Belite Bio Announces Interim Analysis Results from the Pivotal Global Phase 3 DRAGON trial of Tinlarebant in Adolescent Stargardt Disease Subjects
Belite Bio (NASDAQ: BLTE) announced interim analysis results from its pivotal Phase 3 DRAGON trial of Tinlarebant in adolescent Stargardt disease patients. The Data Safety Monitoring Board (DSMB) recommended trial continuation without modifications, maintaining the original 104-subject sample size. The trial is expected to complete by Q4 2025.
Key findings show that Tinlarebant is well-tolerated with a consistent safety profile. Visual acuity remained stable in most subjects, with mean changes from baseline of less than three letter scores under both standard and low luminance throughout the two-year study.
The DRAGON trial is a randomized, double-masked, placebo-controlled study across 11 jurisdictions, with 2:1 randomization (active:placebo). The primary endpoint measures atrophic lesion growth rate. Tinlarebant has received multiple regulatory designations, including Fast Track and Rare Pediatric Disease in the U.S., Orphan Drug status in the U.S., Europe, and Japan, and Pioneer Drug Designation in Japan for Stargardt disease, which currently has no approved treatments.
Belite Bio (NASDAQ: BLTE) ha annunciato i risultati dell'analisi intermedia del suo trial pivotale di Fase 3 DRAGON relativo a Tinlarebant in pazienti adolescenti affetti da malattia di Stargardt. Il Data Safety Monitoring Board (DSMB) ha raccomandato la continuazione dello studio senza modifiche, mantenendo la dimensione originale del campione di 104 soggetti. Si prevede che lo studio si concluda entro il quarto trimestre del 2025.
I risultati chiave mostrano che Tinlarebant è ben tollerato e presenta un profilo di sicurezza coerente. L'acuità visiva è rimasta stabile nella maggior parte dei soggetti, con variazioni medie rispetto al basale inferiori a tre punteggi di lettere sia in condizioni di luminanza standard che bassa durante i due anni di studio.
Il trial DRAGON è uno studio randomizzato, doppio cieco e controllato con placebo condotto in 11 giurisdizioni, con una randomizzazione di 2:1 (attivo:placebo). L'endpoint primario misura il tasso di crescita delle lesioni atrofizzate. Tinlarebant ha ricevuto molteplici designazioni regolatorie, tra cui Fast Track e Rare Pediatric Disease negli Stati Uniti, status di Farmaco Orfano negli Stati Uniti, in Europa e in Giappone, e Designazione di Farmaco Pioniere in Giappone per la malattia di Stargardt, che attualmente non ha trattamenti approvati.
Belite Bio (NASDAQ: BLTE) anunció los resultados del análisis intermedio de su ensayo pivotal de Fase 3 DRAGON sobre Tinlarebant en pacientes adolescentes con enfermedad de Stargardt. La Junta de Monitoreo de Seguridad de Datos (DSMB) recomendó continuar el ensayo sin modificaciones, manteniendo el tamaño de muestra original de 104 sujetos. Se espera que el ensayo finalice en el cuarto trimestre de 2025.
Los hallazgos clave muestran que Tinlarebant es bien tolerado y presenta un perfil de seguridad consistente. La agudeza visual se mantuvo estable en la mayoría de los sujetos, con cambios medios desde la línea base de menos de tres puntuaciones de letras tanto en condiciones de luminancia estándar como baja a lo largo del estudio de dos años.
El ensayo DRAGON es un estudio aleatorizado, doble ciego y controlado con placebo realizado en 11 jurisdicciones, con una aleatorización de 2:1 (activo:placebo). El objetivo primario mide la tasa de crecimiento de lesiones atróficas. Tinlarebant ha recibido múltiples designaciones regulatorias, incluyendo Fast Track y Rare Pediatric Disease en EE. UU., estatus de Medicamento Huérfano en EE. UU., Europa y Japón, y Designación de Medicamento Pionero en Japón para la enfermedad de Stargardt, que actualmente no tiene tratamientos aprobados.
Belite Bio (NASDAQ: BLTE)는 청소년 스타가르트병 환자에 대한 Tinlarebant의 주요 3상 DRAGON 시험의 중간 분석 결과를 발표했습니다. 데이터 안전 모니터링 위원회(DSMB)는 수정 없이 시험을 계속할 것을 권장하며, 원래의 104명 샘플 크기를 유지했습니다. 시험은 2025년 4분기까지 완료될 것으로 예상됩니다.
