Alkeus Pharmaceuticals announced in a press release that the first patient has been dosed in the global phase 3 NORTHSTAR study of oral gildeuretinol (ALK-001) for the treatment of Stargardt disease.

The NORTHSTAR study is a randomized, placebo-controlled, double-masked 24-month trial designed to evaluate efficacy, safety, and pharmacokinetics of gildeuretinol in people living with advanced Stargardt disease and atrophic lesions at baseline. The primary endpoint is the rate of growth of atrophic lesions from months 6 to 24 comparing gildeuretinol to placebo. The key secondary endpoint is the preservation of visual acuity as measured by low luminance visual acuity (LLVA).

According to the company, fundus autofluorescence will be used to measure the growth of atrophic lesions, areas where retinal cells have been lost. Building on previously observed anatomical and functional findings across the approximately 400 patients that have received gildeuretinol to date, the company said LLVA will be used to evaluate participants’ vision in dim lighting conditions, which is expected to be more sensitive than best-corrected visual acuity and has the potential to detect changes in visual acuity when vision has started declining.  

Gildeuretinol has been well tolerated in prior studies, which have included patients as young as 8 years old and a treatment duration as long as 7 plus years. In these trials, there were no reports of chromatopsia, dark adaptation delays, or night blindness, consistent with gildeuretinol’s unique mechanism of action that does not disrupt the visual cycle.

The global phase 3 study design was agreed to by the US Food and Drug Administration (FDA) and the European Medicine Agency with an estimated 55 sites planned in more than 11 countries, the company said. OM