Slide 1: Bringing Hope to Incurable Diseases
Bringing Hope to Incurable Diseases 2025/09 1
Slide 2: Disclaimer
Disclaimer This presentation is provided by Lin Bioscience, Inc. (“LBS”). The information contained within is not reviewed or reviewed by any accountant or any independent third party. Users should read this material in conjunction with all other public financial and operational information filed to
Slide 3: Novel Treatment for Unmet Medical Need
Novel Treatment for Unmet Medical Need Lin BioScience, Inc. (TW TPEx: 6696) is a drug development company established in 2016 focusing on advancing novel therapies and first- in-class treatments for unmet medical needs in various therapeutic areas such as ophthalmology, oncology, and metabolic disea
Slide 4: Pipeline
Pre- Discovery Phase 1 Phase 2 Phase 3 MARKET Pipeline Clinical Developed by Lin BioScience Obtained FDA ODD (AML, ALL), FDA fast track (AML) Acute Leukemia (FDA ODD) Multiple Solid Tumors Oncology Sponsored by Taiwan Industrial Development Programs Bureau’s Innovation Platform Program Glioblastoma
Slide 5: Chairman
Chairman • PhD in Medicine - University of Sydney; Specialization: Neurology & Immunology Tom Lin, MMED, PhD, MBA o Treatment Strategies for Autoimmune Neuropathies (Chairman) • Specialist Certificate in Clinical Neuroscience - University of Melbourne; Specialization: Neurology o Neurological Disord
Slide 6: Management Team
Management Team Irene Wang, PhD, MBA (President & CSO) • PhD in Biochemical Sciences, National Taiwan University, Trained at Scripps Research (TSRI), EMBA from University of California San Diego • Co-invented and applied 125 patents and published 6 papers • Extensive Drug development from preclinica
Slide 7: Management Team
Management Team Serena Chen Tzung-Ju Wu, PhD CFO Associate Director, R&D • Certified Public Accountant & master in • Ph.D. in Cellular and Molecular Pharmacology accounting from National Taipei University. from Rutgers University • Finance manager in a Taiwan biotech company • 10-years of global Pha
Slide 8: LBS-007 for Acute Leukemia & Solid Tumor
LBS-007 FOR ACUTE LEUKEMIA & SOLID TUMOR 8
Slide 9: Congratulations! LBS-007 Received FDA Fast Track Designation
CONGRATULATIONS! LBS-007 Received U.S. FDA Fast Track Designation!! We are excited to announce that the FDA has granted Fast Track Designation to LBS-007 for the potential treatment of relapsed or refractory acute myeloid leukemia (AML) on 26Nov2024. The FDA’s decision to grant LBS-007 Fast Track De
Slide 10: Therapeutic Options for Cancer
THERAPEUTIC OPTIONS FOR CANCER Unmet Medical Needs for Cancer Treatment – Next Generation Therapies • Cancer treatments are like a pyramid— higher levels have fewer side effects but are more costly and less applicable. Genetic Therapy • Chemotherapies form the basis of all cancer treatments. Targete
Slide 11: Non-ATP CDC7 Inhibitor
Non-ATP CDC7 Inhibitor for treatment of Broad Variety of Cancer types DISCOVERY PRE-CLINICAL ✓ PHASE I/II Novel Anti-Cancer MARKET Target Therapy PHASE II/III 1.7 in 100k (1) $5B 4.1 in 100K MARKET (2) Expected 2026 market (1) ALL incidence (2016) Orphan Drug Designation size of AML & ALL (2) AML in
Slide 12: Inhibits CDC7 in Cell Cycle Regulation
Inhibits CDC7 in Cell Cycle Regulation LBS-007 1 TARGETS 2 INHIBITS 3 PREVENTS S Phase Progression CDC7’s role in Cell Division DNA Replication 12
