Conference Presentation

Alkeus to present Phase 2 TEASE gildeuretinol data alongside Phase 3 design at ASRS

The July 18 presentation covers a Phase 2 tolerability study built around a safety endpoint, with Alkeus using the session to preview the Phase 3 NORTHSTAR design that will test disease-modifying efficacy.

By AppliedXLJuly 14, 2026Trial NCT02402660

Executive Summary

Alkeus will use an oral session at a major retina meeting to disclose safety and tolerability findings from a long-running Phase 2 study and to lay out the design of its Phase 3 program.
The Phase 2 study was built to characterize tolerability over 24 months of dosing, not to establish whether the drug slows disease progression, so the presentation speaks to safety, and the efficacy case rests with the trial that follows.
Gildeuretinol has no direct competitor sharing its retinoic acid receptor mechanism in Stargardt disease, so this program is effectively defining the mechanism's clinical profile on its own rather than replicating a validated class result.
The drug already holds Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug status for Stargardt disease, designations that reflect unmet need and review support rather than a demonstrated efficacy result.
The efficacy question that matters to the mechanism's future has moved to the Phase 3 program, which is enrolling toward a primary endpoint built on lesion growth and visual acuity preservation.

The presentation

Alkeus Pharmaceuticals will deliver an oral presentation titled "Oral Gildeuretinol for Slowing Stargardt Disease Progression: Safety and Efficacy Data From the Phase 2 TEASE Study and Phase 3 Study Design" at the ASRS meeting in Montreal on July 18, 2026. Christine Kay, M.D., of Vitreo Retinal Associates will present, with a companion recorded presentation on TEASE baseline characteristics from Kenneth Fan, M.D., of Retina Consultants of Texas available on demand. The session sits within a panel on Stargardt disease therapies, biomarkers, and trial design. Alkeus

What TEASE was designed to answer

TEASE, registered as NCT02402660, is a Phase 2 study whose primary outcome measure is the incidence and clinical significance of ocular and non-ocular adverse events over 24 months of daily ALK-001 dosing, a tolerability endpoint rather than an efficacy one. The trial has run since it started in August 2015, has moved to Enrolling by invitation status, and lists an anticipated enrollment of 160 participants, up from 140 at its last registry update in April 2025. Its primary completion date has moved from December 2021 to December 2024 to June 2026 over the trial's history. No results have been posted to ClinicalTrials.gov for the study. NCT02402660

The mechanism and the field

Gildeuretinol acts on retinoic acid receptor (RAR) signaling and is classified as a gene transcription modulator. Within Stargardt disease, only one other industry trial studies the RAR target, and that is Alkeus's own Phase 3 NORTHSTAR study (NCT07419334), a randomized, placebo-controlled, double-masked 24-month trial enrolling roughly 230 participants with advanced disease and a primary endpoint measuring the rate of atrophic lesion growth from months 6 to 24. The broader Stargardt disease field is dominated by gene-therapy and small-molecule programs targeting different mechanisms, including tinlarebant (RBP4), emixustat (RPE65), and several ABCA4-directed gene therapies, none of which shares gildeuretinol's RAR mechanism. NCT02402660+1

Regulatory standing

Gildeuretinol carries Breakthrough Therapy, Rare Pediatric Disease, Fast Track, and Orphan Drug designations from the FDA for Stargardt disease, and an orphan designation from the European Medicines Agency for ABCA4-related inherited retinal dystrophies. These designations reflect the severity of unmet need in the disease and the FDA's willingness to engage more closely on development, not a demonstrated efficacy outcome. Alkeus

What's ahead

Alkeus has already dosed its first patient in NORTHSTAR, the Phase 3 study that will carry the efficacy claim forward, with the July 18 session used to walk investigators and clinicians through that design alongside the TEASE tolerability record. NORTHSTAR's completion is targeted for October 2029. Alkeus

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