emixustat

Small moleculeexperimental

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. 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). — Wikipedia

Clinical trial activity

8 trials · 3 clinical orgs · 0 marketing orgs

Phase 1
3
Phase 2
4
Phase 3
2
Phase 4
0

Earliest trial started May 1, 2008 (NCT00703183)

Timeline

2000s

  1. Jan 1, 2008

    Earliest Phase 1 Sponsor(trial)

  2. Jan 1, 2009

    Earliest Phase 2 Sponsor(trial)

2010s

  1. Jan 1, 2018

    Earliest Phase 3 Sponsor(trial)

Indications

Mechanism of action

  • RPE65 inhibitor

    Nonretinoid compound with a unique mode of action in the retinal pigment epithelium, where it modulates the biosynthesis of visual chromophore through its effect on retinal pigment epithelium-specific 65 kDa protein isomerase.

Chemistry & pharmacology

Loading structure…

SMILES

NCC[C@@H](O)c1cccc(OCC2CCCCC2)c1
Mol. weight
263.38 g/mol
Lipinski Ro5
Pass
Rule of 3
No
Chirality
Single Stereoisomer
Inorganic
No
Polymer
No

Oral

No

Parenteral

No

Topical

No

Sources

Also known as

  • acu-4429
  • acu-4429
  • acu-4429
  • emixustat
  • emixustat
  • emixustat
  • emixustat
  • emixustat
  • emixustat hcl
  • emixustat hcl
  • emixustat hydrochloride
  • emixustat hydrochloride
  • emixustat hydrochloride

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT03772665Phase 3Nov 7, 2018Acucela Inc.
NCT03033108Phase 2Jan 1, 2017Acucela Inc.
NCT02753400Phase 2Apr 1, 2016Acucela Inc.
NCT02130531Phase 1Apr 1, 2014Acucela Inc.
NCT01802866Phase 2/Phase 3 (Phase 3)Feb 1, 2013Acucela Inc., Otsuka
NCT01002950Phase 2Oct 1, 2009Acucela Inc., Otsuka
NCT00942240Phase 1Jul 1, 2009Acucela Inc., Covance, Otsuka
NCT00703183Phase 1May 1, 2008Acucela Inc., Covance
Showing 8 of 8 trials
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Organizations

Research & Development (3)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
Acucela Inc.For profit8832008
OtsukaFor profit3032009
CovanceFor profit2012008
3 organizations
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