Slide 13: Potently Inhibits Multiple Cancer Cell Lines & Primary Patient Samples of Blood Cancers
Potently Inhibits Multiple Cancer Cell Lines & Primary Patient Samples of Blood Cancers EC50 (nanoM) 5 1000 Acute Leukemia Lymphoma Lung Cancer Breast Cancer Hematopoietic Primary Patient Solid Tumor Cell Line Sample Cell Line 13 Data courtesy of Columbia University
Slide 14: Superior Efficacy at Nanomolar Potency
SUPERIOR EFFICACY AT NANOMOLAR POTENCY Approx. 150 nanomolar of LBS-007 can achieve therapeutic effect on cancer cells AML Pancreatic cancer Lung cancer 1.5 LBS-007 (HL-60) LBS-007 LBS-007 (MV4-11) 1.0 Relative Viability 1.0 Etoposide Relative Viability Relative Viability 1.0 LBS-007 (THP-1) LBS-007
Slide 15: In Vivo Efficacy Demonstrated in Animal Models
In Vivo Efficacy Demonstrated in Animal Models Potent tumor reduction in ALL and solid tumor mouse models Acute Lymphoblastic Leukemia (ALL) Ovarian Cancer VENTRAL DORSAL Ctrl 0.3 0.6 1.2 2.4 Ctrl 0.3 0.6 1.2 2.4 (mg/kg/day) Ctrl 0.3 1 3 (mg/kg) ✓ In vivo dose responsive efficacy ✓ In vivo dose resp
Slide 16: In Vivo Efficacy Demonstrated in Animal Models
In Vivo Efficacy Demonstrated in Animal Models Potent tumor reduction in AML mouse models Aggressive AML mouse model (MLL-AF9) AML patient-derived xenograft in mice Bone Marrow Spleen hCD45+ cells (%) Vehicle LBS-007 Vehicle LBS-007 ✓ Disease burden reduced in aggressive AML mouse model at 3 mg/kg ✓
Slide 17: Clinical Development Summary
Clinical Development Summary Discovery Pre-Clinical Phase 1 Phase 2 Phase 3 LBS-007-CT01 Phase 1/2 (Phase 2 dose expansion after determining optimal dose in Phase 1) Enrollment Estimated to enroll 90 patients Sites Australia, Taiwan, US, China Masking Open Label Treatment duration 7 consecutive days
Slide 18: Major Milestones
MAJOR MILESTONES Opening a New Era in Cancer Treatment ✓ 2023/02/10 - We announced that our new drug, LBS-007, has been approved by the Central Adelaide Local Health Network Human Research Ethics Committee (CALHN HREC) to conduct Phase I/II clinical trials for acute leukemia (including AML and ALL)
Slide 19: LBS-008 for Geographic Atrophy & Stargardt Disease
LBS-008 FOR GEOGRAPHIC ATROPHY & STARGARDT DISEASE 19
Slide 20: 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 (104 subjects, age 12-20, topline data expected Q4 2025) Tinlarebant •
Slide 21: Tinlarebant Market Opportunity (LBS-008)
Tinlarebant Market Opportunity (LBS-008) STARGARDT Advanced AMD % Prevalence of Advanced AMD DISCOVERY PRE-CLINICAL 1 in 8,800(3) The most common PHASE I inherited retinal dystrophy PHASE II All AMD PHASE III Patient population with Prevalence Stargardt Disease: MARKET Global 196M(1) U.S. 20M(2) 53k
Slide 22: LBS-008 Mechanism of Action
LBS-008 Induced Enzymes LBS-008 Mechanism of Action Down-Regulation Visual Pigment Retinoids Visual Chromophore Rhodopsin 11c-Ral at-Ral STARGARDT Gene PHOTORECEPTORS A2E mutation (PR) causes loss of ABCA4 ABCA4 transporter Mutation of ABCA4 causes at-RDH function accumulation of vitamin A 11c-RDH a
Slide 23: Similar Pathophysiology in STGD1 & GA
Similar Pathophysiology in STGD1 & GA • STGD1 and GA share a STGD1: 61-year-old female: similar pathophysiology characterized by excessive accumulation of cytotoxic bisretinoids, retinal cell death, and loss of vision • Vision loss occurs slowly, despite peripheral expansion of ‘dead retina’, until
Slide 24: Phase 2 STGD1 Trial
