Oral geographic atrophy treatment misses primary endpoint in phase 3 study

Issue: October 25, 2024

ByAlex Young

ByAnthony DeFino

Fact checked byChristine Klimanskis, ELS

Oral gildeuretinol acetate did not meet the primary endpoint of geographic atrophy lesion growth rate in a phase 3 study, according to a press release from Alkeus Pharmaceuticals.

“There is significant unmet need for an oral therapy to treat macular degenerative diseases, including GA secondary to AMD and Stargardt disease, where there are no approved oral treatments,” Seemi Khan, MD, MPH, MBA, chief medical officer of Alkeus Pharmaceuticals, told Healio. “These new data support our belief in the potential of gildeuretinol to transform the treatment landscape for patients suffering from debilitating eye diseases, such as in Stargardt and GA.”

Retina — Oral gildeuretinol acetate did not meet the primary endpoint of geographic atrophy lesion growth rate in a phase 3 study.

The SAGA study investigated ALK-001, a specialized form of deuterated vitamin A that can reduce dimerization of vitamin A without affecting vision, according to the release.

The study enrolled 198 patients with geographic atrophy (GA) secondary to age-related macular degeneration and explored the growth rate of GA lesions from baseline to 24 months as the primary efficacy endpoint. Change in low luminance visual acuity at 24 months was a secondary endpoint.

Compared with placebo, gildeuretinol demonstrated a 0.25 square millimeters per year reduction in GA lesion growth, which Alkeus called “clinically meaningful.” There was a significant reduction in the loss of low luminance visual acuity at 24 months (P = .03), according to the release.

Topline SAGA data will be presented in October at the American Academy of Ophthalmology meeting, according to the release.

“We are very encouraged by GA results and are continuing to analyze the data,” Khan said. “We are determined to bring gildeuretinol to those who need it, and we plan to discuss the SAGA data further with regulatory agencies to determine the best path forward.”

Editor’s note: This article was updated on Sept. 19, 2024, to include comments from Seemi Khan, MD, MPH, MBA.

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Press Release

Disclosures: Khan reports being the chief medical officer of Alkeus Pharmaceuticals.

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Oral geographic atrophy treatment misses primary endpoint in phase 3 study

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