May 2, 2022

Encouraging Clinical Trial Results for Alkeus’ Stargardt Disease Treatment

Eye On the Cure Research News

The company is also conducting a Phase 3 clinical trial of the emerging therapy for geographic atrophy.

Get updates on Eye On the Cure Research News

Alkeus Pharmaceuticals reported that its emerging treatment for Stargardt disease slowed the growth of retinal atrophy – i.e., the area of damage to the retina – by approximately 30 percent per year for patients in its Phase 2 TEASE clinical trial at seven clinical research centers in the US. Results from the clinical trial were reported by Hendrik Scholl, MD, PhD, a clinical researcher from the Institute of Molecular and Clinical Ophthalmology Basel, at the 2022 meeting of the Association for Research in Vision and Ophthalmology (ARVO) held in Denver, May 1–5.

Stargardt disease is the leading cause of inherited macular degeneration, affecting about 30,000 people in the US. The condition is often diagnosed in children and young adults and leads to significant central vision loss.

The hallmark of Stargardt disease is the accumulation of waste products known as lipofuscin due to the defective processing of vitamin A in the retina. The waste products accumulate in the retinal pigment epithelium (RPE), a single layer of cells which provides critical support for photoreceptors, the cells that make vision possible. When the RPE becomes dysfunctional and degenerates due to lipofuscin accumulation, photoreceptors and central vision are lost.

Known as ALK-001, Alkeus’ emerging, oral therapy is a modified form of vitamin A, which, when metabolized in the retina, results in much less lipofuscin accumulation. Scientists developed ALK-001 by replacing hydrogen atoms in vitamin A with deuterium. Known as deuterated vitamin A, it "burns cleaner" than the natural form. Deuterium is a safe, naturally occurring, non-radioactive form of hydrogen which is present in the human body.

Alkeus continues to enroll Stargardt disease patients throughout the US. Patients as young as 8 years old can potentially participate. To learn more about enrolling in the trial, visit the listing or contact Gabrielle DeBartolomeo at

Alkeus’ clinical trial of ALK-001 for geographic atrophy, the advanced form of dry AMD, is fully enrolled. The company plans to report results for the GA trial in the second half of 2023.