F PHASE 2 STGD1 TRIAL / , wr Fan |
Slide 25: Clinical Trial Design Overview in STGD1 Phase 2
CLINICAL TRIAL DESIGN OVERVIEW IN STGD1 PHASE 2 Reduction in Lesion Growth Rate (DDAF) as Measured by Retinal Imaging is an FDA Accepted Primary Endpoint in STGD1 and GA STGD1 Phase 2 “LBS-008-CT02” (Preliminary 24-Month Interim Data Available) Enrollment 13 subjects* (QDAF, no DDAF)** Sites Austral
Slide 26: Ph2 24-Month: Reduction of Plasma RBP4 Levels
PH2 24-MONTH: REDUCTION OF PLASMA RBP4 LEVELS Target Threshold (≥70% RBP4 reduction) • The 5 mg daily dose was effective to reduce RBP4 level by a mean of approximately 80% relative to baseline • RPB4 levels returned to 87% of the baseline value at End of Study (28-days following drug cessation) • R
Slide 27: Ph2 24-Month: Sustained Lower Lesion Growth Compared to ProgStar
PH2 24-MONTH: SUSTAINED LOWER LESION GROWTH COMPARED TO PROGSTAR • A comparison of incident DDAF lesion growth among ProgStar subjects with similar baseline characteristics as subjects in LBS-008-CT02 (i.e, ≤ 18 years old) was performed LBS-008-CT02 ProgStar Cohort 1, 2 Growth of Incident DDAF Retin
Slide 28: DRAGON & DRAGON II STGD1 Trials
DRAGON & DRAGON II STGD1 TRIALS 28
Slide 29: DRAGON & DRAGON II Clinical Trial Design in STGD1
DRAGON & DRAGON II CLINICAL TRIAL DESIGN IN STGD1 Reduction in Lesion Growth Rate (DDAF) as Measured by Retinal Imaging is an 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 (have DDAF) Sites Globa
Slide 30: DRAGON Clinical Trial Demographics and Baseline Characteristics
DRAGON CLINICAL TRIAL DEMOGRAPHICS AND BASELINE CHARACTERISTICS Mean (SD), Total N=104 N (%), Total N=104 Age (Years) 15.4 (2.47) Sex Male 65 (62.5%) Baseline Height (cm) 168.12 (10.349) Female 39 (37.5%) Baseline Weight (kg) 61.75 (16.891) Race Baseline BMI (kg/m2) 21.62 (4.578) White 38 (36.5%) As
Slide 31: DRAGON Interim Analysis Conclusions
DRAGON INTERIM ANALYSIS CONCLUSIONS • No modification of the study is required • Continue the study without sample size increase • Tinlarebant (5 mg p.o., daily) continues to be safe and well tolerated in adolescent STGD1 patients • At the time of the Interim Analysis, the overall withdrawal rate is
Slide 32: DRAGON Interim Safety Data: Treatment-Emergent Adverse Events
DRAGON INTERIM SAFETY DATA TREATMENT-EMERGENT ADVERSE EVENTS Frequency N=104 Adverse Events Severity (# and % of patients) Xanthopsia Mild 28 (26.9%) Delayed Dark Adaptation Mild 27 (26.0%) Night Vision Impairment Mild 15 (14.4%) Headache Mild 8 (7.7%) • Tinlarebant (5 mg p.o., daily) continues to
Slide 33: DRAGON Interim Visual Acuity Data Change from Baseline
DRAGON INTERIM VISUAL ACUITY DATA CHANGE FROM BASELINE (ETDRS LETTER SCORE, MEAN) Visual Acuity Was Stabilized 33
Slide 34: Phase 3 PHOENIX Trial in Geographic Atrophy
PHASE 3 PHOENIX TRIAL IN GEOGRAPHIC ATROPHY 34
Slide 35: Tinlarebant: ≥70% Reduction of RBP4
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 35
Slide 36: Clinical Trial Design Overview in GA
CLINICAL TRIAL DESIGN OVERVIEW IN GA • Established Efficacy Endpoint – Reduction in GA Phase 3 “PHOENIX” (1) atrophic lesion growth rate as measured by retinal imaging is the FDA accepted primary Enrollment 529 subjects endpoint for STGD1 and GA Sites Global • Early Intervention – Targeting patients
Slide 37: Bringing Hope to Incurable Diseases
Bringing Hope to Incurable Diseases info@linbioscience.com 37