Key Takeaways

  • Alkeus Pharmaceuticals has dosed the first patient in its global phase 3 NORTHSTAR trial evaluating oral gildeuretinol (ALK-001) for Stargardt disease
  • The 24-month, placebo-controlled study will assess whether gildeuretinol can slow the growth of atrophic retinal lesions and preserve visual acuity
  • The FDA- and EMA-aligned trial is expected to enroll patients at approximately 55 sites across more than 11 countries

Alkeus Pharmaceuticals has dosed the first patient in its global phase 3 NORTHSTAR study evaluating oral gildeuretinol (ALK-001) for Stargardt disease. The first participant was enrolled and dosed at Erie Retina Research in Pennsylvania.

The NORTHSTAR trial is designed to assess the efficacy, safety and pharmacokinetics of gildeuretinol in patients with advanced Stargardt disease.

“We are tremendously excited to begin dosing in our pivotal global phase 3 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,” said Michel Dahan, President and CEO of Alkeus Pharmaceuticals. “We are particularly excited about the potential of gildeuretinol to preserve visual acuity as observed in our studies to date.”

The randomized, placebo-controlled, double-masked 24-month study (NCT07419334) will evaluate patients with advanced Stargardt disease who have atrophic retinal lesions at baseline. The trial’s primary endpoint is the rate of growth of atrophic lesions between months 6 and 24, while the key secondary endpoint is preservation of visual acuity as measured by low luminance visual acuity (LLVA).

According to the company, the phase 3 study design was developed in consultation with both the FDA and the European Medicines Agency (EMA). Approximately 55 sites across more than 11 countries are expected to participate in the global trial.

“There remains a significant unmet need in Stargardt disease, and continued progress in research is important for patients and families affected by this condition,” said David R.P. Almeida, MD, principal investigator at Erie Retina Research. “This study demonstrates a shared commitment to better understand the disease and to evaluate gildeuretinol’s potential as a treatment for Stargardt disease.”

The NORTHSTAR study will use fundus autofluorescence (FAF) imaging to measure the progression of atrophic lesions, which reflect retinal cell loss. Investigators will also assess LLVA, a measure of visual function under low-light conditions that may be more sensitive than traditional best-corrected visual acuity testing in detecting early declines in vision.

Alkeus reported that approximately 400 patients have received gildeuretinol across clinical studies to date. The company said the investigational therapy has demonstrated a favorable tolerability profile, including treatment durations exceeding 7 years and use in patients as young as 8 years old. No cases of chromatopsia, delayed dark adaptation or night blindness have been reported, findings that Alkeus attributes to the drug’s mechanism of action, which does not disrupt the visual cycle.