Slide 1: Bringing Hope to Incurable Disease
Bringing Hope to Incurable Disease 2025/01
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 Yvonne Chen Serena Chen, CPA (COO) (CFO) • Certified pharmacist & master in Science (Medicinal • Certified Public Accountant & master in accounting Chemistry) from National Taiwan University from National Taipei University. • Served as Global Project Lead and Manager in Pfizer • Fina
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 Masking Open Label Treatment duration 7 consecutive days for on
Slide 18: Major Milestones
MAJOR MILESTONES Opening a New Era in Cancer Treatment ✓ 2023/02/10 - We announced that our candidate drug, LBS-007, has been approved by the Central Adelaide Local Health Network Human Research Ethics Committee (CALHN HREC) in Australia to conduct Phase I/II clinical trials for acute leukemia (incl
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 (completed enrollment, 104 subjects, age 12-20, interim analysis expec
Slide 21: Tinlarebant Market Opportunity (LBS-008)
Tinlarebant Market Opportunity (LBS-008) STARGARDT Advanced AMD % Prevalence of Advanced AMD DISCOVERY PRE-CLINICAL 1 in 10,000 The most common PHASE I inherited retinal dystrophy PHASE II PHASE III Patient population with All AMD Stargardt Disease: Prevalence MARKET Global 196M(1) 30k US 146k China
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 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 the disease reaches the cen
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 Growth of Incident DDAF Retinal Lesions LBS-008-CT02 ProgStar Cohort 1, 2 N=51* Change in incident DDAF lesion size (mm2) Patient Pool N=12 p<0.001 (aged ≤18 years) Mean change in incident DDAF lesion 0.51± 0.4 mm2 1.00 ± 1.3 mm2 size
Slide 28: Visual Acuity Outcomes During the Phase 2 Study
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)* • For the 6 subjects with prior BCVA loss: • Pre-enrollment, mean BCVA loss pre-enrollment was
Slide 29: Ph2 24-Month: Well-Tolerated Drug-Related Adverse Events
PH2 24-MONTH: WELL-TOLERATED DRUG-RELATED ADVERSE EVENTS 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 FM100 Mild 1/13 (7.7%) Intermittent
Slide 30: DRAGON & DRAGON II STGD1 Trials
DRAGON & DRAGON II STGD1 TRIALS 30
Slide 31: 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 32: Phase 3 PHOENIX Trial in Geographic Atrophy
PHASE 3 PHOENIX TRIAL IN GEOGRAPHIC ATROPHY 32
Slide 33: 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
Slide 34: Clinical Trial Design Overview in GA
CLINICAL TRIAL DESIGN OVERVIEW IN GA • Established Efficacy Endpoint – Reduction in GA Phase 3 “PHOENIX” (1) Lesion Growth Rate (DDAF) as Measured by Approximately 430 subjects targeted Retinal Imaging is the FDA Accepted Primary Enrollment (Enrolling) Endpoint for STGD1 and GA Sites Global • Early
Slide 35: Bringing Hope to Incurable Disease
Bringing Hope to Incurable Disease info@linbioscience.com 35