Belite Bio Announces Landmark Phase 3 Success for Stargardt Disease

In what experts are calling a “historic breakthrough” for inherited eye diseases, Belite Bio today reported positive topline results from its global Phase 3 DRAGON trial of Tinlarebant, the first therapy to show clinical benefit for Stargardt disease in a pivotal study.

Stargardt disease, a rare genetic condition that causes progressive vision loss, often beginning in youth, currently has no approved treatment. The condition affects an estimated 50,000 people in the United States.

Belite Bio’s DRAGON trial enrolled 104 adolescents with Stargardt disease type 1 (STGD1) across multiple international centers. According to the company, Tinlarebant met its primary endpoint with a statistically significant 36% reduction in the growth rate of retinal lesions compared with placebo (p=0.0033), marking the first time any therapeutic candidate has achieved efficacy in a global Phase 3 trial for STGD1.

A post-hoc analysis using an alternate statistical model showed consistent results, with significance strengthened to p<0.0001.

“This marks a pivotal moment for families affected by Stargardt disease,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “Tinlarebant not only slowed retinal degeneration, but did so as an oral therapy, something never before demonstrated in a retinal degenerative disease.”

Investigators reported that Tinlarebant also achieved statistically significant lesion-reduction effects in both eyes for the primary and key secondary endpoints. Visual acuity remained largely stable across both treatment and placebo groups, a pattern consistent with the condition’s natural history over 24 months.

Safety data were also encouraging. The company said Tinlarebant was generally well tolerated, with only four treatment-related discontinuations and mainly mild ocular side effects such as xanthopsia and delayed dark adaptation. Headaches were the most common non-ocular treatment-related adverse event.

Leading ophthalmologists who participated in the study emphasized the magnitude of the findings.

“Seeing well-controlled Phase 3 data with such a clear slowing of lesion growth is deeply encouraging,” said Professor Michel Michaelides of Moorfields Eye Hospital in London. “It suggests we may finally be nearing an approved treatment option for this devastating disease.”

Dr. Quan Dong Nguyen of Stanford University added that slowing lesion progression could eventually translate into preserved visual function: “It is remarkable to recognize that with these robust results, we may soon have the first available therapy for Stargardt disease.”

Tinlarebant works by reducing levels of retinol-binding protein 4 (RBP4), thereby limiting the retinal accumulation of toxic vitamin A by-products implicated in STGD1 and other degenerative conditions. The trial confirmed that the 5 mg dose reduced RBP4 by roughly 80% during treatment.

Belite Bio plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration in the first half of 2026. Tinlarebant has already received multiple regulatory designations, including Breakthrough Therapy and Orphan Drug status in the U.S., Europe, and Japan.

The company will discuss the Phase 3 results during a webcast today at 8:00 a.m. ET.

If approved, Tinlarebant would become the first therapy for Stargardt disease