Belite Bio Announces Completion of DRAGON, a 2-Year, Phase 3 Trial of Oral Tinlarebant in the Treatment of Stargardt Disease
Belite Bio (NASDAQ: BLTE) has announced the completion of its pivotal Phase 3 DRAGON clinical trial evaluating Tinlarebant for treating Stargardt disease type 1 (STGD1). The trial enrolled 104 adolescent subjects across 11 jurisdictions, with 94 subjects completing the study.
Tinlarebant has received multiple regulatory designations, including Breakthrough Therapy, Fast Track, and Rare Pediatric Disease Designations in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and Pioneer Drug Designation in Japan. The company expects to report topline results in Q4 2025 and plans to file New Drug Applications in 1H 2026.
Belite Bio (NASDAQ: BLTE) ha annunciato il completamento del suo studio clinico di fase 3 DRAGON, che valuta Tinlarebant per il trattamento della malattia di Stargardt di tipo 1 (STGD1). Lo studio ha reclutato 104 partecipanti adolescenti in 11 giurisdizioni, con 94 soggetti che hanno completato lo studio.
Tinlarebant ha ottenuto diverse designazioni regolatorie, tra cui Breakthrough Therapy, Fast Track e Rare Pediatric Disease Designations negli Stati Uniti, Orphan Drug Designation negli Stati Uniti, in Europa e in Giappone, e Pioneer Drug Designation in Giappone. L’azienda prevede di fornire risultati principali nel Q4 2025 e pianifica di presentare le domande di nuovo farmaco (NDA) nel primo semestre del 2026.
Belite Bio (NASDAQ: BLTE) ha anunciado la finalización de su ensayo clínico pivotal de fase 3 DRAGON, que evalúa Tinlarebant para el tratamiento de la enfermedad de Stargardt tipo 1 (STGD1). El ensayo inscribió 104 sujetos adolescentes en 11 jurisdicciones, con 94 sujetos que completaron el estudio.
Tinlarebant ha recibido múltiples designaciones regulatorias, entre ellas designaciones de Terapia Innovadora, Fast Track y Rare Pediatric Disease en EE. UU., Orphan Drug Designation en EE. UU., Europa y Japón, y Pioneer Drug Designation en Japón. La empresa espera reportar resultados preliminares en el Q4 de 2025 y planea presentar solicitudes de nuevo medicamento (NDA) en el primer semestre de 2026.
Belite Bio (NASDAQ: BLTE)는 Phase 3 DRAGON 임상 시험의 핵심 결과를 발표했습니다. 이 시험은 STGD1로의 Tinlarebant 치료를 평가합니다. 연구에는 11개 관할구역에서 104명의 청소년 피험자가 등록되었고, 94명의 피험자가 연구를 완료했습니다.
Tinlarebant는 미국에서 Breakthrough Therapy, Fast Track, Rare Pediatric Disease Designations를 포함한 다수의 규제 지정과 Orphan Drug Designation을 미국, 유럽, 일본에서, Pioneer Drug Designation을 일본에서 받았습니다. 회사는 2025년 4분기에 주요 결과를 보고할 것으로 예상되며, 2026년 상반기에 NDA를 제출할 계획입니다.
Belite Bio (NASDAQ: BLTE) a annoncé l’achèvement de son essai clinique pivot de phase 3 DRAGON évaluant le Tinlarebant pour le traitement de la maladie de Stargardt type 1 (STGD1). L’essai a recruté 104 sujets adolescents dans 11 juridictions, dont 94 sujets ont terminé l’étude.
Tinlarebant a reçu plusieurs désignations réglementaires, notamment Breakthrough Therapy, Fast Track et Rare Pediatric Disease Designations aux États‑Unis, Orphan Drug Designation aux États‑Unis, en Europe et au Japon, et Pioneer Drug Designation au Japon. L’entreprise prévoit de communiquer des résultats préliminaires au T4 2025 et de déposer des demandes de nouveau médicament (NDA) au premier semestre 2026.
Belite Bio (NASDAQ: BLTE) hat den Abschluss seiner pivottialen Phase-3-Studie DRAGON bekannt gegeben, die Tinlarebant zur Behandlung der Stargardt-Krankheit Typ 1 (STGD1) bewertet. Die Studie rekrutierte 104 jugendliche Probanden in 11 Rechtsstaaten, wobei 94 Probanden abgeschlossen haben.
Tinlarebant erhielt mehrere regulatorische Bezeichnungen, darunter Breakthrough Therapy, Fast Track und Rare Pediatric Disease Designations in den USA, sowie Orphan Drug Designation in den USA, Europa und Japan und Pioneer Drug Designation in Japan. Das Unternehmen erwartet Endergebnisse im Q4 2025 zu melden und plant, im 1. Halbjahr 2026 eine NDA einzureichen.
