Slide 1: Q2 2023 Financial Results Conference Call
Q2 2023 Financial Results Conference Call August 9, 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 (STGD1) • Ph2 18-month interim data continues to show slowing of lesion growth; 24-month data expected in Q4 2023 Tinlarebant • Ph3, 2-year treatment, global trial (“DRAGON” Study) is ongoing (completed enrollment, 100 sub
Slide 5: STGD1 Clinical Trials
STGD1 Clinical Trials BELITE BIO / 5
Slide 6: 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 the FDA Accepted Primary Endpoint in STGD1 and GA STGD1 Phase 2 “LBS-008-CT02” STGD1 “Dragon” Phase 3* (Preliminary 18-Month Interim Data Available) (Enrolling) Enro
Slide 7: Ph2 18-month: Reduction of Plasma RBP4 Levels
Tinlarebant (LBS-008) Ph2 18-month: Reduction of Plasma RBP4 Levels 100 90 80 Mean RBP4 concentration (%) 70 60 50 40 Target Threshold (≥70% RBP4 reduction) 30 20 10 0 Ph2 V1 D1 Ph2 V7 M3 Ph2 V9 M6 Ph2 V11 M9 Ph2 V13 M12 Ph2 V15 M15 Ph2 V17 M18 • Plasma RBP4 was reduced by approximately 80% relative
Slide 8: Ph2 18-month: Change in combined lesion size
Tinlarebant (LBS-008) Ph2 18-month: Change in combined lesion size QDAF lesion (boundary DDAF lesion (“dead outlined in blue) in STGD1 retina”) in STGD1 patient patient as measured by as measured by FAF fundus autofluorescence imaging. (FAF) imaging. DDAF lesions indicate QDAF lesions may be irrever
Slide 9: Ph2 18-month: Lesion growth rates versus ProgStar
Tinlarebant (LBS-008) Ph2 18-month: Lesion growth rates versus ProgStar • A comparison of DAF and DDAF lesion growth among ProgStar subjects with similar baseline characteristics as subjects in LBS-008-CT02 (i.e, ≤ 18 years old with no DDAF at baseline) was performed DAF Lesion Growth from Baseline
Slide 10: Ph2 18-month: Visual Acuity Data
Tinlarebant (LBS-008) Ph2 18-month: Visual Acuity Data • A trend for maintaining BCVA in a majority of subjects was observed following 18 months of treatment • Mean change in BCVA (ETDRS letters lost) within the study cohort over 18 months of treatment was -3 ± 1 letters in the Study eye and -2 ± 2
Slide 11: Ph2 18-month: Well-Tolerated Drug-Related Adverse Events
Tinlarebant Ph2 18-month: Well-Tolerated Drug-Related Adverse Events (LBS-008) Adverse Events Severity Frequency (#patients) % Recovered Xanthopsia/Chromatopsia Mild 9/12 (75%) 6/9 (66.7%) Delayed Dark Adaptation Mild 9/12 (75%) 1/9 (11.1%) Night Vision Impairment Mild 1/12 (8.3%) 0 • Tinlarebant (5
Slide 12: Fenretinide Proof-of-Concept Study
Fenretinide Proof-of-Concept Study BELITE BIO / 12
Slide 13: 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 14: Phase 3 Geographic Atrophy
Phase 3 Geographic Atrophy BELITE BIO / 14
Slide 15: 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 / 15
Slide 16: 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 Approximately 430 subjects targeted by Retinal Imaging is the FDA Accepted Enrollment (Expected to begin enrolling in mid-2023) Primar
Slide 17: Q2 2023 Financial Results
Q2 2023 Financial Results For more info please visit: www.belitebio.com
Slide 18: Q2 2023 Financial Results
Q2 2023 Financial Results (In thousands of USD) For the Three Months Ended June 30 2022 2023 Total operating expenses 2,506 6,871 - R&D 1,579 5,516 - G&A 927 1,355 Net loss (2,391) (6,811) • Cash: $57.4 million BELITE BIO / 18
Slide 19: Key Milestones
Key Milestones For more info please visit: www.belitebio.com
Slide 20: Key Milestones
Key Milestones Q1, 23 ➢ Initiated Phase 3 PHOENIX study in GA Q2, 23 ➢ April 25 - ARVO Presentation of 18-month data from Phase 2 study in STGD1 H2, 23 ➢ Completed the enrollment of Phase 3 DRAGON study in STGD1 ➢ Topline 24-month data from Phase 2 study in STGD1 expected H1, 24 ➢ Interim results fr
Slide 21: Q&A
QA For more info please visit: www.belitebio.com