5 Key Takeaways

• 1

  Alkeus Pharmaceuticals has launched the global phase 3 NORTHSTAR trial for the investigational oral treatment gildeuretinol for Stargardt disease.

• 2

  The NORTHSTAR trial aims to enroll approximately 230 patients, randomized 1:1 to receive either placebo or gildeuretinol for 2 years.

• 3

  The primary endpoint of the trial will assess changes in the rate of growth of retinal atrophic lesions from month 6 to month 24.

• 4

  A key secondary endpoint will evaluate changes in low-luminance visual acuity, which showed positive results in previous phase 2 trials.

• 5

  Gildeuretinol has demonstrated significant benefits in both Stargardt disease and geographic atrophy programs in earlier studies.