Vitamin A

Christine N. Kay, MD, presented TEASE clinical trial findings on gildeuretinol (ALK-001) for Stargardt disease at the AAO Meeting, showing a 21% reduction in atrophic lesion growth. Gildeuretinol, an oral vitamin A-derived drug, aims to slow disease progression and has FDA orphan drug and breakthrough designations, with further data expected in 2025.

Alkeus Pharmaceuticals to present results from gildeuretinol studies for geographic atrophy and Stargardt disease at AAO 2024 in Chicago.

Retinol, a retinoid from Vitamin A, is FDA-approved for treating acne and photo-aging but can cause side effects like itching, redness, and peeling, known as retinization. Consistency in use reduces these effects, and starting retinol in the 20s or 30s is recommended for anti-aging benefits.

Drugs increasing vitamin A may help treat motor neurone disease (MND), according to Scottish research. These drugs target cells activating vitamin A, potentially slowing brain deterioration. Early signs include muscle twitches, weak grip, and slurred speech. There's no cure, but treatments aim to reduce impact. The study, published in Frontiers in Neuroscience, suggests new drug targets for future therapies.

Alkeus Pharmaceuticals' TEASE-3 study shows gildeuretinol (ALK-001) halts early-stage Stargardt disease progression for up to 6 years in teenagers, marking a significant breakthrough. Gildeuretinol, a deuterated vitamin A, reduces vitamin A dimerisation, addressing the ABCA4 gene mutation causing retinal damage. The study highlights the importance of early intervention, with ongoing trials aiming to further validate these findings.

Usher Syndrome (USH), a leading cause of deaf-blindness, lacks a cure, with treatments focusing on managing symptoms. The US leads in prevalence, with 21,000 cases in 2023. Market size was ~USD 77 million, expected to grow. Luxturna is the only approved therapy for retinal degeneration. Emerging treatments like NPI-001 and Ultevursen are in trials, aiming to address the disease's root cause.