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Tinlarebant

Phase 3

Geographic Atrophy | Small molecule | Ophthalmology |Belite Bio, Inc|Last Updated: Jul 23, 2026

Target and mechanism

Molecular targetRBP4
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment429

FDA Designations

BREAKTHROUGH_THERAPYFAST_TRACKORPHAN_DRUGRARE_PEDIATRIC_DISEASEPRIORITY_REVIEW

Clinical trial landscape

Tinlarebant · 5 trials · 6 indications

Phase 3 2Phase 2 1Phase 1 2
NCT05949593Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Geographic AtrophyGeographic Atrophy
ACTIVE NOT_RECRUITING429 Analytics
NCT05244304Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt DiseaseStargardt Disease 1
COMPLETED104 Analytics
PHASE3ACTIVE NOT_RECRUITING
Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Geographic Atrophy
Geographic AtrophyUnlock trial analytics
PHASE3COMPLETED
Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease
Stargardt Disease 1Unlock trial analytics

Study Endpoints

Primary Endpoints

To measure the rate of change (growth rate slope) in geographic atrophy (GA) lesion size
From baseline to Month 24]
To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
Baseline thru month 24
To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy
From baseline to Month 24
To measure the pharmacokinetics (PK) of tinlarebant in plasma following a single oral dose in healthy volunteers aged 50-85.
Up to 168 hours

Area under the plasma concentration versus time curve from time 0 to the last time point with quantifiable concentration (AUC0-t)

To measure the concentrations of retinol-binding protein 4 (RBP4), a pharmacodynamic (PD) biomarker, in plasma following a single oral dose in healthy volunteers aged 50-85
Up to 168 hours

Time of minimal RPB4 levels post-dose (Tmin)

To evaluate systemic and ocular safety and tolerability of tinlarebant.
From baseline to 24 months

To evaluate safety and tolerability of daily dosing of tinlarebant assessed by incidence and/or severity of ocular and non-ocular adverse events.

The optimal dose for Phase 2.
Up to 24 months

To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease.

Secondary Endpoints

To measure the change in best-corrected visual acuity (BCVA) as assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale
From baseline to Month 24
To measure changes in the area and size of the inner/outer segment junction of photoreceptors by spectral domain optical coherence tomography (SD-OCT)
From baseline to Month 24
To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Baseline thru month 24
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
LBS-008, TinlarebantEXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
TinlarebantEXPERIMENTAL5 mg tablet taken orally once a day
Cohort 1: 5 mg, fastedACTIVE_COMPARATORA single dose (5 mg) of tinlarebant will be administered to each study participant on study Day 1.
Cohort 2: 10 mg, fastedACTIVE_COMPARATORA single dose (10 mg) of tinlarebant will be administered to each study participant on study Day 1.

Interventions

NameTypeDescription
TinlarebantDRUG5 mg tablet taken orally once a day
PlaceboDRUGPlacebo tablets for tinlarebant 5 mg prepared similarly.
Tinlarebant (LBS-008)DRUGA single dose of Tinlarebant (LBS-008) will be administered to each study participant on study Day 1.
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Eligibility Criteria

Age Range60 Years to 85 Years
SexALL
Healthy VolunteersNo
Study Sites49

Inclusion Criteria: * Subjects must have a confirmed diagnosis of GA with atrophic lesions in 1 or both eyes. * Minimum BCVA is required in the study eye Exclusion Criteria: * The presence of diabetic macular edema or macular disease in either eye. * Diabetic retinopathy more advanced than mild n...

Countries:United StatesAustraliaChinaCzechiaFranceSwitzerlandTaiwanUnited KingdomBelgiumGermanyHong KongNetherlandsJapan
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Recent Changes (Last 90 Days)

MEDIUMAug 23, 2026NCT05244304TRIAL_REMOVED: changed
MEDIUMAug 23, 2026NCT05244304TRIAL_REMOVED: changed
MEDIUMAug 23, 2026NCT05244304TRIAL_REMOVED: changed

Frequently asked questions about Tinlarebant

What is Tinlarebant used for?

Tinlarebant is an investigational small molecule being developed for Stargardt Disease 1 (STGD1) and Geographic Atrophy, both of which are ophthalmologic conditions. It is also being studied in healthy volunteers for pharmacokinetic and pharmacodynamic purposes. Tinlarebant is currently in Phase 3 clinical development for these indications.

How does Tinlarebant work?

Tinlarebant is a small molecule therapeutic being developed by Belite Bio, Inc. It is designed to target the underlying pathophysiology of Stargardt Disease and Geographic Atrophy, though the specific molecular target has not been disclosed in available information. The drug is being evaluated for its ability to slow disease progression in these retinal conditions.

Who makes Tinlarebant?

Tinlarebant is being developed by Belite Bio, Inc, a biopharmaceutical company listed on the NASDAQ under the ticker symbol BLTE. The company is conducting clinical trials to evaluate the safety and efficacy of Tinlarebant for the treatment of Stargardt Disease and Geographic Atrophy.

What phase is Tinlarebant in?

Tinlarebant is currently in Phase 3 clinical development for Stargardt Disease and Geographic Atrophy. It has received several FDA designations including Breakthrough Therapy, Fast Track, Orphan Drug, Rare Pediatric Disease, and Priority Review. The drug is investigational and has not yet been approved by regulatory authorities.

What clinical trials is Tinlarebant in?

Tinlarebant has been studied in several clinical trials. NCT05244304 is a completed Phase 3 trial in adolescent Stargardt Disease. NCT05667688 is a completed Phase 1 trial in healthy volunteers. NCT05949593 is an active Phase 3 trial in Geographic Atrophy. NCT06388083 is an active Phase 2/3 trial in Stargardt Disease.

Is Tinlarebant FDA approved?

Tinlarebant is not FDA approved. It is an investigational drug currently in Phase 3 clinical trials for Stargardt Disease and Geographic Atrophy. The drug has received FDA designations including Breakthrough Therapy, Fast Track, Orphan Drug, Rare Pediatric Disease, and Priority Review, which facilitate its development and review process.