Alkeus Secures Rare Pediatric Disease and Fast Track FDA Designations for Gildeuretinol in Stargardt Disease

Alkeus Pharmaceuticals announced that its investigational oral therapy, gildeuretinol (ALK-001), has been granted Rare Pediatric Disease and Fast Track designations by the FDA for the treatment of Stargardt disease.

“Stargardt disease imposes a relentless burden, robbing children and adults of their sight,” Michel Dahan, President and CEO of Alkeus Pharmaceuticals, said in a company news release. “The FDA’s recognition of gildeuretinol’s potential through these designations underscores the pressing need for an effective treatment. This achievement builds on the previously awarded Breakthrough Therapy and Orphan Drug designations, further highlighting the unmet medical need in Stargardt disease and the life-changing potential of gildeuretinol.”

View EyewireTV's recent interview with Mr. Dahan here.

The announcement follows compelling data from Alkeus’ TEASE program, a series of clinical studies evaluating gildeuretinol’s safety and efficacy. Presented at the 2024 American Academy of Ophthalmology annual meeting, results from the TEASE-1 trial demonstrated that gildeuretinol significantly slowed the growth of retinal lesions in Stargardt disease patients over two years.

Key findings from TEASE-1 include:

• A 21.6% reduction in the growth rate of atrophic retinal lesions compared to untreated patients
• A 29.5% reduction when analyzing non-transformed data
• Stabilized visual acuity in early-stage Stargardt disease patients, as shown in interim results from the TEASE-3 study

“TEASE-1 marks the first randomized, controlled trial to show an efficacy endpoint in Stargardt disease,” said Christine Nichols Kay, MD, of Vitreo Retinal Associates in Gainesville, Florida. “The results highlight the importance of early treatment, as seen in the TEASE-3 data, where early-stage patients treated with gildeuretinol experienced no disease progression.”

Rare Pediatric Disease designation provides eligibility for a priority review voucher (PRV) upon drug approval, an incentive that could expedite the development of additional treatments. Fast Track designation facilitates quicker FDA reviews, aiming to bring therapies addressing serious unmet needs to patients sooner.

Stargardt disease, affecting an estimated 30,000 to 87,000 individuals in the US, is caused by defects in the ABCA4 protein, leading to toxic vitamin A by-products that damage the retina. Gildeuretinol is a novel therapy designed to reduce vitamin A dimerization without altering the visual cycle. In clinical and preclinical studies, it demonstrated significant slowing of retinal degeneration and maintained visual function.

Alkeus continues to advance gildeuretinol through its TEASE program, with additional trials underway to confirm its efficacy and safety across different stages of Stargardt disease.