Belite Bio Reports Phase 3 Success for Tinlarebant in Stargardt Disease

Belite Bio has announced promising topline results from its global Phase 3 DRAGON trial, evaluating the oral investigational therapy Tinlarebant in patients with Stargardt disease type 1 (STGD1). This rare, inherited retinal disorder leads to irreversible vision loss and currently has no approved treatment. According to the company, the trial represents the first successful pivotal study in STGD1, marking a major milestone in the field of inherited retinal diseases.

Primary Endpoint Achieved: 36% Reduction in Lesion Growth

The DRAGON trial enrolled 104 patients globally and successfully met its primary efficacy endpoint. Patients treated with Tinlarebant experienced a statistically significant and clinically meaningful 36% reduction in the growth rate of retinal lesions, as measured by definitely decreased autofluorescence (DDAF) via fundus autofluorescence imaging, compared to placebo.

       • Prespecified analysis reached statistical significance (P = 0.0033)

       • Post-hoc analysis reinforced the strength of the result (P < 0.0001)

First Oral Therapy to Show Efficacy in Retinal Degeneration

“The final results from the DRAGON trial mark a historic breakthrough in Stargardt disease,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “Not only was Tinlarebant shown to slow retinal degeneration, but it’s also the first oral treatment to demonstrate clinically meaningful outcomes in a retinal degenerative disease.”

STGD1 is caused by mutations in the ABCA4 gene, resulting in toxic byproduct accumulation in the retina and leading to progressive central vision loss. Tinlarebant is now the first therapeutic candidate shown to significantly alter the disease trajectory.

Favorable Safety Profile and Stable Vision Outcomes

According to Belite Bio’s Chief Scientific Officer Dr. Nathan Mata, the reduction in lesion growth, coupled with a favorable safety profile, confirms Tinlarebant’s therapeutic mechanism and potential impact on patient care.

       • Visual acuity remained stable in both treatment and placebo groups over the 24-month period, aligning with the expected natural history of STGD1

       • Tinlarebant demonstrated strong safety and tolerability, with only four treatment-related discontinuations reported worldwide

Belite Bio plans to present additional analyses at upcoming medical conferences, pending full data evaluation.

Regulatory Strategy and Global Designations

With the DRAGON trial results in hand, Belite Bio is preparing to initiate regulatory discussions with global health authorities. The company anticipates New Drug Application (NDA) submissions in the first half of 2026.

Tinlarebant has received multiple expedited and rare disease designations to support its regulatory pathway. In the United States, it holds Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations. It has also been granted Orphan Drug Designation in the U.S., European Union, and Japan. Additionally, in Japan, Tinlarebant has been recognized with Pioneer Drug Designation, reflecting its potential to address a critical unmet medical need in Stargardt disease.