This biopharmaceutical company develops orally delivered compounds to treat serious eye diseases. Their lead drug candidate targets Stargardt disease and age-related macular degeneration, aiming to provide early intervention for irreversible vision loss.
Funding
$150M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
BCFounders
Product
Problem
Progressive retinal diseases, such as Stargardt disease and geographic atrophy secondary to age-related macular degeneration, lead to irreversible vision loss in both children and adults. These conditions often lack effective treatments, resulting in a high unmet need for therapies that can preserve sight. A key mechanism in these diseases is the accumulation of toxic vitamin A dimers in the retinal pigment epithelium (RPE).
Solution
Alkeus Pharmaceuticals is developing orally delivered therapeutics designed to prevent or delay the progression of severe, degenerative retinal diseases. Their lead investigational therapy, Gildeuretinol, is a new molecular entity engineered to reduce the formation of toxic vitamin A dimers in the retina without disrupting the visual cycle. By addressing this underlying mechanism, Gildeuretinol aims to preserve sight in patients with Stargardt disease and geographic atrophy. Clinical studies, including the TEASE studies for Stargardt disease and the SAGA study for geographic atrophy, are focused on evaluating the safety, tolerability, and efficacy of Gildeuretinol.
Target Audience
The primary target audience includes individuals affected by Stargardt disease and geographic atrophy, as well as their families and the ophthalmologists treating these conditions.
Features
- Oral administration for convenient dosing
- Designed to reduce vitamin A dimerization without modulating the visual cycle
- Investigated for the treatment of Stargardt disease in the TEASE clinical trials
- Investigated for the treatment of geographic atrophy secondary to AMD in the SAGA clinical trial
- Has received FDA Rare Pediatric Disease and Fast Track designations for Stargardt disease