Slide 1: First Half 2022 Financial Results Conference Call
First Half 2022 Financial Results Conference Call August 11, 2022 Nasdaq: BLTE BELITE BIO / 1
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements, including statements regarding the potential implications of clinical data for patients, and Belite Bio's advancement of, and anticipated preclinical activities, clinical development, regulatory milestones, and commerc
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 • 11 years of capital market experience, closed biotech, including 2 IPO experience acr
Slide 4: Business Highlights
Business Highlights • Belite Bio’s lead asset LBS-008 is a novel, orally administered, Retinol Binding Protein 4 (“RBP4”) antagonist intended to slow or halt progression of vision loss in Stargardt disease (STGD1) and dry AMD. • Currently no approved treatments for either STGD1 or dry AMD, significa
Slide 5: Clinical Development Milestones
Clinical Development Milestones • Phase 1 trial in 111 healthy adults in US SAD and Australia SAD & MAD completed. • A 2-year open label Phase 2 trial (6 months of interim safety data and preliminary efficacy data available) in 13 adolescent STGD1 patients is ongoing. 12 months of treatment interim
Slide 6: Clear Clinical Development Pathway
Clear Clinical Development Pathway LBS 008 Reduction in Lesion Growth Rate as Measured by Retinal Imaging is an FDA Accepted Primary Endpoint in STGD1 and dry AMD completed ongoing planned Phase 1b/2 Phase 3 STGD Phase 2/3 Dry AMD Phase 1 Adolescent Adolescent NDA Dry AMD NDA STGD STGD PRV • Complet
Slide 7: Clinical Trial Design for STGD1
Clinical Trial Design for STGD1 LBS 008 STGD1 phase 3 STDG1 phase 2 (“Dragon”) Enrollment 13 participants 60 participants Sites Aus & TW Global Masking Open Label Double Blind 2:1 ratio Placebo N/A (LBS-008 : Placebo) Duration 2 years 2 years Safety & Tolerability, Efficacy (Lesion Primary measures
Slide 8: Similar Pathophysiology in STGD1 & Dry AMD
LBS Similar Pathophysiology in STGD1 & Dry AMD 008 • 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 Dry
Slide 9: Clinical Data
Clinical Data BELITE BIO / 9
Slide 10: Interim Phase 2 Results: LBS-008 Summary of Related Adverse Events
Interim Phase 2 Results: LBS 008 Summary of Related Adverse Events Relationship to Frequency Adverse Events Severity % Recovered % On-going Drug (#patients) Xanthopsia Mild Definitely Related 6/13 3/6 (50%) 3/6 (50%) Delayed Dark Adaptation Mild Definitely Related 8/13 1/8 (12.5%) 7/8 (87.5%) Night
Slide 11: Interim Phase 2 Data: Change in QDAF in Adolescent STGD1 Subjects
LBSInterim Phase 2 Data: Change in QDAF in Adolescent 008 STGD1 Subjects Distribution of Change in QDAF 23.0% 3/13 in one eye - Reduction or no change in 1 eye - Reduction or no change in both eyes - Increase in both eyes 5/13 5/13 in both eyes Areas of QDAF progressively evolve into ‘dead retina’.
Slide 12: Interim Phase 2 Data: Change in DDAF in Adolescent STGD1 Subjects
LBSInterim Phase 2 Data: Change in DDAF in Adolescent 008 STGD1 Subjects 92.3% 12 of 13 subjects showed no lesion growth 7.7% DDAF, or lesion (“dead retina”) in 1 of 13 subjects had STGD1 patient as measured by lesion growth of 0.3 retinal imaging. This is the area mm² in both eyes where retinal cel
Slide 13: Interim Phase 2 Results: Change of Vision in Adolescent STGD1 Subjects
Interim Phase 2 Results: Change of Vision in Adolescent LBS 008 STGD1 Subjects Change in BCVA BCVA gain in 8 of 13 Subjects (61.5%) 46.1 % 6/13 - Letter gain in 1 eye in one eye - Letter gain in both eyes - Letter decrease 5/13 2/13 Best-Corrected Visual Acuity (BCVA) Test 38.5% in both Provides let
Slide 14: First Half 2022 Financial Results
First Half 2022 Financial Results For more info please visit: www.belitebio.com
Slide 15: First Half 2022 Financial Results
First Half 2022 Financial Results (In thousand USD) For the six months ended June 30 2021 2022 Total operating expenses 4,798 3,559 - R&D 3,640 2,457 - G&A 1,158 1,102 Net loss (4,721) (3,461) • IPO net proceeds: $36.1 million including the overallotment. • Cash: $48.7 million. BELITE BIO / 15
Slide 16: Q&A
QA For more info please visit: www.belitebio.com
Slide 17: Appendix
Appendix For more info please visit: www.belitebio.com
Slide 18: Overview of Stargardt Disease & Dry AMD
Overview of Stargardt Disease & Dry AMD LBS 008 Stargardt Disease (STGD1) • The most common inherited retinal dystrophy (blurring or loss of central vision) in both adults and children • Caused by a dysfunctional retina-specific gene (ABCA4) which causes massive accumulation of toxic vitamin A bypro
Slide 19: LBS-008 Mechanism of Action
LBS-008 Mechanism of Action LBS-008 Induced LBS Down-Regulation Enzymes 008 Visual Pigment Retinoids 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 preservation o
Slide 20: Phase 1b Daily Dosing in Adolescent STGD1: Mean change of RBP4 (%)
Phase 1b Daily Dosing in Adolescent STGD1: LBS 008 Mean change of RBP4 (%) 110 100 90 80 6 Patients RBP4 (%) 70 60 11 Patients 50 40 70% Reduction 30 20 10 Dosing Period 0 1.0 1 Day 2.08 Day 3.015 Day 4.022 Day 5.028 Day 14 6.0 days post treatment Note: After Day 15, data were collected from 6 subje