Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
emixustat
- Development stage
- Phase 3
- Lead developer
- Kubota Vision
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Emixustat is an orally available, synthetic small molecule that acts as a non-retinoid inhibitor of the retinal pigment epithelium-specific 65 kDa protein (RPE65), also known as retinoid isomerohydrolase. By inhibiting RPE65, emixustat modulates the visual cycle and reduces the formation of 11-cis-retinal, thereby decreasing the production of toxic bis-retinoids such as A2E that accumulate in retinal diseases. This mechanism is intended to protect against retinal damage in conditions like Stargardt disease and age-related macular degeneration. Emixustat has been developed primarily for Stargardt disease (with orphan drug status) and has also been investigated for diabetic retinopathy and dry age-related macular degeneration[1][3][4][5][7].
- Brand names
- RETAKU
- Other names
- emixustat hydrochlorideemixustat HCl
02
Targets
RPE65 (Retinal pigment epithelium-specific 65 kDa protein)
Beyond the preview
Go deeper on emixustat.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on emixustat.