ALK-001 is a drug candidate being developed by Alkeus Pharmaceuticals for the treatment of Stargardt Disease (STGD1). Vitamin A dimers are the primary cause of vision loss associated with Stargardt Disease due to retinal toxicity. ALK-001 is a modified form of vitamin A that prevents aggregation into dimers.
Stargardt Disease: Phase II
Stargardt disease (or fundus flavimaculatus) is the most common form of inherited macular degeneration, with a prevalence of about 1 per 10,000 births. The disease causes uncorrectable loss of central vision, with most patients experiencing initial symptoms prior to age 20.
Stargardt disease has been shown to be the result of a mutation in the ABCA4 gene. This mutation leads to production of a dysfunctional Rim protein, which is responsible for the intracellular transport of vitamin A. As a result, vitamin A accumulates and forms toxic aggregates within the photoreceptors, leading to eventual vision loss. ALK-001 is deuterated (chemically altered) vitamin A, which prevents this toxic aggregation.
Alkeus is actively enrolling patients in orally-delivered ALK-001 Phase II clinical trials for Stargardt Disease. There is currently no cure or treatment for Stargardt disease, but competitors are developing alternatives to ALK-001 such as SAR 422459 by Sanofi and MA09-hRPE by Astellas Pharma and Ocata Therapeutics.
Managed Care Segment
The managed care segment for ALK-001 is 2B.
Selected Sample of Epidemiology Sources Used in This Analysis
Bocquet B, Lacroux A, Surget MO, et al. Relative frequencies of inherited retinal dystrophies and optic neuropathies in Southern France: assessment of 21-year data management. Ophthalmic Epidemiol. 2013;20(1):13-25.
Chacón-camacho OF, Granillo-alvarez M, Ayala-ramírez R, Zenteno JC. ABCA4 mutational spectrum in Mexican patients with Stargardt disease: Identification of 12 novel mutations and evidence of a founder effect for the common p.A1773V mutation. Exp Eye Res. 2013;109:77-82.
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