FDA Grants Alkeus Pharmaceuticals Rare Pediatric Disease and Fast Track Status for Gildeuretinol in Treating Stargardt Disease

Alkeus Pharmaceuticals, Inc. has made significant strides in the treatment of Stargardt disease with its investigational oral therapy, gildeuretinol (ALK-001). The U.S. Food and Drug Administration (FDA) has granted this therapy Rare Pediatric Disease and Fast Track designations, underscoring the urgent need for effective treatments for this debilitating condition, which currently has no approved therapies.

Stargardt disease leads to severe vision loss in both children and adults. The FDA’s Rare Pediatric Disease designation is given to treatments intended for serious, life-threatening diseases primarily affecting individuals from birth to 18 years old. This designation could potentially allow Alkeus to receive a priority review voucher upon approval, accelerating the development of other clinical programs. The Fast Track designation, aimed at expediting the review process for treatments addressing serious conditions with unmet medical needs, further emphasizes the promise shown by gildeuretinol.

Gildeuretinol had already received Breakthrough Therapy and Orphan Drug designations from the FDA, marking it as a significant advancement in addressing the substantial unmet medical needs of those with Stargardt disease. Alkeus Pharmaceuticals' President and CEO, Michel Dahan, highlighted these achievements as milestones that reflect the potential of gildeuretinol to become a transformative therapy for patients suffering from this condition.

Alkeus presented data from its TEASE program at the 2024 American Academy of Ophthalmology annual meeting. In particular, the TEASE-1 study, a 24-month, placebo-controlled, double-masked, randomized trial, demonstrated that gildeuretinol significantly slowed the growth rate of atrophic retinal lesions in patients with Stargardt disease by 21.6% compared to untreated patients. Additionally, a sensitivity analysis using non-transformed values showed a 29.5% reduction in lesion growth rates.

Interim results from the TEASE-3 study were also presented, indicating that early-stage Stargardt disease patients treated with gildeuretinol exhibited no disease progression and maintained stable visual acuity over periods ranging from two to six years. Dr. Christine Nichols Kay, who presented the data, emphasized the potential benefits of early intervention in preventing the onset of progressive vision loss.

Both TEASE-1 and TEASE-3 studies underscored the favorable safety and tolerability profile of gildeuretinol, with no significant adverse events related to vitamin A metabolism reported. This is particularly noteworthy given the severe vision impairment caused by Stargardt disease, which affects an estimated 30,000 to 87,000 individuals in the U.S.

The underlying cause of Stargardt disease is a defect in the ABCA4 protein, leading to the harmful accumulation of vitamin A by-products that damage the retina. Alkeus’ TEASE studies, which include four independent clinical trials (TEASE-1, TEASE-2, TEASE-3, and TEASE-4), have provided robust data supporting gildeuretinol's efficacy in reducing the progression of retinal atrophic lesions and stabilizing visual function.

In addition to Stargardt disease, gildeuretinol is also being evaluated for geographic atrophy secondary to age-related macular degeneration (AMD). A study involving 198 patients showed a trend towards reduced lesion growth and functional benefits in low luminance visual acuity.

Founded in 2010 and headquartered in Cambridge, Massachusetts, Alkeus Pharmaceuticals is committed to developing innovative therapies for serious retinal diseases. Their efforts are supported by institutional investors led by Bain Capital Life Sciences, reflecting the high level of interest and belief in the potential of their therapies to make a significant impact on patients' lives. The breakthrough-designated gildeuretinol acetate (ALK-001) continues to be a focal point of their research and development, offering hope to those affected by Stargardt disease and AMD.