Full Press Release Details
Belite Bio Announces
Interim Analysis Results from the Pivotal Global Phase 3 DRAGON trial of Tinlarebant in Adolescent Stargardt Disease Subjects
26, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) ("Belite" or the "Company"), a clinical-stage biopharmaceutical
drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet
medical need today announced that following a pre-specified Interim Analysis of the pivotal global Phase 3 "DRAGON" trial
data of Tinlarebant in adolescent Stargardt disease patients, the Data Safety Monitoring Board (DSMB) has recommended the trial proceed
without any modifications. The Interim Analysis was performed when all subjects completed the one-year assessment.
for the DRAGON trial included an adaptive sample size re-estimation that would determine the need for an increase in sample size in order
to enhance power, based on a treatment effect observed at the Interim Analysis. The recommendation by the DSMB that the trial should
proceed without modifications indicates that a sample size increase is not warranted. In addition, the DSMB recommended to submit the
data for further regulatory review for drug approval.
DSMB, Tinlarebant is well-tolerated and the safety profile remains consistent with previously observed data and the mechanism of action
for Tinlarebant. In addition, visual acuity was stabilized in the majority of subjects, with mean change from baseline of less than three
letter scores under both standard and low luminance, throughout the two-year study.
pleased to have reached this important trial milestone and are excited by the safety profile that we continue to observe for Tinlarebant,"
said Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio. "Following the DSMB's recommendation to continue the trial
with the current sample size, we remain on track to complete the trial by Q4 2025, including a three-month follow-up period, and look
forward to building on the promising efficacy results observed in our completed Phase 2 trial for Tinlarebant."
3 DRAGON trial is a randomized, double-masked, placebo-controlled, global and multi-center study, designed to evaluate the safety and
efficacy of Tinlarebant in adolescent Stargardt disease patients. The DRAGON trial has sites in 11 jurisdictions, including the U.S.,
the United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia. The study enrolled 104
subjects with a 2:1 randomization (active:placebo). The primary efficacy endpoint is the growth rate of atrophic lesion, along with the
assessment of safety and tolerability of Tinlarebant. Tinlarebant has been granted Orphan Drug Designation in the United States, Europe,
and Japan, Rare Pediatric Disease (RPD) designation and Fast Track Designation in the U.S., and Sakigake (Pioneer Drug) Designation in
Date: Thursday, February 27, 2025
Time: 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time)
Webcast Link Instructions
You can join the live webcast by visiting
the link above or the "Presentations & Events" section of the Company's Investor Relations website at https://investors.belitebio.com/presentations-events/events.
A replay will be available following the event.
clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases
that have significant unmet medical needs, such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related
macular degeneration (AMD), in addition to specific metabolic diseases. Belite's lead candidate, Tinlarebant, an oral therapy intended
to reduce the accumulation of toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON
visit us at www.belitebio.com.
Regarding Forward Looking Statements
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,
interim analysis and recommendation from DSMB; Belite Bio's advancement of, and anticipated future activities on preclinical studies,
clinical development, regulatory milestones, and commercialization of its product candidates; and any other statements containing the
words "expect", "hope", "indicate", "look forward to", 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 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.
Wu /ir@belitebio.com
Julie Fallon /belite@argotpartners.com