Alkeus announced the publication in JAMA Ophthalmology of TEASE-1 efficacy and safety results for oral Gildeuretinol in Stargardt disease.
The randomized, double-masked, placebo-controlled TEASE-1 trial enrolled 50 patients aged 18 to 60 with retinal atrophy at baseline and reported that daily oral Gildeuretinol met the study’s primary endpoint, producing a 21.6% reduction in the transformed (square root) growth rate of retinal atrophic lesions versus the untreated group (0.182 mm/year versus 0.232 mm/year; mean difference -0.050 mm/year; 95% CI, -0.072 to -0.027; p<0.001).
A prespecified sensitivity analysis using untransformed lesion area showed a 29.3% reduction in lesion growth (0.867 mm2/year versus 1.230 mm2/year; mean difference -0.363 mm2/year; 95% CI, -0.498 to -0.228; p<0.001).
The company claims Gildeuretinol was well tolerated through 24 months, with most adverse events mild or moderate and no patient-reported delayed dark adaptation or night blindness in TEASE-1.
Alkeus is enrolling the global Phase 3 NORTHSTAR trial, aiming to enroll approximately 230 participants aged 8 to 45 to evaluate atrophic lesion growth from months 6 to 24, with low-luminance visual acuity as a key secondary endpoint.