주요 발견은 Tinlarebant가 잘 견디며 일관된 안전성 프로필을 가지고 있음을 보여줍니다. 두 해에 걸쳐 표준 및 저조도 조건에서 기준선 대비 평균 변화가 세 글자 점수 미만으로 대부분의 피험자에서 시력 안정성이 유지되었습니다.
DRAGON 시험은 11개 관할권에서 실시되는 무작위, 이중 맹검, 위약 대조 연구로, 2:1 비율(활성:위약)로 무작위 배정됩니다. 주요 목표는 위축 병변 성장률을 측정합니다. Tinlarebant는 미국에서 Fast Track 및 Rare Pediatric Disease를 포함한 여러 규제 지정을 받았고, 미국, 유럽 및 일본에서 희귀 의약품으로, 일본에서는 스타가르트병에 대한 선도 의약품 지정을 받았으며, 현재 승인된 치료법이 없습니다.
Belite Bio (NASDAQ: BLTE) a annoncé les résultats de l'analyse intermédiaire de son essai pivot de Phase 3 DRAGON sur Tinlarebant chez des patients adolescents atteints de la maladie de Stargardt. Le Data Safety Monitoring Board (DSMB) a recommandé de poursuivre l'essai sans modifications, en maintenant la taille d'échantillon originale de 104 sujets. L'essai devrait se terminer d'ici le quatrième trimestre 2025.
Les résultats clés montrent que Tinlarebant est bien toléré et présente un profil de sécurité cohérent. L'acuité visuelle est restée stable chez la plupart des sujets, avec des variations moyennes par rapport à la ligne de base inférieures à trois scores de lettres, tant en conditions de luminance standard qu'en faible luminance tout au long de l'étude de deux ans.
L'essai DRAGON est une étude randomisée, en double aveugle et contrôlée par placebo menée dans 11 juridictions, avec une randomisation de 2:1 (actif:placebo). Le critère principal mesure le taux de croissance des lésions atrophiques. Tinlarebant a reçu plusieurs désignations réglementaires, y compris Fast Track et Rare Pediatric Disease aux États-Unis, le statut de médicament orphelin aux États-Unis, en Europe et au Japon, et la désignation de médicament pionnier au Japon pour la maladie de Stargardt, qui n'a actuellement aucun traitement approuvé.
Belite Bio (NASDAQ: BLTE) gab die Ergebnisse der Zwischenanalyse seiner entscheidenden Phase-3-DRAGON-Studie zu Tinlarebant bei jugendlichen Patienten mit Stargardt-Krankheit bekannt. Das Data Safety Monitoring Board (DSMB) empfahl die Fortsetzung der Studie ohne Änderungen und behielt die ursprüngliche Stichprobengröße von 104 Probanden bei. Die Studie soll bis zum vierten Quartal 2025 abgeschlossen sein.
Wichtige Ergebnisse zeigen, dass Tinlarebant gut vertragen wird und ein konsistentes Sicherheitsprofil aufweist. Die Sehschärfe blieb bei den meisten Probanden stabil, mit durchschnittlichen Veränderungen von weniger als drei Buchstabenpunkten sowohl unter Standard- als auch unter niedrigen Lichtverhältnissen während der zweijährigen Studie.
Die DRAGON-Studie ist eine randomisierte, doppelblinde, placebokontrollierte Studie in 11 Jurisdiktionen mit einer Randomisierung von 2:1 (aktiv:placebo). Der primäre Endpunkt misst die Wachstumsrate atrophischer Läsionen. Tinlarebant hat mehrere regulatorische Auszeichnungen erhalten, darunter Fast Track und Rare Pediatric Disease in den USA, Orphan Drug-Status in den USA, Europa und Japan sowie die Pioneer Drug Designation in Japan für die Stargardt-Krankheit, für die derzeit keine zugelassenen Behandlungen existieren.
- DSMB confirms no need for sample size increase, suggesting strong efficacy signals
- Visual acuity stabilized in majority of subjects
- Favorable safety profile consistent with previous data
- Multiple regulatory fast-track designations received
- Trial completion not expected until Q4 2025
- No current treatment approval for commercialization
Insights
Belite Bio's announcement regarding the Phase 3 DRAGON trial of Tinlarebant represents a significant milestone in addressing Stargardt disease, a rare genetic disorder causing progressive vision loss with no currently approved treatments.
