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Belite Bio (NASDAQ: BLTE) said the UK Medicines and Healthcare Products Regulatory Agency agreed to accept a Conditional Marketing Authorization application for Tinlarebant to treat Stargardt disease based on the Phase 3 DRAGON interim analysis.
The pivotal DRAGON trial enrolled 104 subjects across 11 jurisdictions with 2:1 randomization; the primary endpoint is atrophic lesion growth rate. The company expects topline final data in Q4 2025, which it plans to submit to MHRA for a full Marketing Authorization application.
Belite Bio (NASDAQ: BLTE) ha comunicato che la UK Medicines and Healthcare Products Regulatory Agency ha accettato di prendere in esame una domanda di Autorizzazione all’immissione in commercio condizionata per Tinlarebant per trattare la malattia di Stargardt basata sull’analisi intermedia di fase 3 DRAGON.
Lo studio chiave DRAGON ha arruolato 104 soggetti in 11 giurisdizioni con randomizzazione 2:1; l’obiettivo primario è la velocità di crescita delle lesioni atrofiche. L’azienda si aspetta i dati finali preliminari nel quarto trimestre del 2025, che intende presentare alla MHRA per una domanda completa di autorizzazione all’immissione in commercio.
Belite Bio (NASDAQ: BLTE) dijo que la UK Medicines and Healthcare Products Regulatory Agency acordó aceptar una solicitud de Autorización de Comercialización Condicionada para Tinlarebant para tratar la enfermedad de Stargardt basada en el análisis interino de fase 3 DRAGON.
El ensayo pivotal DRAGON reclutó 104 sujetos en 11 jurisdicciones con una asignación aleatoria de 2:1; el criterio primario es la tasa de crecimiento de las lesiones atróficas. La compañía espera datos finales preliminares en el Q4 de 2025, que planea presentar a la MHRA para una solicitud completa de autorización de comercialización.
Belite Bio (NASDAQ: BLTE)은 UK Medicines and Healthcare Products Regulatory Agency가 조건부 승인을 통한 마케팅 허가 신청을 Tinlarebant에 대해 Stargardt 질환 치료용으로 수락했다고 밝혔다. 이는 3상 DRAGON의 중간 분석을 기반으로 한다.
주요 DRAGON 임상은 104명의 참가자를 11개 관할구역에서 2:1 비율로 무작위 배정했으며, 주요 평가 변수는 위축된 병변의 성장 속도다. 회사는 2025년 4분기 이상의 상위 데이터를 기대하며, 이 데이터를 MHRA에 제출해 마케팅 승인을 위한 전체 제출서를 준비할 계획이다.
Belite Bio (NASDAQ: BLTE) a indiqué que l’agence britannique Medicines and Healthcare Products Regulatory Agency a accepté d’examiner une demande d’Autorisation de Mise sur le Marché Conditionnelle pour Tinlarebant afin de traiter la maladie de Stargardt, sur la base de l’analyse intermédiaire de la phase 3 DRAGON.
L’essai pivot DRAGON a recruté 104 sujets dans 11 juridictions avec une randomisation 2:1; le critère principal est le taux de croissance des lésions atrophiques. La société prévoit les premiers résultats finaux au T4 2025, qu’elle entend soumettre à la MHRA pour une demande complète d’autorisation de mise sur le marché.
Belite Bio (NASDAQ: BLTE) teilte mit, dass die britische Medicines and Healthcare Products Regulatory Agency zugestimmt hat, eine bedingte Zulassung für Tinlarebant zur Behandlung von Stargardt-Krankheit auf Basis der Zwischenanalyse der Phase-3-Studie DRAGON zu akzeptieren.
Die entscheidende DRAGON-Studie rekrutierte 104 Probanden in 11 Rechtsordnungen mit einer 2:1-Randomisierung; der primäre Endpunkt ist das Wachstum atrophischer Läsionen. Das Unternehmen erwartet Enddaten im 4. Quartal 2025, die es bei der MHRA für einen vollständigen Zulassungsantrag einreichen möchte.
Belite Bio (NASDAQ: BLTE) قالت إن هيئة تنظيم الأدوية والمنتجات الصحية البريطانية (MHRA) وافقت على قبول طلب تفويض تسويق مشروط لـ Tinlarebant لعلاج مرض Stargardt بناءً على التحليل المرحلي المتقدم لـ DRAGON.
اختبار DRAGON المحوري شمل 104 مشاركاً عبر 11 ولاية/قائمـة قضائية بتوزيع عشوائي 2:1؛ النتيجة الأساسية هي معدل نمو الآفات الضامرة. تتوقع الشركة بيانات نهائية أولية في الربع الرابع من 2025, التي تخطط لتقديمها إلى MHRA كطلب كامل للحصول على تفويض التسويق.
