Alkeus Pharmaceuticals, Inc.

Alkeus Pharmaceuticals’ lead candidate gildeuretinol acetate (ALK‑001), an oral deuterated form of vitamin A, has shown statistically significant and clinically meaningful efficacy in slowing or halting progression of Stargardt disease across multiple clinical trials. In early-stage patients from the TEASE‑3 trial, no disease progression was observed over several years. The therapy has been well-tolerated and received Breakthrough Therapy Designation from the FDA. Additional studies are ongoing for both Stargardt disease and geographic atrophy secondary to AMD; topline data from pivotal trials are expected soon with an NDA planned for submission in 2025.[4][5][6]