tinlarebant (BPN-14967) / Lin BioSci - LARVOL DELTA

Belite Bio…announced that United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) has agreed to accept a Conditional Marketing Authorization application for Tinlarebant for the treatment of Stargardt disease based on the interim analysis results from the Phase 3 DRAGON trial (GlobeNewswire) - "The Company remains on track to report final topline data from the Phase 3 DRAGON trial in the fourth quarter of 2025. These results are expected to be submitted to the MHRA for full Marketing Authorization Application."

MHRA filing • P3 data: top line • Ophthalmology

Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease (clinicaltrials.gov) - P3 | N=104 | Completed | Sponsor: Belite Bio, Inc | Active, not recruiting ➔ Completed

Trial completion • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Belite Bio Announces China NMPA Agrees to New Drug Application with Priority Review based on Interim Analysis Results for the Treatment of Stargardt Disease with Tinlarebant (GlobeNewswire) - "NMPA’s response is based on the interim analysis results showing statistical significance in the primary endpoint of the Phase 3 DRAGON trial. The Company remains on track to report final topline data from the Phase 3 DRAGON trial in the fourth quarter of 2025."

China filing • P3 data: top line • Priority review • Retinal Disorders

DRAGON II: A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease (clinicaltrials.gov) - P2/3 | N=60 | Recruiting | Sponsor: Belite Bio, Inc | Trial completion date: Jul 2027 ➔ Jan 2028 | Trial primary completion date: Jun 2027 ➔ Dec 2027

Trial completion date • Trial primary completion date • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Discovery of the RBP4 antagonist tinlarebant, a promising therapeutic for treating Stargardt disease and atrophic age-related macular degeneration (ACS-Fall 2025) - "We will describe the discovery and development of tinlarebant, a selective RBP4 antagonist that dissociates circulating RBP4-TTR-retinol complexes, effectively reduces serum RBP4 levels in vivo, and inhibits bisretinoid synthesis in models of enhanced retinal lipofuscinogenesis. Tinlarebant is currently in Phase 3 clinical trials for Stargardt disease and atrophic AMD."

Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

Stargardt's Disease: Molecular Pathogenesis and Current Therapeutic Landscape. (PubMed, Int J Mol Sci) - "Small-molecule therapies target vitamin A dimerization (e.g., ALK-001), inhibit lipofuscin accumulation (e.g., soraprazan), or modulate the visual cycle (e.g., emixustat hydrochloride). Gene therapy trials explore ABCA4 supplementation including strategies like RNA exon editing (ACDN-01) and bioengineered ambient light-activated OPSIN...Trials like DRAGON (Phase 3, tinlarebant), STARLIGHT (phase 2, bioengineered OPSIN) show promise, but optimizing efficacy remains challenging. With the key problem of establishing genotype-phenotype correlations, the future of STGD1 therapy may rely on approaches targeting oxidative stress, lipid metabolism, inflammation, complement regulation, and genetic repair."

Journal • Review • Age-related Macular Degeneration • Gene Therapies • Inflammation • Inherited Retinal Dystrophy • Macular Degeneration • Metabolic Disorders • Ophthalmology • Retinal Disorders • ALK

An Open-Label, Single-Arm, 2-Year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Subjects with Stargardt Disease (ANZCTR) - P2 | N=12 | Active, not recruiting | Sponsor: RBP4 Pty Ltd | Recruiting ➔ Active, not recruiting

Enrollment closed • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Belite Bio Announces Completion of Enrollment in the Pivotal Global Phase 3 PHOENIX Trial Evaluating Oral Tinlarebant in Geographic Atrophy (Belite Bio Press Release) - "Belite Bio, Inc...announced the completion of enrollment in the PHOENIX trial, a global, 24-month Phase 3 pivotal trial evaluating the safety and tolerability of Tinlarebant and its potential to reduce atrophic lesion growth rate in patients diagnosed with geographic atrophy (GA) in dry age-related macular degeneration (AMD)...The PHOENIX study is a 24-month, randomized, double-masked, placebo-controlled, multicenter, pivotal Phase 3 trial. The study is ongoing across sites in the United States, the United Kingdom, France, Czech Republic, Switzerland, China, Taiwan, and Australia."

Enrollment closed • Dry Age-related Macular Degeneration

A Phase 1b, Open-Label, Repeat Dose Study to Evaluate the Pharmacokinetics and Pharmacodynamics of tinlarebant in Chinese Healthy Adult Participants (ChiCTR) - P1 | N=8 | Not yet recruiting | Sponsor: Beijing Tongren Hospital Affiliated to Capital Medical University; Belite Bio (Shanghai) Limited

New P1 trial

Belite Bio Announces FDA Granting of Breakthrough Therapy Designation for Tinlarebant for the Treatment of Stargardt Disease (GlobeNewswire) - "Designation is based on the pivotal Phase 3 DRAGON trial interim analysis results demonstrating Tinlarebant’s efficacy and favorable safety profile; Trial completion expected by Q4 2025 (including a three-month follow-up period)"

Breakthrough therapy • Trial completion date • Inherited Retinal Dystrophy • Ophthalmology

Belite Bio Announces Poster Presentations at the Association for Research in Vision and Ophthalmology 2025 Annual Meeting (GlobeNewswire) - "Belite Bio...today announced that the Company will give two poster presentations at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting being held May 4–8, 2025, in Salt Lake City, Utah. The presentations will highlight baseline patient characteristics from the ongoing DRAGON trial and pharmacokinetic and pharmacodynamic properties from the ongoing DRAGON II trial, both in Stargardt disease."

