Alkeus Pharmaceuticals has dosed the first patient in its pivotal global Phase III NORTHSTAR study, investigating oral gildeuretinol (ALK-001) for Stargardt disease.
The double-masked, placebo-controlled, randomised trial will assess the safety, efficacy, and pharmacokinetics of gildeuretinol among individuals with advanced Stargardt disease and baseline atrophic lesions.
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Stargardt disease is a rare and progressively inherited retinal disorder that results in irreversible loss of central vision in children and adults and currently has no approved treatment options.
The primary endpoint compares the growth rate of atrophic lesions from six to 24 months between gildeuretinol and placebo groups while the secondary endpoint examines preservation of visual acuity via low-luminance visual acuity (LLVA) measurement.
The trial uses fundus autofluorescence (FAF) to quantify atrophic lesion growth, measuring the extent of retinal cell loss.
LLVA is leveraged for more sensitive evaluation of participants’ vision in low light, potentially detecting changes earlier than best corrected visual acuity (BCVA).
Gildeuretinol has shown a favourable safety profile in previous studies, with no reports of chromatopsia, delayed dark adaptation, or night blindness, and treatment durations exceeding seven years.
The global Phase III design has been agreed upon by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), involving an estimated 55 sites across more than 11 countries.
Stargardt disease, driven by a defect in the ABCA4 protein, leads to accumulation of toxic vitamin A dimers and irreversible retinal damage.
Gildeuretinol targets this mechanism without altering the visual cycle and has received breakthrough therapy, rare paediatric disease, fast track and orphan drug designations from the FDA, as well as orphan medicinal product status from the EMA for related retinal conditions.
Alkeus Pharmaceuticals president and CEO Michel Dahan said: “We are tremendously excited to begin dosing in our pivotal global Phase III NORTHSTAR study evaluating oral gildeuretinol in Stargardt disease, which represents an important step forward in the development of a much-needed treatment for patients impacted by this severely debilitating disease.
“We are particularly excited about the potential of gildeuretinol to preserve visual acuity as observed in our studies to date. This Phase III programme is designed to demonstrate clinically meaningful outcomes for patients, retina specialists, and regulators that show preservation of vision.”
In July 2024, Alkeus Pharmaceuticals reported the latest interim data from the TEASE-3 trial, where gildeuretinol acetate demonstrated the ability to stall the progression of early-stage Stargardt disease.
