Alkeus Pharmaceuticals has announced that its oral investigational therapy, gildeuretinol acetate (ALK-001), failed to meet the primary endpoint in the SAGA phase 3 clinical trial for geographic atrophy (GA). The study investigated the efficacy of ALK-001 in reducing the growth rate of GA lesions in patients with age-related macular degeneration (AMD). While the primary endpoint was not met, the company reported a statistically significant reduction in the loss of low luminance visual acuity at 24 months.
The SAGA study enrolled 198 patients with GA secondary to AMD. The primary efficacy endpoint was the growth rate of GA lesions from baseline to 24 months. Secondary endpoints included the change in low luminance visual acuity at 24 months. Gildeuretinol is a specialized form of deuterated vitamin A designed to reduce vitamin A dimerization without affecting vision.
Key Findings
Compared to placebo, gildeuretinol demonstrated a 0.25 square millimeters per year reduction in GA lesion growth. Alkeus characterized this reduction as "clinically meaningful." Furthermore, the study showed a significant reduction in the loss of low luminance visual acuity at 24 months (P = .03).
Company Perspective
"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," said Seemi Khan, MD, MPH, MBA, chief medical officer of Alkeus Pharmaceuticals. "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."
Next Steps
Topline data from the SAGA trial are scheduled to be presented at the American Academy of Ophthalmology meeting in October. Alkeus plans to engage with regulatory agencies to discuss the SAGA data and determine the optimal path forward for gildeuretinol.
"We are very encouraged by GA results and are continuing to analyze the data," Khan added. "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."