P3 data • PK/PD data • Ophthalmology

Baseline Characteristics of Adolescent Stargardt Disease Subjects Participating in a Phase 3 Study of Tinlarebant (DRAGON Trial) (ARVO 2025) - P3 | "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • P3 data • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders • RBP4

Pharmacokinetic and Pharmacodynamic Properties of an Oral Investigational Treatment for Stargardt Disease in Adolescent Japanese Patients (ARVO 2025) - P2/3 | "Purpose Tinlarebant is an orally administered drug that targets plasma retinol binding protein 4 (RBP4) as a means to reduce vitamin A delivery to the eye and slow lesion growth in patients with Stargardt disease (STGD1)...Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • PK/PD data • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders • RBP4

Belite Bio Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Corporate Update (GlobeNewswire) - "DRAGON Trial: Ongoing, 24-month, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in adolescent STGD1 subjects...In addition, the DSMB recommended to submit the data for further regulatory review for drug approval. Primary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability will also be assessed. Trial completion expected by Q4 2025 (including a three-month follow-up period)."

DSMB • Trial completion date • Ophthalmology

Investigation of an Oral Retinol Binding Protein 4 Antagonist in the Treatment of Childhood-Onset Stargardt Disease (AAO 2024) - "Best corrected visual acuity was stable, with a mean loss of five letters, and all adverse events were mild in severity. Conclusion The safety and efficacy of tinlarebant over 24 months of treatment suggests that reduced retinol delivery to the eye may be an effective approach to slow disease progression in Stargardt disease."

Clinical • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders • RBP4

Tinlarebant (LBS-008) for Adolescents with Stargardt Disease (AAO 2024) - No abstract available

Late-breaking abstract • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

DRAGON II: A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease (clinicaltrials.gov) - P2/3 | N=60 | Recruiting | Sponsor: Belite Bio, Inc | Not yet recruiting ➔ Recruiting

Enrollment open • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

A Phase 1b, Single-centre, Open-label Study to Evaluate Food Effect and the Drug-Drug Interaction of Omeprazole, Itraconazole and Rifampin on Tinlarebant in Healthy Adult Subjects (ANZCTR) - P1 | N=48 | Completed | Sponsor: RBP4 Pty Ltd | Recruiting ➔ Completed

Trial completion • Age-related Macular Degeneration • Dry Age-related Macular Degeneration

Belite Bio Receives Sakigake (Pioneer Drug) Designation of Tinlarebant for Stargardt Disease in Japan (GlobeNewswire) - "Belite Bio, Inc...today announces that its lead pipeline, Tinlarebant, has been granted Sakigake Designation by the Ministry of Health, Labour and Welfare in Japan (MHLW) for the treatment of STGD1...Enrollment of a pivotal global Phase 3 trial of Tinlarebant in adolescent STGD1 subjects (DRAGON) has been completed with interim data expected in 4Q 2024."

Enrollment closed • Japanese regulatory • P3 data • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

This is a dose-finding study followed by 2-year extension study to evaluate safety and tolerability of Tinlarebant in adolescent subjects with Stargardt disease (ANZCTR) - P1/2 | N=10 | Completed | Sponsor: RBP4 PTY LTD | Active, not recruiting ➔ Completed

Trial completion • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders • RBP4

DRAGON II: A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease (clinicaltrials.gov) - P2/3 | N=60 | Not yet recruiting | Sponsor: Belite Bio, Inc

New P2/3 trial • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Safety, Tolerability, and Efficacy of Tinlarebant from the 24-Month Phase 2 study in Adolescent Patients Affected by Stargardt Disease (ARVO 2024) - "Tinlarebant produced a sustained and reversible reduction of RBP4 during 24 months of treatment and was found to be safe and well tolerated. The reported ocular AEs were anticipated based on the mechanism of Tinlarebant action. The absence of transition from QDAF lesions to atrophic lesions in 5 of 12 subjects over 24 months of Tinlarebant treatment, and the significantly reduced growth of incident atrophic lesions, is encouraging for the future development of Tinlarebant."

Clinical • P2 data • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders • RBP4

This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease (clinicaltrials.gov) - P1/2 | N=13 | Completed | Sponsor: RBP4 Pty Ltd | Active, not recruiting ➔ Completed

Trial completion • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

A Phase 1b, Single-centre, Open-label Study to Evaluate Food Effect and the Drug-Drug Interaction of Omeprazole, Itraconazole and Rifampin on Tinlarebant in Healthy Adult Subjects (ANZCTR) - P1 | N=48 | Recruiting | Sponsor: RBP4 Pty Ltd

New P1 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration

An Open-Label, Single-Arm, 2-Year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Subjects with Stargardt Disease (ANZCTR) - P2 | N=12 | Recruiting | Sponsor: RBP4 Pty Ltd

New P2 trial • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders