Alkeus Pharmaceuticals Announces Positive Results from SAGA Study of Gildeuretinol Acetate in GA Patients
Alkeus Pharmaceuticals announced results from the SAGA study, a phase 3 clinical trial of oral gildeuretinol acetate (ALK-001) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The study demonstrated a reduction in the growth rate of GA lesions by 0.25 square millimeters per year compared to placebo (P=0.07), signaling a positive trend toward slowing disease progression. Additionally, gildeuretinol significantly reduced the loss of low luminance visual acuity (LLVA) at 24 months (P=0.03), showcasing its potential to preserve vision in GA patients.
The results will be presented as a late-breaking abstract at the the American Academy of Ophthalmology (AAO) meeting during Retina Subspecialty Day on October 18 in Chicago.
“These data clearly indicate a clinically meaningful trend in slowing the growth rate of GA lesions, which is extremely encouraging,” Seemi Khan, MD, MPH, Chief Medical Officer of Alkeus Pharmaceuticals, said in a company news release. “The SAGA data represent the first clinical demonstration that slowing vitamin A dimerization could be beneficial in treating GA secondary to AMD. We are eager to discuss these findings with the U.S. Food and Drug Administration to determine the optimal path forward.”
The SAGA study was a 24-month, double-masked, randomized, placebo-controlled trial involving 198 patients. The primary efficacy endpoint was the growth rate of GA lesions as assessed by Fundus Autofluorescence (FAF), while the key secondary endpoint focused on changes in LLVA over 24 months. Gildeuretinol exhibited a favorable safety profile, consistent with previous studies of the drug in patients with Stargardt disease.
“These results reinforce the potential of gildeuretinol as an oral therapy for the treatment of macular degenerative diseases,” said Michel Dahan, President and CEO of Alkeus Pharmaceuticals. “We are committed to bringing oral gildeuretinol to patients in need, starting with those affected by Stargardt disease, pending regulatory approval.”
Gildeuretinol acetate (ALK-001) is a novel, deuterated form of vitamin A designed to reduce the dimerization of vitamin A without disrupting visual function. Gildeuretinol has received breakthrough therapy and orphan drug designations from the FDA for Stargardt disease. The SAGA study’s positive results provide additional support for its potential in treating GA secondary to AMD, with the full topline data set to be revealed at the AAO meeting in October.