Gildeuretinol Receives FDA Fast Track and Rare Pediatric Disease Designations for Stargardt Disease
Key Insights
Gildeuretinol (ALK-001) receives Rare Pediatric Disease and Fast Track designations from the FDA for Stargardt disease (search) treatment, highlighting its potential as a groundbreaking therapy.
Clinical data from the TEASE program demonstrated that gildeuretinol slowed the growth rate of atrophic retinal lesions by 21.6% compared to untreated patients.
Early-stage Stargardt disease (search) patients treated with gildeuretinol in the TEASE-3 study showed no disease progression and maintained stable visual acuity.
Alkeus Pharmaceuticals (search)' gildeuretinol (ALK-001), an investigational oral therapy, has been granted Rare Pediatric Disease and Fast Track designations by the U.S. Food and Drug Administration (FDA) for the treatment of Stargardt disease (search). These designations highlight the potential of gildeuretinol to address the significant unmet medical need in this rare and progressive retinal disease affecting both children and adults.
Michel Dahan, President and CEO of Alkeus Pharmaceuticals (search), emphasized the importance of these milestones, noting the absence of approved treatments for Stargardt disease (search). The FDA's Rare Pediatric Disease designation may allow Alkeus to receive a priority review voucher (PRV) upon approval, potentially expediting the development of other clinical programs. The Fast Track designation will facilitate the development and accelerate the review process for gildeuretinol, which has already been granted Breakthrough Therapy and Orphan Drug designations.
Clinical Trial Data
Data from Alkeus’ TEASE program were presented at the 2024 American Academy of Ophthalmology annual meeting by Dr. Christine Nichols Kay. The TEASE-1 study, a 24-month, placebo-controlled, double-masked, randomized trial, demonstrated that gildeuretinol slowed the growth rate of atrophic retinal lesions by 21.6% compared to the untreated group (p<0.001). Specifically, the growth rates of atrophic retinal lesions were 0.18 mm/year in the gildeuretinol arm and 0.23 mm/year in the untreated arm.
Interim data from the TEASE-3 study indicated that early-stage Stargardt disease (search) patients treated with gildeuretinol showed no disease progression and remained asymptomatic for two to six years. Furthermore, gildeuretinol treatment in these patients was associated with relatively stable visual acuity.
Dr. Kay stated that TEASE-1 is the first randomized, controlled trial in Stargardt disease (search) to demonstrate an efficacy endpoint. She also highlighted the potential benefit of early intervention with gildeuretinol to prevent progressive central vision loss.
Safety and Tolerability
Gildeuretinol demonstrated a favorable safety and tolerability profile in both TEASE-1 and TEASE-3 studies. Notably, there were no adverse events related to hyper- or hypo-vitaminosis A, such as xerophthalmia, chromatopsia, dark adaptation delays, or night blindness.
About Stargardt Disease
Stargardt disease (search) is a leading cause of severe vision impairment in children and young adults, affecting an estimated 30,000 to 87,000 individuals in the U.S. The disease is characterized by a defect in the ABCA4 protein (search), leading to the accelerated dimerization of vitamin A and the formation of toxic by-products that damage the retina, resulting in progressive vision loss. Currently, there is no approved treatment for Stargardt disease.
About the TEASE Program
The TEASE program consists of four independent clinical studies evaluating oral gildeuretinol (ALK-001) in Stargardt disease (search): TEASE-1, TEASE-2, TEASE-3, and TEASE-4. TEASE-1 met its primary efficacy endpoint, showing a statistically significant reduction in the growth rate of retinal atrophic lesions. TEASE-2 is an ongoing, fully enrolled trial with topline data expected in 2025. TEASE-3 is an open-label study in early-stage Stargardt disease patients, and TEASE-4 is an open-label extension study.
About Gildeuretinol Acetate (ALK-001)
Gildeuretinol acetate (ALK-001) is designed to reduce the dimerization of vitamin A without affecting the visual cycle. Preclinical studies have shown that gildeuretinol can decrease vitamin A dimerization and prevent retinal degeneration in animal models of Stargardt disease (search). In addition to the TEASE program, gildeuretinol is being evaluated in the SAGA study for geographic atrophy (search) (GA) secondary to age-related macular degeneration (search) (AMD).