Alkeus Pharma to Present Positive Gildeuretinol Data in Early Stargardt at ARVO 2024

Alkeus Pharmaceuticals, Inc., based in Cambridge, Massachusetts, has announced promising new data from its TEASE-3 clinical trial involving gildeuretinol in teenagers and young adults with early-stage Stargardt disease. These findings are set to be presented at the ARVO 2024 Annual Meeting in Seattle, which will take place from May 5-9.

The TEASE-3 trial, an open-label study, is part of a suite of clinical trials assessing the efficacy of gildeuretinol acetate (ALK-001) in Stargardt disease. TEASE-3 focuses on evaluating the treatment in patients at the early stages of this genetic eye disorder. The oral presentation titled "Gildeuretinol Arrested Stargardt Disease, The TEASE-3 Study," delivered by Dr. Michael B. Gorin from UCLA, is scheduled for May 7, from 2:30 p.m. to 2:45 p.m. PDT. This session, numbered 334, will take place in the Yakima 1 room at the Seattle Convention Center – Arch Building.

Several poster presentations will also feature significant findings from Alkeus' research. On May 8, from 2:15 p.m. to 5:15 p.m. PDT, Gabrielle DeBartolomeo from Alkeus will present "Gildeuretinol Rapidly Replaces Plasma Vitamin A, a Phase 1 Trial" during Poster Session 441. This session will cover new drugs, mechanisms of action, and toxicity, with DeBartolomeo's poster numbered 5103 and located at Board A0360. On May 9, Dr. Hendrik P.N. Scholl from the Institute of Molecular and Clinical Ophthalmology in Basel will discuss "Gildeuretinol Enables the Vitamin A Cycle" in Poster Session 535. This poster, numbered 6438 and placed at Board B0987, will be presented from 11:45 a.m. to 5:00 p.m. PDT.

Further, Monica Hennessey from Alkeus will present a poster titled "Industry and Academic Leaders Chose Microperimetry as Most Promising Functional Endpoint for Geographic Atrophy Trials" on May 9, during Poster Session 505. This session, focusing on clinical research in AMD, will take place from 8:00 a.m. to 5:00 p.m. PDT, with Hennessey's poster numbered 5702 at Board B0108. Additionally, Adam Qu from Alkeus will advocate for reducing patient burden in clinical trials for geographic atrophy in the same session. His poster, numbered 5693 and located at Board B0099, will also be presented on May 9.

The TEASE trials are a series of clinical studies investigating gildeuretinol acetate (ALK-001) in Stargardt disease, comprising TEASE-1, TEASE-2, TEASE-3, and TEASE-4. TEASE-1, a randomized, double-masked, placebo-controlled trial involving 50 patients, demonstrated a 21% reduction in the growth rate of retinal atrophic lesions compared to untreated patients, with gildeuretinol proving to be well-tolerated. TEASE-2 is an ongoing trial with 80 patients expected to deliver topline data in 2025. TEASE-4 is an open-label extension study.

Gildeuretinol acetate (ALK-001) is a novel deuterated vitamin A molecule aimed at reducing vitamin A dimerization without impairing vision. Preclinical studies showed that gildeuretinol decreased vitamin A dimerization to normal levels seen in unaffected individuals and prevented retinal degeneration and vision loss in animals with Stargardt disease. The U.S. FDA has granted this molecule breakthrough therapy and orphan drug designations.

Besides the TEASE trials, Alkeus is also conducting a Phase 3 (SAGA) study of gildeuretinol in 200 patients with geographic atrophy secondary to age-related macular degeneration (AMD), with topline data expected in 2024.

Alkeus Pharmaceuticals, founded in 2010 and headquartered in Cambridge, develops therapies for severe eye diseases with high unmet needs, supported by institutional investors led by Bain Capital Life Sciences.