Alkeus Pharmaceuticals has announced encouraging results from its Phase 2/3 SAGA study evaluating the effects of gildeuretinol acetate (ALK-001) in patients with geographic atrophy (GA). The study found that ALK-001, a modified form of vitamin A, demonstrated a clinically meaningful reduction in GA lesion growth and a significant improvement in low luminance visual acuity (LLVA), a key secondary endpoint, over 24 months.

SAGA was a double-masked, randomized, placebo-controlled trial to investigate the safety, pharmacokinetics, tolerability, and efficacy of ALK-001 in 198 enrolled patients with GA secondary to AMD. The primary efficacy endpoint was the growth rate of GA lesions from baseline to 24 months as assessed by fundus autofluorescence (FAF). Patients taking the oral therapy once daily demonstrated a reduction of 0.25 mm2/year in lesion growth vs placebo (P=0.07).

Topline data from the SAGA study will be presented as a late-breaking abstract at the annual meeting of the AAO, Oct. 18-21 in Chicago, the company said in press release.

Gildeuretinol acetate is a specialized form of deuterated vitamin A designed to reduce the dimerization of vitamin A without disrupting vision. In addition to SAGA, the drug is also being tested in patients with Stargardt disease in 4 TEASE studies. At the American Society of Retina Specialists meeting in Stockholm, Sweden, in July, the company presented data from TEASE-3 that indicated ALK-001 delayed photoreceptor loss and vision loss in Stargardt patients. Gildeuretinol has received breakthrough therapy designation and orphan drug designation by the FDA.