inhibitor; plant metabolite
manganese Manganese: A trace element with atomic symbol Mn, atomic number 25, and atomic weight 54.94. It is concentrated in cell mitochondria, mostly in the pituitary gland, liver, pancreas, kidney, and bone, influences the synthesis of mucopolysaccharides, stimulates hepatic synthesis of cholesterol and fatty acids, and is a cofactor in many enzymes, including arginase and alkaline phosphatase in the liver. (From AMA Drug Evaluations Annual 1992, p2035). manganese(4+) : A manganese cation that is monoatomic and has a formal charge of +4.	2.21	1	elemental manganese; manganese group element atom	Escherichia coli metabolite; micronutrient
deuterium Deuterium: The stable isotope of hydrogen. It has one neutron and one proton in the nucleus.	4.78	4	dihydrogen
fluorine Fluorine: A nonmetallic, diatomic gas that is a trace element and member of the halogen family. It is used in dentistry as fluoride (FLUORIDES) to prevent dental caries.	2.31	1	diatomic fluorine; gas molecular entity	NMR chemical shift reference compound
retinylamine retinylamine: structure	2.54	2
retinol Vitamin A: Retinol and derivatives of retinol that play an essential role in metabolic functioning of the retina, the growth of and differentiation of epithelial tissue, the growth of bone, reproduction, and the immune response. Dietary vitamin A is derived from a variety of CAROTENOIDS found in plants. It is enriched in the liver, egg yolks, and the fat component of dairy products.. vitamin A : Any member of a group of fat-soluble retinoids produced via metabolism of provitamin A carotenoids that exhibit biological activity against vitamin A deficiency. Vitamin A is involved in immune function, vision, reproduction, and cellular communication.. all-trans-retinol : A retinol in which all four exocyclic double bonds have E- (trans-) geometry.. retinol : A retinoid consisting of 3,7-dimethylnona-2,4,6,8-tetraen-1-ol substituted at position 9 by a 2,6,6-trimethylcyclohex-1-en-1-yl group (geometry of the four exocyclic double bonds is not specified).	5.86	4	retinol; vitamin A	human metabolite; mouse metabolite; plant metabolite
retinaldehyde Retinaldehyde: A diterpene derived from the carotenoid VITAMIN A which functions as the active component of the visual cycle. It is the prosthetic group of RHODOPSIN (i.e., covalently bonded to ROD OPSIN as 11-cis-retinal). When stimulated by visible light, rhodopsin transforms this cis-isomer of retinal to the trans-isomer (11-trans-retinal). This transformation straightens-out the bend of the retinal molecule and causes a change in the shape of rhodopsin triggering the visual process. A series of energy-requiring enzyme-catalyzed reactions convert the 11-trans-retinal back to the cis-isomer.. all-trans-retinal : A retinal in which all four exocyclic double bonds have E- (trans-) geometry.	3.14	4	retinal; vitamin A	gap junctional intercellular communication inhibitor; human metabolite; mouse metabolite
retinol acetate retinol acetate: structure given in first source	3.27	1	acetate ester
beta carotene beta Carotene: A carotenoid that is a precursor of VITAMIN A. Beta carotene is administered to reduce the severity of photosensitivity reactions in patients with erythropoietic protoporphyria (PORPHYRIA, ERYTHROPOIETIC).. provitamin A : A provitamin that can be converted into vitamin A by enzymes from animal tissues.	3.27	1	carotenoid beta-end derivative; cyclic carotene	antioxidant; biological pigment; cofactor; ferroptosis inhibitor; human metabolite; mouse metabolite; plant metabolite; provitamin A
isotretinoin Isotretinoin: A topical dermatologic agent that is used in the treatment of ACNE VULGARIS and several other skin diseases. The drug has teratogenic and other adverse effects.. isotretinoin : A retinoic acid that is all-trans-retinoic acid in which the double bond which is alpha,beta- to the carboxy group is isomerised to Z configuration. A synthetic retinoid, it is used for the treatment of severe cases of acne and other skin diseases.	3.27	1	retinoic acid	antineoplastic agent; keratolytic drug; teratogenic agent
fenretinide Fenretinide: A synthetic retinoid that is used orally as a chemopreventive against prostate cancer and in women at risk of developing contralateral breast cancer. It is also effective as an antineoplastic agent.. 4-hydroxyphenyl retinamide : A retinoid obtained by formal condensation of the carboxy group of all-trans retinoic acid and the anilino group of 4-hydroxyaniline. Synthetic retinoid agonist. Antiproliferative, antioxidant and anticancer agent with a long half-life in vivo. Apoptotic effects appear to be mediated by a mechanism distinct from that of 'classical' retinoids.	6.6	3	monocarboxylic acid amide; retinoid	antineoplastic agent; antioxidant
retinal dimer retinal dimer: structure in first source	2.25	1
sodium iodate sodium iodate: RN given refers to iodic acid, Na salt	2.21	1	organic molecular entity

