Alkeus Pharmaceuticals announced gildeuretinol (ALK-001), an investigational oral therapy, has received Rare Pediatric Disease and Fast Track designations from the FDA for the treatment of Stargardt disease.

With this designation, Alkeus may be eligible to receive a priority review voucher upon approval that could be used to advance another clinical development program, the company said in a press release. Gildeuretinol has previously received Breakthrough Therapy and Orphan Drug designations from the FDA.

Data from Alkeus’ TEASE program in Stargardt disease were most recently presented during the 2024 American Academy of Ophthalmology annual meeting by Christine Nichols Kay, MD, of Vitreo Retinal Associates in Gainesville, Fla.

In TEASE-1, a placebo-controlled, double-masked, randomized 24-month study in patients with Stargardt disease, gildeuretinol slowed the growth rate of atrophic retinal lesions area (square root) by 21.6% compared to untreated patients during the 2-year study. Gildeuretinol also demonstrated a 29.5% reduction in growth rate of atrophic lesions in a sensitivity analysis using non-transformed values. The growth rates of atrophic retinal lesions were 0.18 mm/year (0.87 mm²/year untransformed area) in the gildeuretinol treated arm, and 0.23 mm/year (1.23 mm²/year) in the untreated arm, mean difference 0.05 mm/year with 95% confidence interval, 0.03 to 0.07, p<0.001. The difference was 0.36 mm²/year using non-transformed analysis with 95% confidence interval, 0.23 to 0.50, p<0.001.

In addition, Dr. Kay presented interim data from the TEASE-3 study demonstrating that early-stage Stargardt disease patients treated with gildeuretinol showed no disease progression and remained asymptomatic while on therapy for between 2 and 6 years. Gildeuretinol treatment in early-stage Stargardt patients was associated with relatively stable visual acuity.

“TEASE-1 is the first randomized, controlled trial in Stargardt disease that has shown an efficacy endpoint, which is very exciting as an inherited retinal disease specialist taking care of patients with this devastating condition,” said Dr. Kay. “In addition, the TEASE-3 data indicate the potential value of treating patients with Stargardt disease as early as possible, before onset of progressive central vision loss.”

In both TEASE-1 and TEASE-3, gildeuretinol demonstrated a favorable safety and tolerability profile. There were no adverse events related to hyper- or hypo-vitaminosis A such as xerophthalmia, chromatopsia, dark adaptation delays or night blindness.