In short
Gildeuretinol acetate (development code ALK-001) is an investigational small molecule being studied for Stargardt disease, an inherited form of macular degeneration. It is currently in Phase 3 clinical testing, with one authorised trial (NCT07419334) planning to enroll 224 participants. The trial compares gildeuretinol acetate 14 mg capsules taken orally against a placebo to see whether it can slow the growth of retinal atrophic lesions.
Gildeuretinol acetate, also known by its development code ALK-001, is an investigational small molecule. It is a form of vitamin A acetate labelled with deuterium at position C20, which is why it is sometimes called C20-D3-retinyl acetate. The substance has not received marketing authorisation and remains under clinical investigation.
Key points
- Gildeuretinol acetate (ALK-001) is an investigational small molecule, not yet an authorised medicine.
- It is being studied in Stargardt disease (STGD1), an inherited retinal condition.
- The single registered trial is a Phase 3 study called NORTHSTAR.
- The trial plans to enroll 224 participants and is currently authorised.
- Treatment is given as 14 mg capsules taken orally.
- The main measure is the annual growth rate of retinal atrophic lesions from month 6 to month 24.
What is gildeuretinol acetate?
Gildeuretinol acetate is a chemically synthesised small molecule classified as an investigational substance. It is a deuterated form of vitamin A acetate, meaning that one of the hydrogen atoms in the molecule has been replaced by deuterium, a heavier isotope of hydrogen. This modification is intended to change how the molecule behaves in the body. The substance appears in registry records under the names gildeuretinol acetate, ALK-001, and C20-D3-retinyl acetate. It is not currently an authorised medicine.
How does gildeuretinol acetate work?
Gildeuretinol acetate is known to interact with the ABCA4 transporter protein. ABCA4 is a protein found in the retina that helps clear vitamin A by-products from photoreceptor cells. In Stargardt disease, mutations in the ABCA4 gene lead to accumulation of toxic vitamin A dimers in the retina, which damages retinal cells and causes progressive vision loss. The deuterated structure of gildeuretinol acetate is designed to reduce the formation of these toxic by-products while still providing the normal functions of vitamin A.
Which conditions is gildeuretinol acetate studied for?
- Stargardt disease (STGD1) — an inherited form of macular degeneration that causes progressive loss of central vision, typically beginning in childhood or adolescence. The Phase 3 NORTHSTAR trial is evaluating whether gildeuretinol acetate can slow the progression of retinal atrophic lesions in people with this condition.
How is gildeuretinol acetate given?
In the registered clinical trial, gildeuretinol acetate is given as capsules for oral use at a dose of 14 mg. The placebo comparator capsules are manufactured to be visually identical to the active capsules in size, shape, colour, and packaging, so that participants and investigators cannot tell them apart.
What do the clinical trials show?
One clinical trial of gildeuretinol acetate is registered: a Phase 3 study called NORTHSTAR (NCT07419334). The trial is currently authorised, meaning it has received regulatory approval to begin enrolling participants. It plans to enroll 224 participants with Stargardt disease.
The primary outcome measure is the annualised growth rate of retinal atrophic lesions, assessed by fundus autofluorescence (FAF) imaging, measured from month 6 to month 24 of treatment. Fundus autofluorescence is an imaging technique that shows areas of retinal damage by detecting natural fluorescence in the back of the eye. The trial is designed to evaluate whether gildeuretinol acetate can slow the progression of retinal atrophic lesion growth compared with placebo.
Because the trial is in its early stages and no results have yet been reported, there are no efficacy or safety findings available from this study at present.
What side effects are known?
The registered sources do not provide information on side effects of gildeuretinol acetate. As the Phase 3 trial is still in the authorised stage, no safety data from this study have been published. Side effect information would be expected to emerge once the trial reports results.