Alkeus Pharmaceuticals shares results of SAGA study of oral gildeuretinol acetate for GA
Oral gildeuretinol demonstrated a clinically meaningful reduction in the GA lesion growth rate at 24 months in the SAGA study.
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Alkeus Pharmaceuticals, Inc. released that the SAGA study of oral gildeuretinol acetate (ALK-001) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) demonstrated a reduction of 0.25 sqmm/year vs. placebo (p=0.07) in the primary endpoint of GA lesion growth rate at 24 months. The company explained that gildeuretinol demonstrated a statistically significant reduction in the loss of low luminance visual acuity (LLVA) at 24 months (p=0.03) and showed a favorable safety profile consistent with other studies of gildeuretinol in Stargardt disease.1
The SAGA study was a 24-month, double-masked, randomized, placebo-controlled trial to designed to investigate the safety, pharmacokinetics, tolerability, and efficacy in patients with GA secondary to AMD. A total of 198 patients were enrolled in the study. The primary efficacy endpoint was the growth rate of GA lesions from baseline to 24 months as assessed by fundus autofluorescence (FAF). The first key secondary endpoint was the change in LLVA at 24 months.1
As a part of this announcement1 from the company, Seemi Khan, MD, MPH, MBA, Chief Medical Officer of Alkeus Pharmaceuticals shared his thoughts on the data and next steps for the candidate, saying, “These data clearly indicate a clinically meaningful trend in slowing the growth rate of GA lesions, which is extremely encouraging. The SAGA data represent the first clinical demonstration that slowing vitamin A dimerization could be beneficial in the treatment of GA secondary to AMD. Results from SAGA build upon the positive data from TEASE-1, a study of gildeuretinol in Stargardt disease. We look forward to discussing these results with the US Food and Drug Administration to determine the optimal path forward. We extend our gratitude to the patients, investigators, and trial sites for their participation in this study.”
According to Alkeus pharmaceuticals, it is estimated that the median time of progression to legal blindness in patients with GA is slightly over 6 years. The prevalence of GA in the United States is estimated to be over 1 million people, with 160,000 new cases occurring each year. As of publication, there is no oral therapy to treat GA approved by the FDA.1
Jeffrey S. Heier, MD, director of the vitreoretinal service and director of retina research at Ophthalmic Consultants of Boston weighed in on this study. In a quote given to Modern Retina, he said, "An oral therapy with a favorable safety profile and a clinically meaningful impact on a functional endpoint in GA would be extremely beneficial in the management of GA patients. The demonstration of a decrease in the rate of decline in low luminance visual acuity at 24 months, a key secondary endpoint, is a very substantial and promising outcome. GA is a devastating disease that can have a considerable impact on the lives of the millions of patients around the world suffering from this blinding condition. These are promising results that warrant further exploration.”
“Gildeuretinol is the first oral therapy to demonstrate a clinically meaningful impact on a functional endpoint in GA, which is extremely encouraging,” said Michel Dahan, president and CEO of Alkeus Pharmaceuticals in a quote given to Modern Retina. “These results reinforce the potential of gildeuretinol as an oral therapy for the treatment of macular degenerative diseases. We are continuing to analyze the data, and we plan to discuss the results further with regulatory agencies to determine the best path forward.”
Topline results from this study have been accepted as a late-breaking abstract at the 128th Annual Meeting of the American Academy of Ophthalmology (AAO) during Retina Subspecialty Day on Friday, October 18, 2024 in Chicago.1
