Study of Alkeus' Oral Gildeuretinol Demonstrate Trend in Slowing GA Progression and Visual Function Improvement
Alkeus Pharmaceuticals announced findings from its SAGA study, which evaluated the investigational oral therapy gildeuretinol (ALK-001) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The study showed a statistically significant reduction in GA lesion growth and improvements in visual function, marking a major milestone in the treatment of this serious condition.
Patients treated with gildeuretinol experienced a 15.3% reduction in GA lesion growth rate compared to placebo between 6 and 24 months (P=0.047). Additionally, the therapy demonstrated significant improvements in low luminance visual acuity (LLVA), with treated patients losing 4.4 fewer letters on the vision chart than those on placebo (P=0.031). Although the study's primary endpoint—a 13.4% reduction in GA growth rate from baseline to 24 months—was not met with statistical significance (P=0.075), the results point to a clinically meaningful trend, Alkeus stated in a news release.
The findings were presented at the 128th Annual Meeting of the American Academy of Ophthalmology (AAO) by Alexander Melamud, MD, a retina specialist and principal investigator in the SAGA study. The trial included 198 patients and was designed to investigate the safety, pharmacokinetics, tolerability, and efficacy of gildeuretinol over 24 months.
“These data indicate a significant slowing of the growth rate of GA lesions and a reduction in visual function decline,” said Seemi Khan, MD, Chief Medical Officer of Alkeus Pharmaceuticals. “We are excited by the potential of gildeuretinol as an oral therapy, offering functional benefits in GA and potentially delaying or preventing its onset.”
In the study's secondary endpoints, gildeuretinol demonstrated a trend toward a functional benefit in best corrected visual acuity (BCVA), with 3.3 fewer letters lost compared to placebo (P=0.099). Importantly, the safety and tolerability profile of gildeuretinol was consistent with previous clinical studies in Stargardt disease, another condition the therapy is being explored for.
According to Alkeus, one notable outcome of the study was the significant reduction in the development of wet AMD. Only 11% of participants in the gildeuretinol group developed wet AMD compared to 44% in the placebo group.
“This is a significant step forward for next-generation treatments,” said Charles Wykoff, MD, Director of Research at Retina Consultants of Texas. “While the primary endpoint was not met, the consistent signal of functional preservation could greatly benefit patients with GA and earlier stages of AMD.”
“Gildeuretinol is the first oral medication to show a meaningful positive effect on low luminance visual acuity in macular degeneration,” added Dr. Melamud. “This therapy has the potential to improve quality of life by preserving low light vision, a key aspect of daily living for patients with AMD.”
The SAGA study was funded in part by the National Institutes of Health’s National Eye Institute.