Slide 1: Q1 2024 Financial Results Conference Call
Q1 2024 Financial Results Conference Call May 14, 2024, 4:30 p.m. ET Nasdaq: BLTE For more info please visit: www.belitebio.com
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 Belite Management Team Tom Lin, MMED, PhD, MBA Nathan Mata, PhD Hao-Yuan Chuang, CFA, MBA, FRM (Chairman, CEO) (CSO) (CFO) • 10+ years of executive management • 15+ years of ophthalmic drug • 13+ years of capital market roles in biotech development experience across experience; c
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, interim Tinlarebant (L
Slide 5: STGD1 Clinical Trials
STGD1 Clinical Trials BELITE BIO / 5
Slide 6: Analysis of ABCA4 Mutations in the Phase 2 Study Cohort
Tinlarebant Analysis of ABCA4 Mutations in the Phase 2 Study Cohort (LBS-008) Function Subject cDNA change Protein change CADD v1.6(1) Assessment tested ClinVar in vitro • Severe biallelic ABCA4 1 c.1922G>C p.Cys641Ser 25.7 likely severe not provided mutations were found in 1 c.2905A>G p.Lys969Glu 2
Slide 7: Analysis of ABCA4 Mutations in the Phase 2 Study Cohort
Tinlarebant Analysis of ABCA4 Mutations in the Phase 2 Study Cohort (LBS-008) Function Subject cDNA change Protein change CADD v1.6(1) Assessment tested ClinVar in vitro • Subjects 1, 3, 4, 12, and 13 1 c.1922G>C p.Cys641Ser 25.7 likely severe not provided 1 c.2905A>G p.Lys969Glu 28.9 likely severe
Slide 8: Analysis of ABCA4 Mutations in the Phase 2 Study Cohort (cont.)
Tinlarebant Analysis of ABCA4 Mutations in the Phase 2 Study Cohort (cont.) (LBS-008) Function Subject cDNA change Protein change CADD v1.6(1) Assessment tested ClinVar in vitro • Subjects 9 & 10 (brothers) 1 c.1922G>C p.Cys641Ser 25.7 likely severe not provided 1 c.2905A>G p.Lys969Glu 28.9 likely s
Slide 9: Visual Acuity Outcomes - Background
Tinlarebant (LBS-008) Visual Acuity Outcomes - Background Disease BCVA at BCVA at Annual Letter Age at Age at Subject Race Gender Duration Diagnosis Exam Enrollment Loss First Exam Enrollment (yrs) (Right / Left) (Right/Left) (Right/Left) 1 Caucasian Female 13 15 2 36/37 34/36 - • A subgroup of 6 su
Slide 10: Change in Best Corrected Visual Acuity
Tinlarebant (LBS-008) Change in Best Corrected Visual Acuity • Visual acuity was stabilized during the study with a mean loss of 5 letters in the cohort over 24 months of treatment (equivalent to 2.5 letters lost/year, left panel)* • For the 6 subjects: • Pre-enrollment mean bilateral loss of ~10 le
Slide 11: 24-Month Change in QDAF and DDAF Lesion Size
Tinlarebant (LBS-008) 24-Month Change in QDAF and DDAF Lesion Size QDAF Lesion Incident DDAF • Transition to, and growth of, incident DDAF in cohort (Baseline) Lesion (Month 24) • In 5 of 12 subjects (42%), • no change in QDAF lesion size • no incident DDAF lesion growth observed • In 7 of 12 subjec
Slide 12: Measurement of DDAF: Region Finder vs. Novel Method
Tinlarebant Measurement of DDAF: Region Finder vs. Novel Method (LBS-008) Region Finder Novel Lesion Size Quantification Method • Relies heavily on the grader's • Utilizes mathematical classification of lesions based on the proximity of their gray values ability to visually determine the to that of
Slide 13: A Newly Developed Grading Algorithm Reveals Atrophic Macular Lesions
Tinlarebant A Newly Developed Grading Algorithm Reveals Atrophic Macular Lesions (LBS-008) • FAF Analysis using new grading methodology showed atrophic lesions within the macula (6 mm outer ring) in 12 eyes of 8 subjects at Baseline • In these subjects, growth of atrophic macular lesions was stabili
Slide 14: Phase 3 Geographic Atrophy
Phase 3 Geographic Atrophy BELITE BIO / 14
Slide 15: 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 (Enrolling) primary endpoint for STGD1 and GA Si
Slide 16: Q1 2024 Financial Results
Q1 2024 Financial Results For more info please visit: www.belitebio.com
Slide 17: 2024 First-Quarter Financial Results
2024 First-Quarter Financial Results (In thousand USD) For the Three Months ended March 31 2023 2024 Total operating expenses 6,881 8,328 - R&D 5,723 6,765 - G&A 1,158 1,563 Net loss (6,895) (7,871) • Cash & U.S treasury bills: $95.5 million • Raised $25 million in gross proceeds in a registered dir
Slide 18: Q&A to begin shortly
Q&A to begin shortly For more info please visit: www.belitebio.com