Positive
- MHRA accepted a Conditional Marketing Authorization application
- Phase 3 DRAGON enrolled 104 subjects across 11 jurisdictions
- Topline final data expected Q4 2025
Negative
- MHRA acceptance is for a conditional application, not full approval
- Full Marketing Authorization submission depends on final topline data in Q4 2025
- Regulatory pathway is based on an interim analysis rather than final readout
Insights
MHRA acceptance of a Conditional Marketing Authorization filing based on Phase 3 interim data is a materially positive regulatory milestone.
Belite Bio secured MHRA agreement to accept a Conditional Marketing Authorization application for Tinlarebant in Stargardt disease based on the Phase 3 DRAGON interim analysis. The Phase 3 DRAGON trial enrolled 104 adolescents across 11 jurisdictions with 2:1 randomization and uses atrophic lesion growth rate as the primary endpoint.
This outcome shortens the regulatory pathway in the UK conditional framework, but it depends on the final topline readout and subsequent full data submission. Conditional authorization implies benefit-risk was assessed favorably on interim data, while full approval will require the final dataset and longer follow‑up for safety and efficacy.
Watch the final topline data expected in Q4 2025 and the company’s planned full Marketing Authorization submission timeline thereafter; those items will determine whether the conditional filing converts to standard approval. Monitor any MHRA conditions and the complete safety profile on final analysis within the next few months.
11/02/2025 – 09:00 AM
- MHRA response is based on the Phase 3 DRAGON interim analysis results
- Topline final data expected in Q4 2025
SAN DIEGO – Belite Bio (NASDAQ: BLTE) shares are trading higher after the company announced that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) has agreed to consider its application for Conditional Marketing Authorization (CMA) for Tinlarebant in the treatment of Stargardt disease. This decision is based on interim analysis data from the Phase 3 DRAGON trial.
The news marks a significant milestone for Belite Bio, a clinical-stage biopharmaceutical firm focused on developing treatments for degenerative retinal diseases. Stargardt disease, a genetic form of macular degeneration, currently lacks approved therapies, representing a substantial unmet medical need.
“This agreement from the MHRA is encouraging and represents a crucial step toward potentially bringing the first approved treatment to patients with Stargardt disease,” stated Dr. Tom Lin, Chairman and CEO of Belite Bio, in a press release. He further emphasized the company’s commitment to working closely with regulatory bodies to advance Tinlarebant through late-stage development.
The Phase 3 DRAGON trial is a global, randomized, double-masked, and placebo-controlled study, involving 104 adolescent subjects across 11 regions worldwide, with a 2:1 randomization favoring Tinlarebant over placebo. The trial’s primary goal is to assess the drug’s impact on the growth rate of atrophic lesions, a hallmark of Stargardt disease, alongside evaluations of safety and tolerability.
While the MHRA’s acceptance of the CMA application is a positive development, investors should note that it is contingent on the final data readout from the DRAGON trial, anticipated in Q4 2025. Full marketing authorization will depend on these results and their subsequent submission to the MHRA.
Tinlarebant: A Novel Approach to Retinal Disease
Tinlarebant (LBS-008) is an oral therapy designed to tackle the root cause of Stargardt disease—the accumulation of vitamin A-based toxins known as bisretinoids. These toxins, byproducts of the visual cycle, contribute to retinal degeneration. Tinlarebant works by modulating the levels of serum retinol binding protein 4 (RBP4), the protein responsible for transporting retinol (vitamin A) from the liver to the eye, thereby reducing the formation of bisretinoids.
The drug has already received several designations, including Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation in the U.S. and Europe highlighting its potential to address a critical unmet need.
Analyst Perspective
The MHRA’s decision is viewed favorably by analysts monitoring Belite Bio. The conditional marketing authorization pathway offers a faster route to market, conditional upon demonstrating continued efficacy and safety in the final data analysis. Investors will be closely watching the topline results next year, as they will dictate whether Tinlarebant can secure full approval and potentially disrupt the treatment landscape for Stargardt disease.
This development also highlights the increasing regulatory openness to innovative therapies targeting rare diseases with significant unmet needs. The case of Belite Bio’s Tinlarebant will be instrumental in determining the broader implications for similar therapies targeting degenerative retinal conditions.
Julie Fallon
[email protected]
FAQ
What did MHRA agree for Belite Bio (BLTE) on November 2, 2025?
MHRA agreed to accept a Conditional Marketing Authorization application for Tinlarebant based on the Phase 3 DRAGON interim analysis.
When will Belite Bio (BLTE) report final topline DRAGON data?
The company expects to report final topline data in Q4 2025.
How many patients and countries were included in the Phase 3 DRAGON trial for BLTE?
The trial enrolled 104 subjects across 11 jurisdictions with a 2:1 randomization.
What is the primary endpoint of the DRAGON Phase 3 trial for BLTE?
The primary efficacy endpoint is the growth rate of atrophic lesions, plus safety and tolerability assessments.
Does MHRA acceptance mean Tinlarebant is fully approved for Stargardt disease?
No; MHRA accepted a conditional application based on interim results, not a full marketing approval.
Will Belite submit a full Marketing Authorization application to MHRA for BLTE?
Yes; the company plans to submit results from the final topline DRAGON data to MHRA for a full application.
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