tinlarebant (LBS-008) / Lin BioScience, Belite Bio - LARVOL DELTA

Belite Bio Announces Submission of New Drug Application to the Ministry of Health, Labour, and Welfare in Japan under the Sakigake Designation System for Tinlarebant for the Treatment of Stargardt Disease Type 1 (Yahoo Finance) - "Tinlarebant will be reviewed under the Sakigake Designation System - Japan's expedited regulatory pathway for innovative medical products."

Japan filing • Japanese regulatory • Inherited Retinal Dystrophy

Interim Findings from a Two-year Phase 2 Study of the Safety and Tolerability of Tinlarebant in Adolescent Stargardt (STGD1) Subjects (Macula 2023) - "Tinlarebant has shown acceptable safety profile and trend for reducing retinal autofluorescence, suggesting a reduction in cytotoxic autofluorescent bisretinoids. There was also stabilization or improvement in visual acuity in a majority of subjects and promising anatomical outcomes. An international, pivotal phase 3 STGD1 study (DRAGON) is currently enrolling subjects."

Clinical • P2 data • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

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

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

Clinical • 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

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

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

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

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

Belite Bio…announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation of the New Drug Application (NDA) for tinlarebant for the treatment of Stargardt Disease Type 1 (STGD1) (GlobeNewswire) - "The FDA has set a Prescription Drug User Fee Act (PDUFA) date of February 12, 2027. The filing was based on the results from the Phase 3 DRAGON trial, which evaluated tinlarebant for the treatment of STGD1....If approved, tinlarebant would be the first-ever approved treatment for Stargardt Disease Type 1."

FDA filing • PDUFA • Priority review • Inherited Retinal Dystrophy

Belite Bio…announced additional, positive secondary endpoint data from its Phase 3 DRAGON trial of tinlarebant in Stargardt disease type 1 (STGD1) (GlobeNewswire) - "The findings, along with previously reported topline data, were delivered in an oral presentation at the American Society of Retina Specialists (ASRS) 2026 Annual Meeting....Notably, the presentation reported that quantitative autofluorescence (qAF), a marker of toxic bisretinoid accumulation, showed a marked divergence between treatment groups. At month 25, in subjects treated with tinlarebant, qAF values remained stable to slightly decreased from baseline (approximately 2%), whereas placebo-treated subjects showed an approximate 20% increase in qAF from baseline. The presentation also provided encore data. As previously announced, the Phase 3 DRAGON trial...met its primary efficacy endpoint, demonstrating a statistically significant and clinically meaningful 35.7% reduction in the growth rate of retinal lesions..."

P3 data • Inherited Retinal Dystrophy

Belite Bio Completes Rolling Submission of New Drug Application to U.S. Food and Drug Administration for Tinlarebant for the Treatment of Stargardt Disease Type 1 (GlobeNewswire) - "The rolling NDA was initiated in April 2026 and was submitted under Breakthrough Therapy Designation (BTD), which was granted by the FDA due to the high unmet need among patients living with STGD1. The completed application will undergo the 60-day review period with the FDA, and if accepted, a Prescription Drug User Fee Act (PDUFA) target action date will be assigned."

FDA filing • Inherited Retinal Dystrophy

Belite Bio…announced that the Swiss Agency for Therapeutic Products (Swissmedic) has granted tinlarebant, the Company’s lead product candidate, orphan drug status (ODS) for the treatment of Stargardt disease (STGD1) (GlobeNewswire) - "Tinlarebant is the first therapeutic candidate to demonstrate clinical efficacy in Stargardt disease, having met the primary endpoint, reductions in lesion growth rate, in the pivotal, global Phase 3 DRAGON trial."

Orphan drug • Inherited Retinal Dystrophy

Belite Bio Initiates Rolling Submission of New Drug Application to the U.S. Food and Drug Administration for Tinlarebant for the Treatment of Stargardt Disease (GlobeNewswire) - "The Company expects to complete the NDA rolling submission in the second quarter of 2026....'I am proud of the data package that we are submitting to the FDA, including the transformative results from our Phase 3 DRAGON trial...' said Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio."

FDA filing • Inherited Retinal Dystrophy

LBS-008-CT08: An Open-Label, Single-Arm, 3-Year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Subjects with Stargardt Disease (clinicaltrialsregister.eu) - P2/3 | N=6 | Not yet recruiting | Sponsor: Belite Bio Inc.

New P2/3 trial • 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 | Active, not recruiting | Sponsor: Belite Bio, Inc | Recruiting ➔ Active, not recruiting

Enrollment closed • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Effects of Gastric Acid Suppression, Cytochrome P4503A Inhibition and Induction, and Food on the Pharmacokinetics of Tinlarebant in Healthy Adults. (PubMed, Clin Pharmacol Drug Dev) - "A randomized, open-label, two-period, interaction study in healthy adults was conducted in four parts to determine the effects of gastric acid suppression (omeprazole 40 mg QD), cytochrome P4503A (CYP3A) inhibition (itraconazole 200 mg BD) and induction (rifampin 600 mg QD), and food on the pharmacokinetics of tinlarebant (5 mg single dose). These data show that the pharmacokinetics of tinlarebant is not significantly altered by gastric acid suppression or food. Dosing patients with tinlarebant and strong CYP3A inhibitors is unlikely to compromise safety based on its pharmacokinetic-pharmacodynamic relationships, but tinlarebant should be contraindicated with strong CYP3A inducers due to potential treatment failure."

Clinical • Journal • PK/PD data • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders • RBP4

PHOENIX: Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Geographic Atrophy (clinicaltrials.gov) - P3 | N=429 | Active, not recruiting | Sponsor: Belite Bio, Inc | Recruiting ➔ Active, not recruiting | Trial completion date: Nov 2027 ➔ Aug 2027

Enrollment closed • Trial completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration

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