Belite Bio, Inc. · NASDAQ:BLTE
Belite Bio — Q1 2025 Earnings Presentation
26 slides from Belite Bio's earnings presentation, May 14, 2025
Slide 1: Early Intervention with an Oral Treatment for Macular Degeneration
Early Intervention with an Oral Treatment for Macular Degeneration Mission for Vision Nasdaq: BLTE BELITE BIO / 1
Slide 2: Forward-Looking Statements and Legal Disclaimer
Forward-Looking Statements and Legal Disclaimer This presentation (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire,
Slide 3: Experienced Leadership Team
Experienced Leadership Team Belite Management Team Tom Lin, MMED, PhD, MBA Hendrik Scholl, MD, MA Nathan Mata, PhD Hao-Yuan Chuang, CFA, MBA, FRM (Chairman, CEO) (CMO) (CSO) (CFO) • 10+ years of executive management • 25+ years of expertise in treating retinal • 15+ years of ophthalmic drug • 13+ ye
Slide 4: Belite Bio Pipeline Overview
Belite Bio Pipeline Overview PRECLINICAL PHASE 1 PHASE 2 PHASE 3 NDA Stargardt Disease (STGD1) • Ph2 24-month final data continues to show slowing of lesion growth • Ph3, 2-year treatment, global trial (“DRAGON” Study) is ongoing (completed enrollment, 104 subjects, age 12-20) Tinlarebant • Ph2/3, 2
Slide 5: Tinlarebant Overview
Tinlarebant Overview BELITE BIO / 5
Slide 6: Market Opportunity
Market Opportunity Tinlarebant STARGARDT ADVANCED AMD % Prevalence of Advanced AMD 1 in 8,800 (3) DISCOVERY PRE-CLINICAL The most common inherited PHASE I retinal dystrophy PHASE II PHASE III Patient population with All AMD Prevalence Stargardt Disease: MARKET Global 196M(1) 53k 109k (3) (3) U.S. 20
Slide 7: Visual Cycle
Normal Processing of Vitamin A in the Tinlarebant Induced Visual Cycle Enzymes Down-Regulation Tinlarebant Visual Pigment Retinoids Visual Chromophore Rhodopsin 11c-Ral at-Ral PHOTORECEPTORS (PR) ABCA4 11c-RDH at-RDH RETINAL PIGMENT 11c-Rol RPE65 at-RE LRAT at-Rol EPITHELIUM (RPE) RBP Receptor RBP4
Slide 8: Stargardt Disease 1 (STGD1)
Formation of Toxic Vitamin A Byproducts in Tinlarebant Induced Stargardt Disease 1 (STGD1) Enzymes Down-Regulation Tinlarebant Visual Pigment Retinoids Visual Chromophore Rhodopsin 11c-Ral at-Ral A2E STARGARDT PHOTORECEPTORS Gene mutation causes loss of (PR) ABCA4 ABCA4 transporter 11c-RDH function
Slide 9: Mechanism of Tinlarebant Action
Tinlarebant Mechanism of Tinlarebant Action Tinlarebant Induced Down-Regulation Visual Pigment Enzymes Retinoids Bisretinoids are derived from vitamin A (retinol). Therefore, reducing the delivery of retinol to the eye is Visual Chromophore expected to reduce bisretinoid levels in the eye leading to
Slide 10: Similar Pathophysiology in STGD1 & GA
Tinlarebant Similar Pathophysiology in STGD1 & GA • STGD1 and GA share a STGD1: 61-YEAR-OLD FEMALE similar pathophysiology characterized by excessive accumulation of cytotoxic bisretinoids, retinal cell death, and loss of vision • Vision loss occurs slowly, despite peripheral expansion GA: 73-YEAR-O
Slide 11: Fenretinide Proof-of-Concept Study
Fenretinide Proof-of-Concept Study BELITE BIO / 11
Slide 12: Reduction of RBP4 Slows Lesion Growth in GA Subjects
Reduction of RBP4 Slows Lesion Growth in GA Subjects Sirion’s Ph 2 Proof-of-Concept Fenretinide Study in GA Reinforces Tinlarebant Potential Only 1 out of 3 subjects treated with 300 mg of fenretinide achieved RBP4 Fenretinide is an orally administered reduction ≥ 70% relative to baseline synthetic
Slide 13: Phase 2 STGD1 Trial
Phase 2 STGD1 Trial BELITE BIO / 13
Slide 14: Clinical Trial Design Overview in STGD1 Phase 2
Tinlarebant Clinical Trial Design Overview in STGD1 Phase 2 Reduction in Lesion Growth Rate (DDAF) as Measured by Retinal Imaging is the FDA Accepted Primary Endpoint in STGD1 and GA STGD1 Phase 2 “LBS-008-CT02” (Preliminary 24-Month Interim Data Available) Enrollment 13 subjects* (QDAF, no DDAF)**
Slide 15: Ph2 24-month: Reduction of Plasma RBP4 Levels
Tinlarebant Ph2 24-month: Reduction of Plasma RBP4 Levels Target Threshold (≥70% RBP4 reduction) • The 5 mg daily dose was effective to reduce RBP4 level by a mean of approximately 80% relative to baseline • RPB4 levels returned to 87% of the baseline value at End of Study (28-days following drug ce
Slide 16: Ph2 24-month: Sustained Lower Lesion Growth Compared to ProgStar
Ph2 24-month: Sustained Lower Lesion Growth Compared to ProgStar Tinlarebant • A comparison of incident DDAF lesion growth among ProgStar subjects with similar baseline characteristics as subjects in LBS- 008-CT02 (i.e, ≤ 18 years old) was performed Growth of Incident DDAF Retinal Lesions LBS-008-CT
Slide 17: DRAGON Clinical Trial Interim Analysis
DRAGON Clinical Trial Interim Analysis BELITE BIO / 17
Slide 18: DRAGON & DRAGON II Clinical Trial Design in STGD1
DRAGON & DRAGON II Clinical Trial Design in STGD1 Tinlarebant Reduction in lesion growth rate (DDAF) as measured by retinal imaging is the FDA accepted primary endpoint in STGD1 and GA STGD1 “DRAGON” Phase 3(1) STGD1 “DRAGON II” Phase 1b/2/3 Enrollment 104 subjects (have DDAF) 60 subjects (have DDAF
Slide 19: DRAGON Clinical Trial Demographics and Baseline Characteristics
DRAGON Clinical Trial Demographics and Baseline Characteristics Tinlarebant Mean (SD), Total N=104 N (%), Total N=104 Age (Years) 15.4 (2.47) Sex Male 65 (62.5%) Baseline Height (cm) 168.12 (10.349) Female 39 (37.5%) Baseline Weight (kg) 61.75 (16.891) Race Baseline BMI (kg/m2) 21.62 (4.578) White 3
Slide 20: DRAGON Interim Analysis Conclusions
DRAGON Interim Analysis Conclusions Tinlarebant • No modification of the study is required • Continue the study without sample size increase • Tinlarebant (5 mg p.o., daily) continues to be safe and well tolerated in adolescent STGD1 patients • At the time of the Interim Analysis, the overall withdr
Slide 21: DRAGON Interim Safety Data Treatment-Emergent Adverse Events
DRAGON Interim Safety Data Treatment-Emergent Adverse Events Tinlarebant Frequency N=104 Adverse Events Severity (# and % of patients) Xanthopsia Mild 28 (26.9%) Delayed Dark Adaptation Mild 27 (26.0%) Night Vision Impairment Mild 15 (14.4%) Headache Mild 8 (7.7%) • Tinlarebant (5 mg p.o., daily) c
Slide 22: DRAGON Interim Visual Acuity Data
DRAGON Interim Visual Acuity Data Change from Baseline (ETDRS Letter Score, Mean) Tinlarebant Visual Acuity Was Stabilized BELITE BIO / 22
Slide 23: Phase 3 PHOENIX Trial in Geographic Atrophy
Phase 3 PHOENIX Trial in Geographic Atrophy BELITE BIO / 23
Slide 24: Tinlarebant: ≥ 70% Reduction of RBP4
Tinlarebant Tinlarebant: ≥ 70% Reduction of RBP4 Phase 1, 5mg Daily Dosing in Healthy Adults: Mean Percent Reduction of RBP4 (excludes placebo) Final dose 70% Reduction Dosing Period BELITE BIO / 24
Slide 25: Clinical Trial Design Overview in GA
Tinlarebant Clinical Trial Design Overview in GA • Established Efficacy Endpoint – Reduction in GA Phase 3 “PHOENIX” (1) lesion growth rate (DDAF) as measured by Approximately 500 subjects targeted retinal imaging is the FDA accepted primary Enrollment (Enrolling) endpoint for STGD1 and GA Sites Glo
Slide 26: Thank You
Thank You For more info please visit: www.belitebio.com