Alkeus Pharmaceuticals announces results from the SAGA study of oral gildeuretinol in patients with GA

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Alkeus Pharmaceuticals Inc. announced 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.

According to the company, gildeuretinol demonstrated a statistically significant reduction in the loss of low luminance visual acuity (LLVA) at 24 months (p=0.03). Gildeuretinolshowed a favorable safety profile consistent with other studies of gildeuretinol in Stargardt disease.

The company noted that topline results are scheduled to be presented as a late breaker at the 128th annual meeting of the American Academy of Ophthalmology (AAO) during Retina Subspecialty Day on Friday, October 18, in Chicago.

Seemi Khan, MD, MPH, MB., chief medical officer of Alkeus Pharmaceuticals, pointed out in a statement that these data indicate a clinically meaningful trend in slowing the growth rate of GA lesions.¹

“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,” he said in the statement. “We look forward to discussing these results with the U.S. 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.”

SAGA was a 24-month, double-masked, randomized, placebo-controlled trial to investigate the safety, pharmacokinetics, tolerability and efficacy in patients with GA secondary to AMD. The study enrolled 198 patients, with a primary efficacy endpoint the growth rate of GA lesions from baseline to 24 months as assessed by Fundus Autofluorescence (FAF).¹

The first key secondary endpoint was the change in LLVA at 24 months.

David S. Boyer, MD, a retina specialist with Retina-Vitreous Associates Medical Group of Los Angeles, and a principal investigator in SAGA said he was encouraged by the results of an oral treatment that showed a significant reduction of the growth rate of GA, as well as its effect on visual acuity.

“The patient population afflicted with GA is in desperate need of an oral treatment to slow disease progression,” Boyer said. “I’m extremely excited by these data and believe this is a significant advancement of our scientific understanding of the GA disease mechanism.”

GA is a progressive condition that causes irreversible loss of central vision. It is estimated that the median time of progression to legal blindness is slightly over 6 years. Currently, there is no oral therapy approved by the FDA to treat GA. Moreover, the prevalence of GA in the United States is estimated to be more than 1 million people, with 160,000 new cases being diagnosed each year.¹

Michel Dahan, president and CEO of Alkeus Pharmaceuticals, said in a statement the study results reinforce the potential of gildeuretinol as an oral therapy for the treatment of macular degenerative diseases.

“We are driven by our mission to positively impact the lives of the many people around the world living with degenerative eye diseases,” he said. “Patients, parents and children, and their treating physicians are waiting for treatment options to preserve sight. We will continue to work diligently to bring oral gildeuretinol to those in need starting with individuals impacted by Stargardt disease, subject to regulatory approval.”

According to the company, gildeuretinol acetate is a novel molecule created as a specialized form of deuterated vitamin A designed to reduce the dimerization of vitamin A without disrupting vision.

In preclinical studies, gildeuretinol decreased vitamin A dimerization down to the normal rate and prevented retinal degeneration and loss of visual function in animals with Stargardt disease.

In TEASE-1, a randomly assigned, placebo-controlled, double-masked clinical trial of gildeuretinol in late-stage Stargardt patients, gildeuretionol showed clinically and statistically significant slowing of the growth of retinal lesions over 2 years of treatment.¹

Additional clinical trials of gildeuretinol in Stargardt disease are ongoing, and ildeuretinol has received breakthrough therapy designation and orphan drug designation for Stargardt disease from the FDA.

Reference

1. Alkeus Pharmaceuticals Announces Results from the SAGA Study of Oral Gildeuretinol in Patients with Geographic Atrophy Secondary to Age-Related Macular Degeneration | Alkeus Pharmaceuticals Inc. Alkeuspharma.com. Published 2023. Accessed September 19, 2024. https://alkeuspharma.com/alkeus-pharmaceuticals-announces-results-from-the-saga-study-of-oral-gildeuretinol-in-patients-with-geographic-atrophy-secondary-to-age-related-macular-degeneration/