The Data Safety Monitoring Board's recommendation to proceed without modifications to the trial design or sample size is particularly notable for two reasons. First, it suggests the observed treatment effect is meeting or exceeding the statistical thresholds established in the trial design. Second, the lack of need for sample size re-estimation likely translates to cost savings and a faster path to potential approval than if additional subjects were required.
The DSMB's unusual step of recommending submission of the data for regulatory review signals confidence in the emerging efficacy profile. The reported visual acuity stabilization (mean change <3 letter scores) is clinically meaningful in Stargardt disease, where progressive deterioration is typically expected.
The safety profile remains consistent with previous observations, which is important for a medication potentially targeting a pediatric/adolescent population with a chronic condition requiring long-term treatment.
From a regulatory perspective, Tinlarebant's impressive collection of designations across major markets (Fast Track and Rare Pediatric Disease in the US, Orphan Drug status in the US, Europe, and Japan, and Pioneer status in Japan) provides multiple advantages:
- Expedited regulatory review timelines
- Reduced regulatory fees
- Extended market exclusivity periods (7 years US, 10 years EU)
- Potential priority review voucher (worth
$100-200 million )
With trial completion expected by Q4 2025, Belite Bio is positioning itself as the frontrunner to address this orphan indication with high unmet need, potentially commanding premium pricing if approved. The global trial footprint across 11 jurisdictions also suggests a coordinated worldwide commercialization strategy.
Belite Bio's Phase 3 DRAGON trial interim analysis results represent a significant de-risking event for the company's lead asset Tinlarebant in Stargardt disease, a condition with no approved treatments.
The DSMB's recommendation to maintain the original sample size of 104 subjects has direct financial implications. First, it eliminates the need for additional recruitment costs, which typically range from
The unusual step of recommending data submission for regulatory review suggests the efficacy signal may be particularly compelling, potentially accelerating the path to market. This is especially valuable for Belite as a clinical-stage company with a market cap of
From a commercial perspective, Tinlarebant's potential as a first-to-market therapy for Stargardt disease positions it for premium pricing. Orphan drugs typically command annual prices of
The Rare Pediatric Disease designation could yield a Priority Review Voucher upon approval, an asset that has historically sold for
The combination of regulatory designations across all major markets (US, Europe, Japan) provides multiple advantages:
- Extended market exclusivity periods (7-10 years depending on region)
- Reduced regulatory fees
- Potential tax credits for clinical research costs
- Expedited review timelines
The visual acuity stabilization noted in the results is particularly significant, as maintaining vision is a clinically meaningful endpoint that should support both regulatory approval and subsequent reimbursement negotiations with payers, enhancing commercial prospects.
- An independent Data Safety Monitoring Board (DSMB) for the Phase 3 DRAGON trial recommends trial continuation without any modifications, maintaining the sample size at 104 subjects following a planned interim efficacy analysis; trial completion 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, and the Pioneer Drug Designation in Japan for Stargardt disease, for which there are no approved treatments
SAN DIEGO, Feb. 27, 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.
The study design 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.
According to the 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.
“We are 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.”
The pivotal Phase 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 Japan.
Webcast Information
Date: Thursday, February 27, 2025
Time: 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time)
Webcast Link: https://event.summitcast.com/view/P4wkBPHuQY4sc9AqJPx7QV/9Z6J7TJewijZNsCPdTmZy7
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.
About Belite Bio
Belite Bio is a 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 II) in adolescent STGD1 subjects and a Phase 3 study (PHOENIX) in subjects with GA. For more information, follow us on Twitter, Instagram, LinkedIn, 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, 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.
Media and Investor Relations Contact:
Jennifer Wu /ir@belitebio.com
Julie Fallon /belite@argotpartners.com
FAQ
What were the key findings from BLTE's Phase 3 DRAGON trial interim analysis?
When will Belite Bio complete the Phase 3 DRAGON trial for Tinlarebant?
What regulatory designations has Tinlarebant received for Stargardt disease treatment?
How is the BLTE Phase 3 DRAGON trial structured?
