Belite Bio Announces First Patient Dosed in Phase 2/3 DRAGON II Trial of Tinlarebant for the Treatment of Stargardt Disease

• Phase 2/3 of the DRAGON II trial will evaluate the efficacy, safety, and tolerability of Tinlarebant in approximately 60 adolescent STGD1 subjects across the U.S., U.K., and Japan
• Phase 1b of DRAGON II trial to evaluate pharmacokinetics and pharmacodynamics of Tinlarebant in Japanese subjects was recently completed
• Tinlarebant granted Orphan Drug and Sakigake (Pioneer Drug) Designation in Japan for the treatment of STGD1
• Pivotal global Phase 3 trial of Tinlarebant in GA subjects (“PHOENIX”) is ongoing and more than 200 subjects have been enrolled

SAN DIEGO, Sept. 10, 2024 (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 needs, today announced that the first patient has been dosed at the Tokyo Medical Center in the Phase 2/3 portion of its DRAGON II clinical trial evaluating Tinlarebant for the treatment of STGD1. Tinlarebant is a novel oral therapy designed to reduce the accumulation of vitamin A based toxins that cause retinal disease, and this Phase 2/3 study will evaluate the efficacy, safety, and tolerability of Tinlarebant in approximately 60 adolescent STGD1 subjects across the U.S., U.K., and Japan.

“We are pleased to have successfully dosed the first subject in the Phase 2/3 portion of our DRAGON II trial. This milestone is a significant step forward in our mission to address the unmet needs of people living with Stargardt Disease,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “We recently completed a Phase 1b study of Tinlarebant in STGD1 patients at the Tokyo Medical Center and are happy to be continuing to partner with them for this next trial. With enrollment now also underway at sites in the U.S. and U.K., we are making meaningful progress toward our goal of delivering a life-changing oral therapy to those affected by this condition.”

“DRAGON II is the first global Stargardt disease trial in Japan. As such, it provides the unprecedented opportunity for Japanese patients and their families to gain access to a potential therapy for this historically untreatable disease,” added Professor Kaoru Fujinami, Principal Investigator at National Hospital Organization, Tokyo Medical Center. “The level of interest we have received in this trial speaks to the urgency of the unmet need for patients living with STGD1. Following Tinlarebant’s receipt of Sakigake designation from Japan’s Ministry of Health, we feel well positioned to rapidly advance this trial and are working diligently to get this much needed therapy to patients.”