Alkeus Pharmaceuticals Announces Positive Interim Results Demonstrating No Signs of Disease Progression in Early-Stage Stargardt Disease Patients Treated with Gildeuretinol

TEASE-3 study data in early-stage Stargardt disease patients presented at ARVO 2024

CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc. today announced the presentation of positive interim data from its TEASE-3 study demonstrating that early-stage Stargardt disease patients treated with gildeuretinol acetate showed no disease progression and remained asymptomatic for the duration of therapy ranging between two and six years. Results were presented during the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting being held May 5-9 in Seattle.

“These exciting results demonstrate the potential of gildeuretinol acetate to prevent vision loss in early-stage Stargardt patients,” said Leonide Saad, Ph.D., President and CEO of Alkeus Pharmaceuticals. “We projected that without treatment, patients in the TEASE-3 study would lose vision, following a similar trajectory of vision loss as their previously-diagnosed siblings with identical ABCA4 genetic mutations. All of us working in our growing Alkeus team are committed to advancing research and having an impact on patients living with vision loss. We look forward to submitting our gildeuretinol data for regulatory review later this year.”

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 disease visible on retinal imaging, but who have not begun experiencing symptoms of vision loss. Retinal imaging and functional outcome measures were used to assess disease progression. To date the TEASE-3 study has enrolled a total of five patients, who receive gildeuretinol as a once-a-day pill.

“Interim results of the TEASE-3 trial demonstrating clinical benefit of gildeuretinol in early-stage Stargardt disease patients are extremely promising,” said Michael B. Gorin, M.D., Ph.D., of the Departments of Ophthalmology and of Human Genetics at the David Geffen School of Medicine at UCLA. “The potential of gildeuretinol to significantly slow disease progression and delay vision loss in young patients is an exciting advancement.”

Stargardt disease is a serious cause of blindness in children and young adults, with an estimated 30,000 people affected in the U.S. and more than 150,000 worldwide. There is no approved treatment. In individuals with Stargardt disease, the ABCA4 protein is defective. This defect in the protein results in the accelerated dimerization of vitamin A, forming toxic by-products that irreversibly damage the retina, resulting in progressive vision loss.

About the TEASE Trials

The TEASE trials consist of four clinical studies of gildeuretinol acetate (ALK-001) in Stargardt disease, denoted as TEASE-1, TEASE-2, TEASE-3 and TEASE-4. The TEASE-1 study was a randomized, double-masked, placebo-controlled trial in 50 patients with Stargardt disease. Gildeuretinol met its prespecified primary efficacy endpoint showing a 21% reduction in the growth rate of retinal atrophic lesions (p<0.001, square root units, 28% reduction for untransformed areas of retinal atrophic lesions) against untreated patients. Gildeuretinol was well-tolerated. The TEASE-2 trial is an ongoing, fully enrolled, randomized, double-masked, placebo-controlled trial in 80 patients with Stargardt disease, expected to read out topline data in 2025. TEASE-3 is an open-label study designed to assess gildeuretinol in early-stage Stargardt patients. 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 the dimerization of vitamin A without disrupting vision. In preclinical studies, gildeuretinol decreased vitamin A dimerization to the normal rate 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 (SAGA) study of gildeuretinol in 200 patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) is expected to read out topline data in 2024.

About Alkeus Pharmaceuticals

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

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