Gildeuretinol for Stargardt disease receives Rare Pediatric Disease, Fast Track Designations

Gildeuretinol for Stargardt disease receives Rare Pediatric Disease, Fast Track Designations | Image Credit: © Araki Illustrations - © Araki Illustrations - stock.adobe.com.

Gildeuretinol (ALK-001; Alkeus Pharmaceuticals) has received Rare Pediatric Disease and Fast Track Designations from the FDA for the treatment of Stargardt disease, according to an announcement from Alkeus Pharmaceuticals.

The investigational oral therapy is a new chemical entity designed to reduce the dimerization of vitamin A without modulating the visual cycle, according to the pharmaceutical company. The disease can result in severe vision impairment in children and young adults, with no treatments currently approved. It is estimated that between 30,000 and 87,000 people in the United States are affected, stated Alkeus.

The investigational therapy has been, and is currently being studied in the Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) clinical program, comprised of 4 independent studies of oral gildeuretinol to treat Stargardt disease: TEASE-1, TEASE-2, TEASE-3 and TEASE-4.

In TEASE-1, a randomized, double-masked, placebo-controlled trial in 50 patients with Stargardt disease, the oral therapy met the primary endpoint, demonstrating a 21.6% reduction in the growth rate of retinal atrophic leasions area (square root) (P < 0.001), along with a 29.5% reduction for untransformed areas of retinal atrophic lesions against untreated patients, according to Alkeus.

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; 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), according to study results.

TEASE-2 is an ongoing and fully enrolled randomized, double-masked, placebo-controlled trial in 80 patients with moderate disease state. Data is expected in 2025, stated the pharmaceutical company.

"TEASE-3, the first clinical trial in early-stage Stargardt disease, is an open-label study of gildeuretinol in genetically confirmed patients with early signs of disease visible on retinal imaging, but who have not begun experiencing symptoms of vision loss," wrote Alkeus in the FDA Designation announcement.

“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,” Christine Nichols Kay, MD, Vitreo Retinal Associates, Gainesville, Florida, said in a statement.

Kay presented data from the TEASE program at the 2024 American Academy of Ophthalmology annual meeting in October.

“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," said Kay.

“Receiving both the FDA’s Rare Pediatric Disease and Fast Track designations are important milestones for Alkeus that highlight the potential for oral gildeuretinol to be a groundbreaking therapy for patients," added Michel Dahan, president and CEO of Alkeus Pharmaceuticals.

"These designations were granted on top of the previously awarded Breakthrough Therapy and Orphan Drug designations. Together, these achievements recognize the significant unmet medical need in Stargardt disease and the overwhelming burden on patients as well as their families and caregivers," said Dahan.

Reference:

Alkeus Pharmaceuticals Receives FDA Rare Pediatric Disease and Fast Track Designations for Gildeuretinol as a Treatment for Stargardt Disease. Alkeus Pharmaceuticals. Press release. November 18, 2024. Accessed November 22, 2024. https://www.globenewswire.com/news-release/2024/11/18/2982878/0/en/Alkeus-Pharmaceuticals-Receives-FDA-Rare-Pediatric-Disease-and-Fast-Track-Designations-for-Gildeuretinol-as-a-Treatment-for-Stargardt-Disease.html