Slide 1: 2022 Full-Year Financial Results Conference Call
2022 Full-Year Financial Results Conference Call April 3, 2023 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: Belite Participants
Belite Participants Management Tom Lin, MMED, PhD, MBA Nathan Mata, PhD H.Y. Chuang, CFA, MBA, FRM (Chairman, CEO) (CSO) (CFO) • 10+ years of executive management role in • 15+ years of ophthalmic drug development • 13+ years of capital market experience, closed biotech, including 2 IPO experience a
Slide 4: Belite Bio Overview
Belite Bio Overview PRECLINICAL PHASE 1 PHASE 2 PHASE 3 NDA Stargardt Disease • Ph2 12-month interim data continues to show halting or slowing of lesion growth; 18-month data expected in April 2023 Tinlarebant • A Ph3, 2-year treatment, global trial (“Dragon” Study) is recruiting subjects (42 subjec
Slide 5: Tinlarebant (LBS-008) Overview
Tinlarebant (LBS-008) Overview BELITE BIO / 5
Slide 6: Market Opportunity
Market Opportunity Tinlarebant (LBS-008) STARGARDT DRY AMD % Prevalence of Advanced AMD DISCOVERY PRE-CLINICAL 1 in 10,000 The most common inherited PHASE I retinal dystrophy PHASE II PHASE III Patient population with All AMD Stargardt’s Disease: Prevalence MARKET Global 196M(1) 30k US 146kChina U.S
Slide 7: Mechanism of Action
Mechanism of Action Tinlarebant Induced Enzymes Down-Regulation Tinlarebant Visual Pigment Retinoids (LBS-008) Visual Chromophore Because bisretinoids are derived from vitamin A (retinol), reducing the delivery of retinol to the eye is expected to reduce bisretinoid levels in the eye leading to pres
Slide 8: Similar Pathophysiology in STGD1 & Dry AMD
Similar Pathophysiology in STGD1 & Dry AMD • STGD1 and dry AMD share STGD1: LATE-ONSET (61-YEAR OLD FEMALE) a similar pathophysiology characterized by excessive accumulation of cytotoxic bisretinoids, retinal cell death, and loss of vision • Vision loss occurs slowly, despite peripheral expansion of
Slide 9: Planned Clinical Development Pathway
Tinlarebant (LBS-008) Planned Clinical Development Pathway Reduction in Lesion Growth Rate as Measured by Retinal Imaging is an FDA Accepted Primary Endpoint in STGD1 and Geographic Atrophy (GA) 2021 2022 2023 2025 2026 Ph1b/2 Ph3 Ph3 Ph3 Ph3 Adolescent STGD1 Adolescent STGD1 GA STGD1 GA (2-year tre
Slide 10: Fenretinide Proof-of-Concept Study
Fenretinide Proof-of-Concept Study BELITE BIO / 10
Slide 11: 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 a synthetic retinoid with reduction ≥ 70% relative to baseline broad r
Slide 12: Growth Rates of Retinal Lesions in STGD1
Growth Rates of Retinal Lesions in STGD1 BELITE BIO / 12
Slide 13: Progression of Questionably Decreased Autofluorescence (QDAF) Lesion in STGD1
Progression of Questionably Decreased Autofluorescence (QDAF) Lesion in STGD1 Baseline Month 12 (QDAF lesion size = 2.45 mm2) (QDAF lesion size = 2.90 mm2) A lesion of questionably decreased autofluorescence at baseline (2.45 mm2) enlarges to 2.90 mm2 over 12 months of observation. The calculated QD
Slide 14: QDAF Lesion Transformation to Definitely Decreased Autofluorescence (DDAF) Lesion in STGD1
