Alkeus says its eye therapy halts progression of genetic disease

[January 10, 2024]  By Sriparna Roy

(Reuters) - Privately held Alkeus Pharmaceuticals said on Wednesday its experimental therapy halted disease progression in patients with early stages of a genetic eye condition that leads to blindness.

The inherited condition, known as Stargardt disease, has no available treatments and is a leading genetic cause of blindness in children and young adults, with an estimated 30,000 people affected in the United States and more than 150,000 worldwide.

Three teenage patients when treated with the oral therapy, gildeuretinol, remained free of symptoms and free of disease progression for their treatment duration for up to six years.

Patients were projected to begin experiencing vision loss within two years, in the absence of the treatment, following the disease trajectory of their older siblings with identical genetic mutations who were not treated with the therapy.

In a previous Phase 2 trial, the therapy successfully slowed the growth rate of retinal lesions in patients with more advanced stages of the disease, when compared to placebo.

"We've been able to demonstrate the very important effectiveness in late stage - we can't bring the vision back, the vision damage is permanent, but we can preserve some of the last retina that is still alive," CEO Leonide Saad told Reuters.

The once-a-day therapy is a form of vitamin A which is designed to slow or halt progressive vision loss.

"This is the most perfect drug I've ever seen," said Vertex Pharmaceuticals' founder Joshua Boger, who is currently the executive chairman at Alkeus.

Alkeus plans to submit its marketing application for review with the U.S. health regulator based on the results of the Phase 2 study, aiming to be ready to launch this year.

Stargardt disease is caused by the changes in a gene called ABCA4, which influences the way the body used vitamin A, resulting in damage to the retina which leads to progressive vision loss.

(Reporting by Sriparna Roy in Bengaluru; Editing by Maju Samuel)

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