Alkeus Pharmaceuticals announces gildeuretinol data presentations during the American Society of Retina Specialists (ASRS) 44th Annual Meeting July 15 - 18. CAMBRIDGE, Mass., July 09, 2026 (GLOBE NEWSWIRE) - Alkeus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases, today announced that data from its clinical studies of investigational oral gildeuretinol for the treatment of Stargardt disease (TEASE) and a study design overview of the currently enrolling Phase 3 NORTHSTAR Study will be presented during the American Society of Retina Specialists (ASRS) 44th Annual Meeting being held July 15 - 18, 2026, in Montreal, Canada. Oral Presentation: * Oral Gildeuretinol for Slowing Stargardt Disease Progression: Safety and Efficacy Data From the Phase 2 TEASE Study and Phase 3 Study Design Date: July 18, 2026 Session: Hereditary Retinal Disease & Genetics Panel. Stargardt Disease: Emerging Therapies, Biomarkers, and Clinical Trial Design Time: 8:48 a.m. - 9:13 a.m. EDT Location: Palais des congrès de Montréal Presenter: Christine N. Kay, M.D., Vitreo Retinal Associates, Gainesville, Fla. Paper on Demand (Recorded Video Presentation) * Baseline Characteristics From the Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE) Clinical Program Hereditary Retinal Disease & Genetics Date: Available on demand for meeting registrants Presenter: Kenneth Fan, M.D., M.B.A., Retina Consultants of Texas Alkeus is actively enrolling and recently announced dosing of the first patient in its global Phase 3 NORTHSTAR Study of gildeuretinol as a potential treatment for patients with Stargardt disease. The study is evaluating gildeuretinol's potential to reduce the growth rate of retinal atrophic lesions and to preserve visual acuity. About Alkeus Pharmaceuticals Alkeus Pharmaceuticals, Inc. is a private biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases. Based in Cambridge, Mass., Alkeus is backed by institutional investors led by Bain Capital Life Sciences. Alkeus is developing therapies for serious diseases of the eye with high unmet need. Alkeus' breakthrough-designated lead candidate, gildeuretinol acetate (ALK-001), currently is being evaluated in a Phase 3 clinical trial for the treatment of Stargardt disease. About Gildeuretinol Acetate (ALK-001) Oral gildeuretinol acetate (ALK-001) is a new molecular entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. Gildeuretinol is being evaluated in clinical trials for the treatment of Stargardt disease and has been studied for geographic atrophy secondary to age-related macular degeneration. Gildeuretinol has received Breakthrough Therapy, Rare Pediatric Disease, Fast Track and Orphan Drug designations for Stargardt disease from the U.S. Food and Drug Administration (FDA). The European Medicines Agency (EMA) has designated gildeuretinol as an orphan medicinal product for the treatment of non-syndromic inherited retinal dystrophies due to defects in the ABCA4 gene, which includes Stargardt disease. About the Phase 3 NORTHSTAR Study The NORTHSTAR Study (NCT07419334) is a Phase 3 randomized, placebo-controlled, double-masked 24-month trial designed to evaluate the efficacy and safety of investigational gildeuretinol in people living with advanced Stargardt disease. The primary endpoint is the rate of growth of atrophic lesions from months 6 to 24 comparing gildeuretinol to placebo. The key secondary endpoint is the preservation of visual acuity as measured by low luminance visual acuity (LLVA). Alkeus aims to enroll approximately 230 participants globally in the study between the ages of 8 and 45, building on previously observed findings across more than 400 patients treated with gildeuretinol to date. About the TEASE Program The Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies consist of four independent clinical studies of oral gildeuretinol (ALK-001) in Stargardt disease, denoted as TEASE-1, TEASE-2, TEASE-3 and TEASE-4. The TEASE-1 (NCT02402660) study was a randomized, double-masked, placebo-controlled trial in patients with advanced Stargardt disease. The TEASE-2 (NCT02402660) trial was a randomized, double-masked, placebo-controlled trial in patients with moderate Stargardt disease. TEASE-3 (NCT02402660) is an open-label study of gildeuretinol in genetically confirmed patients with early signs of Stargardt disease visible on retinal imaging, but who have not begun experiencing symptoms of vision loss. TEASE-4 (NCT04239625) is an open-label extension study. For further information, contact: [email protected] Website: www.alkeuspharma.com

CAMBRIDGE, Mass. - Alkeus Pharmaceuticals, Inc. announced today that it has raised a $150 million Series B financing to support the registration and launch of gildeuretinol (ALK-001), a potential disease-modifying, precision medicine for the treatment of Stargardt disease, a leading genetic cause of blindness in children and young adults. The financing was led by Bain Capital Life Sciences, with additional participation by TCGX, Wellington Management and Sofinnova Investments. Alkeus also announced today that Joshua Boger, Ph.D., a biotech industry pioneer and the founder of Vertex Pharmaceuticals, has joined Alkeus as Executive Chairman.

Alkeus Pharmaceuticals announces presentations of gildeuretinol data during the Research in Vision and Ophthalmology (ARVO) 2025 annual meeting May 4-8, 2025.