ALK-001: once-a-day oral, investigational therapy to prevent blindness
ALK-001 is a chemically-modified vitamin A being investigated for the prevention and treatment of Stargardt disease and age-related macular degeneration (AMD). ALK-001 is taken once a day by mouth.
Toxic vitamin A aggregates (“dimers”) have been implicated in the pathophysiology of Stargardt and dry-AMD. Vitamin A dimers form when two molecules of vitamin A are combined. ALK-001 is a form of vitamin A that has been chemically-modified to slow the formation of toxic vitamin A dimers in the eye.
Specifically, ALK-001 is vitamin A modified by replacing hydrogen with deuterium, a stable, non-radioactive isotope of hydrogen. Substituting vitamin A with ALK-001 safely slows the rate of formation of vitamin A dimers and prevents the disease in mice.
In humans, a Phase 2 clinical trial of ALK-001 in patients with Stargardt disease has shown statistically and clinically-significant efficacy. Results will be presented and published later this year.