Slide 1: Q2 2024 Financial Results Conference Call
Q2 2024 Financial Results Conference Call August 12, 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: 2Q 2024 Highlights
Tinlarebant (LBS-008) 2Q 2024 Highlights • DRAGON II: completed enrollment in Japan for Phase 1b, enrollment ongoing in U.S. and UK • Obtained Pioneer Drug (Sakigake) Designation in Japan for the treatment of STGD1 • PHOENIX: approximately 200 subjects have been enrolled • Raised $25 million in gros
Slide 5: Belite Bio Pipeline Overview
Belite Bio Pipeline Overview PRECLINICAL PHASE 1 PHASE 2 PHASE 3 NDA Stargardt Disease (STGD1) • Ph2, 24-month final data continued 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 (
Slide 6: Phase 2 STGD1 Trial
Phase 2 STGD1 Trial BELITE BIO / 6
Slide 7: Review of Study and Results to Date
Tinlarebant (LBS-008) Review of Study and Results to Date • ABC4 mutations in the study cohort • Severe biallelic ABCA4 mutations were found in 11 of 13 subjects • In vitro testing of the moderate alleles in subjects 3 and 5 showed pathogenicity • Variants with CADD score >20 predicted to be among t
Slide 8: Visual Acuity Outcomes During the Phase 2 Study
Tinlarebant (LBS-008) Visual Acuity Outcomes During the Phase 2 Study • Visual acuity was stabilized during the study with a mean loss of 5 letters in the cohort over 24 months of treatment (2.5 letters lost/year, left panel)1 • For the 6 subjects with prior BCVA loss: • Pre-enrollment, mean BCVA lo
Slide 9: 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 was examined (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 sub
Slide 10: Growth of Atrophic Macular Lesions Stabilized at Months 16-24
Tinlarebant Growth of Atrophic Macular Lesions Stabilized at Months 16-24 (LBS-008) • Analysis of retinal images 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
Slide 11: Ph2 24-month: safe and well-tolerated in adolescent STGD1 patients
Tinlarebant Ph2 24-month: safe and well-tolerated in adolescent STGD1 patients (LBS-008) Adverse Events Severity Frequency (# and % of patients) Xanthopsia/Chromatopsia Mild 10/13 (76.9%) Delayed Dark Adaptation Mild 9/13 (69.2%) Night Vision Impairment Mild 1/13 (7.7%) Increasing error score in FM1
Slide 12: DRAGON & DRAGON II STGD1 Trials
DRAGON & DRAGON II STGD1 Trials BELITE BIO / 12
Slide 13: DRAGON & DRAGON II Clinical Trial Design in STGD1
Tinlarebant DRAGON & DRAGON II Clinical Trial Design in STGD1 (LBS-008) 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
Slide 14: Phase 3 PHOENIX Trial in Geographic Atrophy
Phase 3 PHOENIX Trial in 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” 1 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 S
Slide 16: Q2 2024 Financial Results
Q2 2024 Financial Results For more info please visit: www.belitebio.com
Slide 17: 2024 Second-Quarter Financial Results
2024 Second-Quarter Financial Results For the Three Months ended June 30 (In thousand USD) 2024 2023 Total operating expenses 10,471 6,871 - R&D 9,078 5,516 - G&A 1,393 1,355 Net loss (9,494) (6,812) • Cash, time deposits and U.S treasury bills: $112.3 million BELITE BIO / 17
Slide 18: Q&A to begin shortly
Q&A to begin shortly For more info please visit: www.belitebio.com
Slide 19: Appendix
Appendix For more info please visit: www.belitebio.com
Slide 20: Phase 2 Clinical Trial Design and PK/PD Profile
Tinlarebant (LBS-008) Phase 2 Clinical Trial Design and PK/PD Profile Clinical Trial Design Overview Pharmacokinetic and Pharmacodynamic Profile STGD1 Phase 2 “LBS-008-CT02” Enrollment 13 subjects1 (QDAF, no DDAF)2 Sites Australia & Taiwan Masking Open Label Dose 5 mg/day Treatment 2 years duration
Slide 21: ABCA4 Mutations in the Phase 2 Study Cohort
Tinlarebant (LBS-008) ABCA4 Mutations in the Phase 2 Study Cohort Function Subject cDNA change Protein change CADD v1.6(1) Assessment tested ClinVar • Severe biallelic ABCA4 in vitro mutations were found in 1 c.1922G>C p.Cys641Ser 25.7 likely severe not provided 1 c.2905A>G p.Lys969Glu 28.9 likely s
Slide 22: ABCA4 Mutations in the Phase 2 Study Cohort (cont.)
Tinlarebant ABCA4 Mutations in the Phase 2 Study Cohort (cont.) (LBS-008) Function Subject cDNA change Protein change CADD v1.6(1) Assessment tested ClinVar • Subjects 1, 3, 4, 12, and 13 in vitro 1 c.1922G>C p.Cys641Ser 25.7 likely severe not provided • Did not develop 1 c.2905A>G p.Lys969Glu 28.9
Slide 23: ABCA4 Mutations in the Phase 2 Study Cohort (cont.)
Tinlarebant ABCA4 Mutations in the Phase 2 Study Cohort (cont.) (LBS-008) Function Subject cDNA change Protein change CADD v1.6(1) Assessment tested ClinVar • Subjects 9 & 10 (brothers) in vitro 1 c.1922G>C p.Cys641Ser 25.7 likely severe not provided • Subjects 12 & 13 1 c.2905A>G p.Lys969Glu 28.9 l
Slide 24: Visual Acuity Outcomes Prior to Study Enrollment
Tinlarebant (LBS-008) Visual Acuity Outcomes Prior to Study Enrollment Disease BCVA at BCVA at Annual Letter Age at Age at Subject Race Gender Duration Diagnosis Exam Enrollment Loss • Subgroup of 6 subjects First Exam Enrollment (yrs) (Right / Left) (Right/Left) (Right/Left) • Mean bilateral BCVA l
Slide 25: Novel Lesion Size Grading Method Improves Measurement of Atrophic Macular Lesions
Tinlarebant Novel Lesion Size Grading Method Improves Measurement of Atrophic Macular Lesions (LBS-008) Region Finder Novel Lesion Size Grading Method • Relies heavily on the grader's • Utilizes mathematical classification of lesions based on the proximity of their gray values ability to visually de