Belite Bio Announces China NMPA Agrees to New Drug Application with Priority Review based on Interim Analysis Results for the Treatment of Stargardt Disease with Tinlarebant

Rhea-AI Summary

Belite Bio (NASDAQ: BLTE) announced that China’s NMPA agreed to accept a New Drug Application with priority review for Tinlarebant to treat Stargardt disease based on interim results from the Phase 3 DRAGON trial.

The interim analysis showed statistical significance on the trial’s primary endpoint (growth rate of atrophic lesions). The global, randomized 2:1 DRAGON trial enrolled 104 subjects across 11 jurisdictions. Final topline data are expected in Q4 2025, and the NDA package is being prepared for submission to China CDE per guidance.

Positive

• NMPA accepted NDA for priority review based on interim Phase 3 data
• Interim analysis showed statistical significance on the primary endpoint
• DRAGON trial enrolled 104 subjects across 11 jurisdictions
• Final topline data targeted for Q4 2025

Negative

• Final topline outcome remains pending in Q4 2025
• NDA is currently under preparation and not yet submitted to CDE

Insights

Clinical development expert

NMPA acceptance with priority review based on a statistically significant Phase 3 interim endpoint is a meaningful clinical milestone.

Belite reported that China’s NMPA agreed to accept an NDA with priority review for Tinlarebant based on the Phase 3 DRAGON interim analysis that showed statistical significance on the primary endpoint: growth rate of atrophic lesions. The pivotal trial is randomized, double-masked, placebo-controlled and enrolled 104 subjects across 11 jurisdictions with a 2:1 randomization, focused on adolescent patients with Stargardt disease.

The primary dependencies are explicit: the final topline data expected in Q4 2025 and the full NDA package being prepared per China CDE guidance. The interim positive result reduces one clinical risk, but the program still depends on confirmatory final data and the completeness of the submission package. Watch for the Q4 2025 topline and the regulatory filing completeness in the weeks that follow; those items will materially change regulatory odds and clinical confidence within months.

Regulatory and commercial expert

Priority review acceptance by the NMPA accelerates regulatory timelines in China and raises near-term value drivers.

The NMPA’s agreement to accept an NDA with priority review signals a regulatory pathway in China based on the interim Phase 3 DRAGON data, which the company says met statistical significance for the trial’s primary endpoint. The submission route relies on the interim analysis plus the planned final topline data; the company explicitly targets final results in Q4 2025 and is preparing the NDA per China CDE guidance.

Key risks and near-term catalysts are clear: the final topline in Q4 2025 and the actual content and timing of the NMPA submission. These milestones will determine review timing and potential approval prospects in China over the following months. Monitor the final DRAGON readout and the company’s NDA filing updates as the primary near-term signals of regulatory progress.

• NMPA’s response is based on the Phase 3 DRAGON interim analysis results
• Topline final data expected in Q4 2025
  
  

SAN DIEGO, Oct. 15, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc. (NASDAQ: BLTE), a clinical-stage drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that the Center for Drug Evaluation of China’s National Medical Products Administration (“NMPA”) has agreed to accept the New Drug Application (NDA) with priority review for Tinlarebant for the treatment of Stargardt disease based on the interim analysis results from the Phase 3 DRAGON trial.

“Having China NMPA agreed to review the NDA based on interim Phase 3 data is a remarkable milestone for Belite Bio and the Stargardt community,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “This milestone underscores the strength of the program and the urgent need for therapies in this devastating disease, where no approved treatment options exist. This achievement positions Belite Bio to advance Tinlarebant through the final stages of development and, if successful, bring the first treatment to people living with Stargardt disease.”

NMPA’s response is based on the interim analysis results showing statistical significance in the primary endpoint of the Phase 3 DRAGON trial. The Company remains on track to report final topline data from the Phase 3 DRAGON trial in the fourth quarter of 2025. These results are expected to be submitted to the NMPA as part of the NDA that is currently under preparation in accordance with China CDE’s guidance.

The pivotal Phase 3 DRAGON trial is a randomized, double-masked, placebo-controlled, global study designed to evaluate the safety and efficacy of Tinlarebant in adolescent patients with Stargardt disease. The trial enrolled 104 subjects across 11 jurisdictions, including the U.S., United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia, with a 2:1 randomization (Tinlarebant:placebo). The primary efficacy endpoint is the growth rate of atrophic lesions, alongside the assessment of safety and tolerability.

About Tinlarebant (a/k/a LBS-008)
Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in geographic atrophy (GA), or advanced dry age-related macular degeneration (AMD). Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Breakthrough Therapy Designation, Fast Track Designation and Rare Pediatric Disease designation in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and Sakigake (Pioneer Drug) Designation in Japan for the treatment of STGD1.

About Belite Bio
Belite Bio is a clinical-stage drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical need, such as STGD1 and GA in advanced dry AMD, in addition to specific metabolic diseases. Belite’s lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of bisretinoid toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects and a Phase 3 study (PHOENIX) in subjects with GA. For more information, follow us on X, Instagram, LinkedIn, and Facebook or visit us at www.belitebio.com.

Important Cautions Regarding Forward Looking Statements
This press release contains forward-looking statements about future expectations and plans, as well as other statements regarding matters that are not historical facts. These statements include but are not limited to statements regarding the potential implications of clinical data for patients, and Belite Bio’s advancement of, and anticipated preclinical activities, clinical development, regulatory milestones, and commercialization of its product candidates, the ability of Tinlarebant to treat Stargardt disease and geographic atrophy, and any other statements containing the words “expect”, “hope” and similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including but not limited to Belite Bio’s ability to demonstrate the safety and efficacy of its drug candidates; the clinical results for its drug candidates, which may not support further development or regulatory approval; the timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; the timing to submit trial data to regulatory authorities for drug approval; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio’s drug candidates; the potential efficacy of Tinlarebant, as well as those risks more fully discussed in the “Risk Factors” section in Belite Bio’s filings with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Belite Bio, and Belite Bio undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Media and Investor Relations Contact:
Jennifer Wu
ir@belitebio.com

Julie Fallon
belite@argotpartners.com

FAQ

What did Belite Bio announce about Tinlarebant and the NMPA on October 15, 2025?

China’s NMPA agreed to accept a New Drug Application with priority review for Tinlarebant based on interim Phase 3 DRAGON results.

What did the Phase 3 DRAGON interim analysis show for BLTE Tinlarebant?

The interim analysis reported statistical significance on the primary endpoint: growth rate of atrophic lesions.

When will Belite Bio report final DRAGON topline data for BLTE?

The company expects to report final topline data in Q4 2025.

How many patients and where were enrolled in the DRAGON trial for BLTE?

The randomized DRAGON trial enrolled 104 subjects across 11 jurisdictions including the U.S., U.K., Germany, France, China, and Australia.

What is the DRAGON trial design for Tinlarebant (BLTE)?

DRAGON is a randomized, double-masked, placebo-controlled global Phase 3 study with 2:1 randomization (Tinlarebant:placebo) in adolescent patients with Stargardt disease.

Does NMPA acceptance mean Tinlarebant is approved in China for BLTE?

No; NMPA agreed to review the NDA with priority based on interim data, but final approval depends on the complete review and final topline results.