Belite Bio Continues Phase 3 DRAGON Trial for Tinlarebant in Stargardt Disease

DSMB recommends continuing Phase 3 DRAGON trial for Tinlarebant in Stargardt disease without modifications; completion expected Q4 2025.

Quiver AI Summary

Belite Bio, Inc. has announced that the independent Data Safety Monitoring Board (DSMB) has recommended the continuation of the Phase 3 DRAGON trial for Tinlarebant, an experimental treatment for adolescent Stargardt disease. The trial will proceed without modifications, maintaining its original design and a sample size of 104 subjects. This recommendation was made after a planned interim analysis demonstrated that Tinlarebant is well-tolerated and maintains a consistent safety profile. Most subjects have shown stabilized visual acuity. The trial is expected to be completed by the fourth quarter of 2025. Importantly, Tinlarebant has received various designations in the U.S., Europe, and Japan, underscoring its potential significance, especially given there are no approved treatments for Stargardt disease.

Potential Positives

• The DSMB’s decision to proceed with the trial without modifications suggests strong initial results and confidence in the trial’s methodology.
• Keeping the sample size at 104 subjects indicates that this number is adequate to demonstrate efficacy and safety for Tinlarebant.
• With designations like Fast Track, Rare Pediatric Disease, and Orphan Drug in several regions, Tinlarebant may benefit from a quicker regulatory review process.
• The anticipated completion of the trial by Q4 2025 positions the company for potential future drug approval amid a competitive biopharmaceutical market.

Potential Negatives

• The DSMB’s recommendation not to modify the trial suggests that the expected efficacy of Tinlarebant could be limited, as increasing the sample size was not necessary.
• Concerns may arise regarding the extended timeline for potential treatment availability for patients, with the trial not concluding until Q4 2025.
• The primary endpoint focuses on the growth rate of atrophic lesions, hinting that the treatment’s emphasis is on slowing disease progression rather than reversing damage, which could limit its attractiveness.

FAQ

What is the status of the Phase 3 DRAGON trial for Tinlarebant?

The independent Data Safety Monitoring Board recommends the trial continue without modifications and maintain a sample size of 104 subjects.

What designations has Tinlarebant received?

Tinlarebant has received Fast Track, Rare Pediatric Disease, and Orphan Drug Designations in various regions, along with Pioneer Drug Designation in Japan.

When is the expected completion date for the DRAGON trial?

The DRAGON trial is expected to conclude by Q4 2025, which includes a three-month follow-up period.

What is the main focus of Belite Bio?

Belite Bio is dedicated to developing novel therapeutics for degenerative retinal diseases, including Stargardt disease and Geographic Atrophy.

How is the safety profile of Tinlarebant described?

The safety profile of Tinlarebant is noted to be well-tolerated and consistent with previously reported data, according to the DSMB.

Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.

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Full Release

• An independent Data Safety Monitoring Board (DSMB) for the Phase 3 DRAGON trial has recommended trial continuation without any modifications, maintaining the sample size at 104 subjects following a planned interim efficacy analysis; trial completion is expected by Q4 2025 (including a three-month follow-up period).
• Tinlarebant has been granted Fast Track and Rare Pediatric Disease Designations in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, as well as Pioneer Drug Designation in Japan for Stargardt disease, for which no approved treatments currently exist.

SAN DIEGO, Feb. 27, 2025 (GLOBE NEWSWIRE) — Belite Bio, Inc (NASDAQ: BLTE) (“Belite” or the “Company”), a clinical-stage biopharmaceutical development company focused on innovative therapeutics for degenerative retinal diseases, has announced that the Data Safety Monitoring Board (DSMB) has recommended proceeding with the global Phase 3 “DRAGON” trial of Tinlarebant for adolescent Stargardt disease patients. This recommendation follows a pre-specified Interim Analysis after all subjects completed a one-year assessment.

The study design for the DRAGON trial included an adaptive sample size re-estimation aimed at determining the necessity of increasing sample size based on treatment effects observed. The DSMB’s recommendation suggests that no increase is necessary and that the data should be submitted for further regulatory review regarding potential drug approval.

Per the DSMB, Tinlarebant is well-tolerated, maintaining a safety profile consistent with previously reported data. The majority of subjects showed stabilized visual acuity, with a mean change from baseline of fewer than three letter scores under both standard and low luminance throughout the two-year study.

“We are pleased to have reached this significant trial milestone and are optimistic about the safety profile we continue to observe for Tinlarebant,” said Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio. “The DSMB’s recommendation to continue with the current sample size keeps us on track to complete the trial effectively and efficiently.”