Condition	Indicated	Relationship Strength	Studies	Trials
Retinal Diseases Diseases involving the RETINA.	0	4	2	0
Injury, Ischemia-Reperfusion [description not available]	0	2.57	2	0
Intraocular Pressure The pressure of the fluids in the eye.	0	2.25	1	0
Reperfusion Injury Adverse functional, metabolic, or structural changes in tissues that result from the restoration of blood flow to the tissue (REPERFUSION) following ISCHEMIA.	0	2.57	2	0
Retinal Degeneration A retrogressive pathological change in the retina, focal or generalized, caused by genetic defects, inflammation, trauma, vascular disease, or aging. Degeneration affecting predominantly the macula lutea of the retina is MACULAR DEGENERATION. (Newell, Ophthalmology: Principles and Concepts, 7th ed, p304)	0	8.07	4	0
Central Retinal Edema, Cystoid [description not available]	0	4.62	1	1
Diabetes Mellitus A heterogeneous group of disorders characterized by HYPERGLYCEMIA and GLUCOSE INTOLERANCE.	0	4.62	1	1
Diabetic Retinopathy Disease of the RETINA as a complication of DIABETES MELLITUS. It is characterized by the progressive microvascular complications, such as ANEURYSM, interretinal EDEMA, and intraocular PATHOLOGIC NEOVASCULARIZATION.	0	9.62	1	1
Macular Edema Fluid accumulation in the outer layer of the MACULA LUTEA that results from intraocular or systemic insults. It may develop in a diffuse pattern where the macula appears thickened or it may acquire the characteristic petaloid appearance referred to as cystoid macular edema. Although macular edema may be associated with various underlying conditions, it is most commonly seen following intraocular surgery, venous occlusive disease, DIABETIC RETINOPATHY, and posterior segment inflammatory disease. (From Survey of Ophthalmology 2004; 49(5) 470-90)	0	4.62	1	1
Fundus Flavimaculatus [description not available]	0	9.01	11	1
Age-Related Macular Degeneration [description not available]	0	12.71	22	2
Stargardt Disease A juvenile-onset macular dystrophy characterized by progressive loss of VISUAL ACUITY with normal acuity in peripheral VISUAL FIELDS. Other associated clinical features may include LIPOFUSCIN fundus autofluorescence, atrophy of the RETINAL PIGMENT EPITHELIUM, loss of color vision, PHOTOPHOBIA and PARACENTRAL SCOTOMA. Germline mutations in the ABCA4 gene have been identified in recessive and dominant diseases.	1	16.01	11	1
Atrophy Decrease in the size of a cell, tissue, organ, or multiple organs, associated with a variety of pathological conditions such as abnormal cellular changes, ischemia, malnutrition, or hormonal changes.	0	9.72	1	1
Macular Degeneration Degenerative changes in the RETINA usually of older adults which results in a loss of vision in the center of the visual field (the MACULA LUTEA) because of damage to the retina. It occurs in dry and wet forms.	1	14.71	22	2
Disease Exacerbation [description not available]	0	9.21	9	0
Choroid Neovascularization [description not available]	0	9.21	9	0
Dry Macular Degeneration [description not available]	0	11.54	13	2
Geographic Atrophy A form of MACULAR DEGENERATION also known as dry macular degeneration marked by occurrence of a well-defined progressive lesion or atrophy in the central part of the RETINA called the MACULA LUTEA. It is distinguishable from WET MACULAR DEGENERATION in that the latter involves neovascular exudates.	1	13.54	13	2
Disease Models, Animal Naturally-occurring or experimentally-induced animal diseases with pathological processes analogous to human diseases.	0	2.55	2	0
Angiogenesis, Pathologic [description not available]	0	2.11	1	0
Retrolental Fibroplasia [description not available]	0	2.11	1	0
Retinopathy of Prematurity A bilateral retinopathy occurring in premature infants treated with excessively high concentrations of oxygen, characterized by vascular dilatation, proliferation, and tortuosity, edema, and retinal detachment, with ultimate conversion of the retina into a fibrous mass that can be seen as a dense retrolental membrane. Usually growth of the eye is arrested and may result in microophthalmia, and blindness may occur. (Dorland, 27th ed)	0	2.11	1	0
Retinal Dystrophies A group of disorders involving predominantly the posterior portion of the ocular fundus, due to degeneration in the sensory layer of the RETINA; RETINAL PIGMENT EPITHELIUM; BRUCH MEMBRANE; CHOROID; or a combination of these tissues.	0	6.41	9	0
Amaurosis [description not available]	0	6.41	9	0
Blindness The inability to see or the loss or absence of perception of visual stimuli. This condition may be the result of EYE DISEASES; OPTIC NERVE DISEASES; OPTIC CHIASM diseases; or BRAIN DISEASES affecting the VISUAL PATHWAYS or OCCIPITAL LOBE.	0	6.41	9	0
Eye Disorders [description not available]	0	3.04	1	0
Eye Diseases Diseases affecting the eye.	0	3.04	1	0

emixustat

Description

Cross-References

Synonyms (32)

Research Excerpts

Overview

Treatment

Toxicity

Bioavailability

Research

Studies (24)

Market Indicators

Research Demand Index: 33.36

Study Types

emixustat

Description

Cross-References

Synonyms (32)

Research Excerpts

Overview

Treatment

Toxicity

Bioavailability

Research

Studies (24)

Market Indicators

Research Demand Index: 33.36

Study Types

Related Drugs (15)

Related Conditions (27)