QDAF Lesion Transformation to Definitely Decreased Autofluorescence (DDAF) Lesion in STGD1 Baseline Month 22 (DDAF lesion size = 0.82 mm2) (DDAF lesion size = 2.09 mm2) (QDAF lesion size = 1.53 mm2) (QDAF lesion size = 0.45 mm2) The image data show transformation from QDAF into DDAF. The total area
Slide 15: STGD1 Clinical Trials
STGD1 Clinical Trials BELITE BIO / 15
Slide 16: Interim Phase 2 Results: Change in DDAF & QDAF Lesion Size During Tinlarebant
Tinlarebant Interim Phase 2 Results: Change in DDAF & QDAF Lesion Size During Tinlarebant (LBS-008) DDAF (1) During Tinlarebant Mean lesion Ph1b Ph2 Sources growth rate Ph2 6-m Ph2 12-m Subject No. baseline baseline (mm2) (mm2) (DDAF+QDAF(2)) (mm2) (mm2) The Prospective Cohort 1 0 0 0 0 Study of Chi
Slide 17: Interim Phase 2 Results: Well-Tolerated Drug-Related Adverse Events
Tinlarebant Interim Phase 2 Results: Well-Tolerated Drug-Related Adverse Events (LBS-008) Relationship to Frequency Adverse Events Severity % Recovered % On-going Drug (#patients) Xanthopsia Mild Definitely Related 10/13 6/10 (60%) 4/10 (40%) Delayed Dark Adaptation Mild Definitely Related 9/13 1/9
Slide 18: Tinlarebant: ≥ 70% Reduction of RBP4
Tinlarebant (LBS-008) Tinlarebant: ≥ 70% Reduction of RBP4 Phase 1b, 5mg Daily Dosing in Adolescent STGD1: Mean Percent Reduction of RBP4 Final dose 70% Reduction Dosing Period 0 8 15 22 28 42 Note: 6 subjects in Australian sites only as data of 5 subjects in Taiwan could not be collected due to COV
Slide 19: Clinical Trial Design Overview in STGD1
Tinlarebant (LBS-008) Clinical Trial Design Overview in STGD1 Reduction in Lesion Growth Rate (DDAF) as Measured by Retinal Imaging is an FDA Accepted Primary Endpoint in STGD1 and GA STGD1 Phase 2 STGD1 “Dragon” Phase 3* (12-Month Interim Data Available) (Enrolling) Enrollment 13 subjects (QDAF, no
Slide 20: Phase 3 Advanced Dry AMD
Phase 3 Advanced Dry AMD BELITE BIO / 20
Slide 21: Tinlarebant: ≥ 70% Reduction of RBP4
Tinlarebant (LBS-008) 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 / 21
Slide 22: Clinical Trial Design Overview in GA
Tinlarebant (LBS-008) Clinical Trial Design Overview in GA • Established Efficacy Endpoint – Reduction GA Phase 3 “Phoenix”* in Lesion Growth Rate (DDAF) as Measured by Retinal Imaging is the FDA Accepted Enrollment Approximately 430 subjects Primary Endpoint for STGD1 and GA Sites Global • Early In
Slide 23: 2022 Full-Year Financial Results
2022 Full-Year Financial Results For more info please visit: www.belitebio.com
Slide 24: 2022 Full-Year Financial Results
2022 Full-Year Financial Results (In thousand USD) For the years ended December 31 2021 2022 Total operating expenses 9,797 12,821 - R&D 7,419 8,869 - G&A 2,378 3,952 Net loss (9,818) (12,844) • IPO net proceeds: $38.0 million including the overallotment • Cash: $42.1 million BELITE BIO / 24
Slide 25: 2023 Key Anticipated Milestones
2023 Key Anticipated Milestones Q1 ➢ Initiated PHOENIX Phase 3 study in GA ➢ 42 subjects enrolled in DRAGON Phase 3 study in STGD1 Q2 ➢ April 25 - ARVO Presentation of 18-month Phase 2 efficacy and safety data in STGD1 ➢ May 3 - KOL event to discuss 18-month Phase 2 efficacy and safety data in STGD1
Slide 26: For more info please visit: www.belitebio.com
QA For more info please visit: www.belitebio.com