Drug Profile


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

Latest Information Update: 10 Sep 2017

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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

Highest Development Phases

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

Most Recent Events

  • 26 Jan 2017 Phase-II clinical trials in Stargardt disease 1 in USA (PO) (NCT03033108)
  • 05 Jan 2017 Preclinical trials in Stargardt disease 1 in USA (PO) before January 2017
  • 05 Jan 2017 Emixustat receives Orphan Drug status for Stargardt disease 1 in USA
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