Another new drug offers hope for Stargardt patients

Posted: Monday 29 January 2024
Scientist with drug vial

Another drug to potentially treat Stargardt has shown positive results in slowing down the progression of the disease in a recent trial.

Alkeus Pharmaceuticals has revealed promising early results from its TEASE-3 clinical trial of the drug ALK-001. When given to young people in the early stages of the disease, it appears to be able to halt progression before retinal damage and vision loss occurs.

The trial 

Similar to Remofuscin, the trial looked at early-stage Stargardt disease. It saw three teenagers treated with the drug and during the two to six years of treatment none of the trial participants saw any deterioration in their condition.

Projections suggested that without treatment, the participants would typically experience vision loss within two years, similar to their untreated siblings with identical genetic mutations.

Leonide Saad, CEO of Alkeus Pharmaceutical is hopeful about gildeuretinol acetate stopping sight loss from Stargardt disease, especially if treated early.

Dry AMD 

In addition to the Stargardt trials, Alkeus Pharmaceuticals is also testing the drug on 200 people with late-stage dry age-related macular degeneration (AMD), also known as geographic atrophy (GA).  

For more information, visit Alkeus Pharmaceutical's website.

Close up of blue eye looking up

Stargardt disease link arrow

Stargardt disease is a genetic condition caused by a tiny alteration in a single gene. It is the most common form of juvenile macular dystrophy.

Elderly lady eyes closeup

Age-related macular degeneration link arrow

Age-related macular degeneration (AMD) usually affects people over 50 but can happen earlier. AMD is the biggest cause of sight loss in the UK, affecting more than 700,000 people.

Researcher in laboratory

Explore our research link arrow

Beating macular disease through funding medical research and improving the lives of those living with macular disease.

Friendly support

Support for you link arrow

We provide free information and support to those with macular disease, along with their family and friends, to help people keep their independence.