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

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

A Phase 1b/2 Study of the Safety and Tolerability of Tinlarebant in Adolescent Stargardt Subjects (AAO 2023) - "Tinlarebant is safe and well tolerated, and trends of slower expansion of atrophic lesions and reduced retinal thinning are reported. Maintenance of vision in the majority of subjects is also reported."

Clinical • P1/2 data • Ophthalmology

Tinlarebant (LBS-008) in Adolescent Subjects With Stargardt Disease (AAO 2023) - No abstract available

Clinical • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Economic Burden and Changing Treatment Landscape in Stargardt Disease: A Literature and Database Review (ISPOR-EU 2023) - "Seven trials investigated 5 drug interventions (emixustat, tinlarebant, STG-001, avacincaptad pegol, ALK-001) with the eighth trial investigating a gene therapy (GT) (vMCO-010). The current healthcare costs associated with SD are not well established. With uncertainty around the current economic burden of SD, it will be difficult to determine the economic impact of new treatment options when making access decisions. Future analysis of analogues from other ocular degeneration disorders may prove useful in determining the current economic burden of SD."

HEOR • Review • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders • ALK

A Phase 1b, Open-Label, Parallel Single-Dose Study to Evaluate the Pharmacokinetics and Pharmacodynamics of Tinlarebant in Healthy Volunteers Aged 50-85 (ANZCTR) - P1 | N=16 | Completed | Sponsor: RBP4 Pty Ltd | Initiation date: Dec 2000 ➔ Dec 2021

Trial initiation date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects Lesion(s) in Adolescent Subjects With STGD1 (clinicaltrials.gov) - P3 | N=90 | Active, not recruiting | Sponsor: Belite Bio, Inc | Recruiting ➔ Active, not recruiting

Enrollment closed • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

PHOENIX: Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Geographic Atrophy (clinicaltrials.gov) - P3 | N=429 | Recruiting | Sponsor: Belite Bio, Inc

New P3 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration

Visual cycle modulation via retinol binding 4 antagonism: A promising approach to treat Stargardt disease and atrophic age-related macular degeneration (ACS-Fall 2023) - "We will describe the discovery and development of tinlarebant, an orally bioavailable and selective RBP4 antagonist that modulates the visual cycle by dissociating circulating RBP4-TTR-retinol complexes...We will highlight advanced bispecific analog ACPHS-52, which presents highly favorable in vitro ADME and rodent PK profiles, excellent serum RBP4 lowering capability in vivo, and TTR stabilization activity in a gel-based assay. Furthermore, the compound is also highly efficacious at lowering retinal bisretinoid accumulation in preclinical models that recapitulate the STGD1 and atrophic AMD phenotypes."

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

Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects Lesion(s) in Adolescent Subjects With STGD1 (clinicaltrials.gov) - P3 | N=90 | Recruiting | Sponsor: Belite Bio, Inc | N=60 ➔ 90 | Trial completion date: Jun 2025 ➔ Oct 2025 | Trial primary completion date: Mar 2025 ➔ Aug 2025

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

Belite Bio Presented 18-Month Interim Data from a 24-Month Phase 2 Study of Tinlarebant in Adolescent Stargardt Disease at the 2023 ARVO Meeting (GlobeNewswire) - P1/2 | N=13 | NCT05266014 | Sponsor: RBP4 Pty Ltd | "Belite Bio...presented 18-month data from its ongoing two-year, open-label Phase 2 clinical study of Tinlarebant in adolescent STGD1 patients ('LBS-008-CT02') as part of the poster presentation at the...ARVO Annual Meeting....To date, twelve patients have completed 18 months of treatment in the ongoing two-year Phase 2 study of Tinlarebant....At Month 18, data from fundus autofluorescence (FAF) imaging revealed that nearly 60% of subjects (seven out of 12) showed no incident atrophic retinal lesions. Mean visual acuity was stabilized in the study cohort throughout the 18-month treatment period. Nine of 12 patients experienced mild xanthopsia/chromatopsia and delayed dark adaptation and one of 12 patients experienced night vision impairment....Belite Bio expects the next data readout in the Phase 2 STGD1 study to occur during the fourth quarter of 2023 when all subjects are expected to complete two years of treatment."

P2 data • Ophthalmology

A Phase 1b/2 Study of the Safety and Tolerability of Tinlarebant in Adolescent Patients Affected by Stargardt Disease– 15 month preliminary data (ARVO 2023) - "Stargardt disease is the commonest inherited macular dystrophy. Tinlarebant therapy shows promising results at 15 months in stabilishing the disease course."

Clinical • P1/2 data • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders