Promising oral GA treatment
Researchers are focused on an oral treatment for Stargardt disease. Credit: Oscar Williams

Promising oral GA treatment

November 2, 2024 Staff reporters

A study of oral gildeuretinol acetate (ALK-001) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) demonstrated a significant reduction of lesion growth rate at 24 months, said the drug’s US developer.  

 

Alkeus Pharmaceuticals’ SAGA study involved 198 patients and also demonstrated a statistically significant reduction in the loss of low luminance visual acuity at 24 months (p=0.03), along with a favourable safety profile consistent with ongoing studies of gildeuretinol in Stargardt disease. 

 

ALK-001, a novel form of deuterated vitamin A, has been shown to decrease vitamin A dimerisation to the normal rate and prevent retinal degeneration and loss of visual function in animals with Stargardt disease, said the company.  

 

SAGA’s results reinforce the potential of gildeuretinol as an oral therapy for the treatment of macular degenerative diseases, said Michel Dahan, Alkeus president and CEO. “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.”