بيلايت بايو (بورصة ناسداك: BLTE) أعلنت عن اكتمال تجربتها السريرية المحورية من المرحلة الثالثة DRAGON التي تقيم Tinlarebant لعلاج مرض Stargardt من النوع 1 (STGD1). شملت التجربة 104 مشاركاً مراهقاً عبر 11 ولاية قضائية، مع إكمال 94 مشاركاً للدراسة.
حصل Tinlarebant على عدة designation تنظيمية، بما في ذلك Breakthrough Therapy و Fast Track و Rare Pediatric Disease Designations في الولايات المتحدة، و Orphan Drug Designation في الولايات المتحدة وأوروبا واليابان، و Pioneer Drug Designation في اليابان. تتوقع الشركة تقرير النتائج الأساسية في الربع الرابع من 2025 وتخطط لتقديم طلبات دواء نيو (NDA) في النصف الأول من 2026.
Belite Bio (NASDAQ: BLTE)宣布完成其关键的III期 DRAGON 临床试验，该试验评估 Tinlarebant 用于治疗Stargardt病1型（STGD1）。试验在11个司法辖区招募了104名青少年受试者，其中有94名受试者完成研究。
Tinlarebant 获得多项监管赋权，包括在美国的突破性治疗、快速通道和罕见儿科疾病设计，在美国、欧洲和日本的孤儿药设计，以及在日本的先锋药物设计。公司预计在2025年第四季度公布初步结果，并计划在2026年上半年提交新药申请（NDA）。
- Successful completion of Phase 3 DRAGON trial with high retention rate (94 of 104 subjects)
- Multiple prestigious regulatory designations across major markets (US, Europe, Japan)
- Potential to be first-ever approved treatment for Stargardt disease
- Strong global trial presence across 11 jurisdictions
- Results not yet available - success of treatment still uncertain
- Regulatory approval process still pending with submissions planned for 1H 2026
Insights
Belite's Phase 3 DRAGON trial completion marks significant progress toward potentially first-ever Stargardt disease treatment, with topline data coming Q4 2025.
The completion of the DRAGON Phase 3 trial represents a critical milestone in Belite Bio's development program for Tinlarebant. This 104-subject study conducted across 11 international jurisdictions evaluated Tinlarebant in adolescents with Stargardt disease type 1 (STGD1), an inherited macular degeneration with no currently approved treatments.
The trial's strong completion rate is notable – 94 of 104 enrolled subjects (approximately 90%) finished the two-year study protocol. This robust retention rate strengthens the statistical power of the upcoming data analysis and suggests manageable tolerability for this oral therapy.
Tinlarebant's comprehensive regulatory support – including Breakthrough Therapy, Fast Track, and Rare Pediatric Disease Designations from the FDA, plus Orphan Drug Designation in multiple regions – indicates recognition of both the significant unmet need and Tinlarebant's potential therapeutic value. These designations provide accelerated review pathways that could expedite approval if the data prove positive.
The primary endpoint measuring atrophic lesion growth rate is clinically meaningful as it directly tracks disease progression. With topline data expected in Q4 2025 and potential regulatory submissions in 1H 2026, Belite is positioned at a critical inflection point. Should the trial meet its endpoint, Tinlarebant could become the first-ever approved therapy for this devastating retinal disease, addressing a significant unmet medical need and potentially transforming patient care.
- Tinlarebant has been granted Breakthrough Therapy, Fast Track, and Rare Pediatric Disease Designations in the U.S.; Orphan Drug Designation in the U.S., Europe, and Japan; and Pioneer Drug Designation in Japan for Stargardt disease
- Last subject visit completed in the pivotal Phase 3 DRAGON trial of Tinlarebant in Stargardt disease
- Topline data expected in Q4 2025
SAN DIEGO, Sept. 12, 2025 (GLOBE NEWSWIRE) -- Belite Bio Inc (NASDAQ: BLTE) (“Belite” or the “Company”), a clinical-stage drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced the completion of the last subject visit in the Phase 3 DRAGON clinical trial evaluating Tinlarebant for the treatment of Stargardt disease type 1 (STGD1).
“We are very pleased to announce the successful completion of the DRAGON trial. This is an important milestone in our mission to bring a treatment to patients living with Stargardt disease,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “With no approved therapies available today, Tinlarebant has the potential to be the first treatment for this devastating inherited macular degeneration. We are deeply grateful to the patients, families, investigators, and study teams worldwide who made this clinical trial possible.”
The DRAGON trial enrolled 104 adolescent subjects across 11 jurisdictions, including the United States, United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia, with a 2:1 randomization (Tinlarebant:placebo). A total of 94 subjects completed the study, with the last study visit conducted on September 11, 2025. The primary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability of Tinlarebant will also be assessed.
Belite Bio expects to report top-line results from the DRAGON trial in Q4 2025 and plans to file New Drug Applications in 1H 2026.
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 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
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