Alkeus Pharmaceuticals Announces Presentation of Updated TEASE-3 Study Showing Stagnating of Progression in Patients with Early-Stage Stargardt Disease Treated with Gildeuretinol

Early-stage patient data presented at the 42nd ASRS Annual Scientific Meeting

CAMBRIDGE, Mass., July 18, 2024 (GLOBE NEWSWIRE) — Alkeus Pharmaceuticals, Inc. today announced the presentation of interim data from its TEASE-3 study demonstrating that patients with early-stage Stargardt disease treated with gildeuretinol acetate showed no disease progression and remained asymptomatic over a treatment duration of two to six years. The study update was presented at the 42nd Annual Scientific Meeting of the American Society of Retina Specialists (ASRS), held July 17-20 in Stockholm, Sweden.

“The interim results from the TEASE-3 study of gildeuretinol in patients with early-stage Stargardt disease indicate the potential value of treating patients with confirmed disease-causing ABCA4 genetic mutations as early as possible before the disease causes progressive central vision loss,” said Seemi Kahn, MD, MPH, MBA, Chief Medical Officer of Alkeus Pharmaceuticals. “There is a significant unmet need in this disease area with no treatment, and we look forward to sharing further results from this encouraging study in the future as we work to advance our development program, which has the potential to be the first treatment for Stargardt disease.”

TEASE-3, the first clinical trial in early-stage Stargardt disease, is an open-label study of gildeuretinol in genetically confirmed patients with early signs of the disease visible on retinal imaging but who have not begun to experience symptoms of vision loss. Each participant in TEASE-3 has a sibling who has previously been diagnosed with Stargardt disease, has identical genetic mutations, and has experienced irreversible vision loss. The primary endpoint of disease progression over two years is assessed by retinal imaging and functional outcome measures. After the initial two-year treatment period, patients continue to receive gildeuretinol while enrolled in a long-term, open-label extension study. TEASE-3 enrolled a total of six patients, who are receiving gildeuretinol as a tablet once daily.

“The TEASE-3 study was a unique opportunity to evaluate gildeuretinol in a small group of presymptomatic children for whom we had clinical history information regarding the progression of Stargardt disease in older siblings,” said Michael B. Gorin, MD, PhD, Departments of Ophthalmology and Human Genetics, David Geffen School of Medicine at UCLA. “We were able to evaluate the drug’s ability to delay vision loss in these children compared to age-matched data from their genetically matched Stargardt siblings. The significant delay in vision and photoreceptor loss in this small cohort represents an exciting advance in the treatment of ABCA4-related Stargardt disease and suggests that clinical benefits may be greatest in the early stages of this disease.”

Stargardt disease is a serious cause of blindness in children and young adults. An estimated 30,000 people are affected in the United States and more than 150,000 worldwide. There is no approved treatment. In people with Stargardt disease, the ABCA4 protein is defective. This protein defect results in accelerated vitamin A dimerization, which forms toxic byproducts that irreversibly damage the retina and lead to progressive vision loss.

About TEASE Trials

The TEASE trials consist of four clinical studies of gildeuretinol acetate (ALK-001) in Stargardt disease, designated TEASE-1, TEASE-2, TEASE-3, and TEASE-4. TEASE-1 was a randomized, double-blind, placebo-controlled trial in 50 patients with Stargardt disease. Gildeuretinol met its predefined primary efficacy endpoint, showing a 21% reduction in the rate of retinal atrophic lesion growth (p < 0.001, root-mean-square units, 28% reduction in untransformed areas of retinal atrophic lesions) compared with untreated patients. Gildeuretinol was well tolerated. The TEASE-2 trial is an ongoing, fully enrolled, randomized, double-blind, placebo-controlled trial in 80 patients with Stargardt disease, with preliminary results expected in 2025. TEASE-3 is an open-label study designed to evaluate gildeuretinol in patients with early-stage Stargardt disease. TEASE-4 is an open-label extension study.

About Gildeuretinol Acetate (ALK-001)

Gildeuretinol acetate (ALK-001) is a novel molecule created as a specialized form of deuterated vitamin A designed to reduce vitamin A dimerization without disrupting vision. In preclinical studies, gildeuretinol reduced vitamin A dimerization to the normal level seen in unaffected individuals and prevented retinal degeneration and loss of visual function in animals with Stargardt disease. Gildeuretinol has received Breakthrough Therapy Designation and Orphan Drug Designation from the U.S. Food and Drug Administration. In addition to the TEASE trials, a Phase 3 study (SAGA) of gildeuretinol in 200 patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) is expected to provide top-line data in 2024.

About Alkeus Pharmaceuticals

Alkeus Pharmaceuticals, Inc. is a privately held biopharmaceutical company headquartered in Cambridge, Massachusetts, backed by institutional investors led by Bain Capital Life Sciences. Founded in 2010, Alkeus is developing therapies for serious eye diseases with high unmet needs. Alkeus’ lead candidate, gildeuretinol acetate (ALK-001), has been designated as a breakthrough drug and is currently being evaluated in clinical trials for the treatment of Stargardt disease and geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

For further information please contact:

Media Relations
[email protected]
Website: www.alkeuspharma.com