Replacing vitamin A - Preventing Blindness

Alkeus Pharmaceuticals is a clinical-stage biopharmaceutical company focused on discovering and developing treatments against serious and untreatable diseases of the eye.

The eye is one of the most complex and precious organs of the body : open or closed, the eye is constantly at work, making it fragile and vulnerable and resulting in hundreds of ocular diseases. Building on years of groundbreaking research in ophthalmology and on well-understood biochemical processes, we work on creative ways to combine this scientific knowledge to invent novel medicines and bring them from the laboratories to the bedside.

As of 2020, Alkeus Pharma's lead program, currently in human clinical trials, is based on novel molecules to address aging of the eye (dry age-related macular degeneration, also known as dry-AMD) and premature aging of the eye due to a genetic condition called Stargardt disease. Dry-AMD and Stargardt disease are two major causes of blindness in patients over 50 and in children/young adults (Stargardt). In laboratory experiments and during animal testing, these molecules have shown promise by preventing the formation and accumulation of toxic ocular pigments called "vitamin A dimers" and preserving visual function in animals.

Alkeus Pharma's lead drug candidate is ALK-001, an orally-delivered compound that could potentially treat Stargardt disease and age-related macular degeneration. Stargardt disease is a rare genetic condition that results in symptoms of irreversible vision loss early in life (6-30 years old). The genetic defect has been shown to cause poor recycling of vitamin A resulting in accelerated formation of toxic vitamin A-related compounds in the eye. As mice are different from humans, well-controlled clinical trials are necessary to assess the effects of ALK-001 on the vision of those with Stargardt disease or age-related macular degeneration. ALK-001 is currently being tested in a Phase 2 trial for the treatment of Stargardt disease and a Phase 3 trial for the treatment of Geographic Atrophy secondary to AMD and

Because Stargardt disease is a rare disease, it makes it hard to find patients: we therefore invite you to register your name in our confidential mailing list if you have been diagnosed with Stargardt or to contact us by email at We will keep you informed of future trials.

Among its other interests, Alkeus Pharma is investigating methods to treat other rare genetic diseases such as Leber Hereditary Optic Neuropathy (LHON), an inherited disease that leads to sudden loss of vision in young adults as well as in autosomal recessive Retinitis Pigmentosa and in Cone Rod Dystrophy, two forms of serious vision loss that often occur early in life.