Cambridge, Massachusetts — September 3, 2026
Alkeus Pharmaceuticals announced the publication of TEASE-1 efficacy and safety results in JAMA Ophthalmology, providing the first peer-reviewed report from a randomized, double-masked, placebo-controlled clinical study of investigational gildeuretinol acetate (ALK-001) in patients with Stargardt disease. The 24-month study demonstrated that daily oral gildeuretinol met its primary endpoint, significantly reducing the growth rate of retinal atrophic lesions compared with the untreated group. The findings are important for the development of a potential treatment for Stargardt disease, a rare inherited retinal disorder associated with progressive and irreversible central vision loss. The publication strengthens the clinical evidence supporting gildeuretinol as Alkeus advances its broader development program for patients with this significant unmet medical need.
Gildeuretinol Reduced Progression of Retinal Atrophy
The TEASE-1 clinical study evaluated 50 adults with Stargardt disease who had retinal atrophy at baseline, with participants receiving either daily oral gildeuretinol or an untreated comparator over 24 months. The primary analysis showed a 21.6% reduction in the transformed growth rate of retinal atrophic lesions with gildeuretinol compared with the untreated group, representing a statistically significant treatment effect. A prespecified sensitivity analysis using untransformed retinal lesion area also supported the finding, showing a 29.3% reduction in lesion growth rate. These results are particularly relevant because expansion of retinal atrophic lesions is closely associated with the progressive loss of central visual function in Stargardt disease. By slowing the rate at which these lesions develop, gildeuretinol may have the potential to slow disease progression, although its clinical benefit remains subject to confirmation in larger and later-stage studies.
Safety Profile Supports Continued Clinical Development
Alongside the efficacy findings, gildeuretinol demonstrated a favorable tolerability profile during the 24-month TEASE-1 study. Most adverse events were reported as mild or moderate, and importantly, the study reported no patient-reported delayed dark adaptation or night blindness. The safety findings are relevant because gildeuretinol is designed to address the underlying biology of retinal disease without directly modulating the visual cycle. Gildeuretinol is a next-generation oral small molecule designed to reduce vitamin A dimerization, a process associated with the formation of toxic bisretinoid compounds implicated in retinal damage. The TEASE-1 publication therefore provides both efficacy and safety evidence supporting continued investigation of the candidate as a potential long-term treatment option for people living with Stargardt disease, for which there is currently no approved therapy specifically designed to slow the progression of retinal atrophy.
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Alkeus Advances Phase 3 NORTHSTAR Study
Following the TEASE-1 findings, Alkeus is advancing gildeuretinol into the global Phase 3 NORTHSTAR study, designed to further evaluate the candidate in people with Stargardt disease. The randomized, placebo-controlled, double-masked study is planned to enroll approximately 230 participants between 8 and 45 years of age and will evaluate treatment over 24 months. The primary endpoint is the rate of retinal atrophic lesion growth from months 6 to 24, while preservation of visual acuity measured by low-luminance visual acuity is a key secondary endpoint. The Phase 3 program represents an important next step in determining whether the retinal lesion-growth reduction observed in TEASE-1 can be reproduced in a larger patient population. With the peer-reviewed TEASE-1 publication now adding clinical evidence to the program, gildeuretinol remains a closely watched investigational therapy in Stargardt disease, with future NORTHSTAR results expected to play a critical role in determining its potential path toward regulatory approval.
Source:Alkeus Pharmaceuticals press relese
