Phase 2/3 Study of ALK-001 in Geographic Atrophy

Summary

This is a double-masked, multicenter, randomized, placebo-controlled clinical trial, evaluating the efficacy and safety of ALK-001 in participants with Geographic Atrophy (GA) secondary to age-related macular degeneration (AMD). Up to 200 participants will receive ALK-001 while up to 100 participants will receive a placebo.

Timeline

Start

2019-05-07

Primary completion

2023-07-25

Completion

2024-06-30

Outcome

Missed primary endpoint

paper The prespecified primary endpoint was not met. PMID 42242606 ↗

release “reduction of 0.25 sqmm/year vs. placebo (p=0.07) in the primary endpoint of GA lesion growth rate at 24 months.” alkeuspharma.com ↗

Publications

• Background Kaufman Y, Ma L, Washington I. Deuterium enrichment of vitamin A at the C20 position slows the formation of detrimental vitamin A dimers in wild-type rodents. J Biol Chem. 2011 Mar 11;286(10):7958-7965. doi: 10.1074/jbc.M110.178640. Epub 2010 Nov 12.
• Background Mihai DM, Jiang H, Blaner WS, Romanov A, Washington I. The retina rapidly incorporates ingested C20-D(3)-vitamin A in a swine model. Mol Vis. 2013 Jul 25;19:1677-83. Print 2013.
• Background Saad L, Washington I. Can Vitamin A be Improved to Prevent Blindness due to Age-Related Macular Degeneration, Stargardt Disease and Other Retinal Dystrophies? Adv Exp Med Biol. 2016;854:355-61. doi: 10.1007/978-3-319-17121-0_47.
• Boyer DS, Saad L, Washington I, Melamud A, Graham KB, O'Malley AE, DeBartolomeo GM, Jiang H, Harris LD, Kay CN, Ferrone PJ, Khan S; SAGA Study Team. Gildeuretinol Acetate versus Placebo for Geographic Atrophy Secondary to Age-Related Macular Degeneration: The Study of ALK-001 in Geographic Atrophy Randomized Clinical Trial. Ophthalmol Retina. 2026 Jun 3:S2468-6530(26)00268-X. doi: 10.1016/j.oret.2026.05.018. Online ahead of print.
• Evans JR, Lawrenson JG. Antioxidant vitamin and mineral supplements for slowing the progression of age-related macular degeneration. Cochrane Database Syst Rev. 2023 Sep 13;9(9):CD000254. doi: 10.1002/14651858.CD000254.pub5.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	ALK-001	Small molecule	14 mg	Oral

Indications