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Drug Profile


Alternative Names: ACU-02; ACU-4429; ACU-4429 HCl; Emixustat HCl; Emixustat hydrochloride

Latest Information Update: 17 Dec 2018

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At a glance

  • Originator Acucela
  • Developer Acucela; Otsuka Pharmaceutical
  • Class Cyclohexanes; Eye disorder therapies; Phenyl ethers; Propanolamines; Small molecules
  • Mechanism of Action Retinoid isomerohydrolase inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Stargardt disease
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Phase III Stargardt disease
  • Phase II/III Dry age-related macular degeneration
  • Phase II Diabetic retinopathy

Most Recent Events

  • 07 Nov 2018 Phase-III clinical trials in Stargardt disease in USA (PO) (NCT03772665)
  • 28 Jun 2018 9243873- updated intro, orgT, TT and HE. Brief profile review done
  • 24 Jun 2018 Biomarkers information updated
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