CANbridge Pharmaceuticals Inc. · HKEX:1228
CANbridge Pharmaceuticals — Investor Presentation (November 2022)
33 slides from CANbridge Pharmaceuticals's investor presentation, November 2022
Slide 1: Corporate Presentation
Slide Title CANbridge Pharmaceuticals Corporate Presentation November 2022
Slide 2: Disclaimer
Disclaimer THIS DOCUMENT OR THE INFORMATION CONTAINED HEREIN IS NOT INTENDED TO AND DOES NOT CONSTITUTE ANY OFFER OR INVITATION, SOLICITATION, COMMITMENT OR ADVERTISEMENT OF ANY OFFER FOR Slide Title SUBSCRIPTION, PURHCASE OR SALE OF ANY SECURITIES, NOR SHALL ANY PART OF THIS DOCUMENT FORM THE BASIS
Slide 3: Our Vision
Our Vision Slide Title To be a Global Biopharmaceutical Company Delivering Life-changing Therapies to Patients Built Upon a Foundation in China 3
Slide 4: Business Overview
Slide Title 01 Business Overview 4
Slide 5: Key Investment Highlights
Key Investment Highlights Slide Title Positioned to drive rapid and comprehensive product development and market access in China and globally A comprehensive portfolio of rare disease- Track record of sourcing and developing focused therapies with significant revenue innovative and validated therapi
Slide 6: Significant Global Opportunity Targeting Rare Diseases
Significant Global Opportunity Targeting Rare Diseases Proven large global rare disease markets. China represents potentially biggest untapped market Slide Title Global Rare Disease Drug Market1 (By Region) Rare Disease Prevalence in U.S., EU & China (est.) 106.6-128.0 (US$ billion) 383 China Rare D
Slide 7: A Rapidly Evolving Market for Developing Rare Diseases Products
A Rapidly Evolving Market for Developing Rare Diseases Products Slide Title Emerging Favorable Regulatory Framework Inflection Point Global Expansion to Hunterase Further Improve Launch in Special rare ASP Potential Commercial Path Self-pay disease funding market 1983 2000 2018 2020 2021 Now 2022 Ne
Slide 8: Our Comprehensive and Diversified Pipeline
Our Comprehensive and Diversified Pipeline A portfolio of biologics, small molecules and gene therapy solutions with validated mechanisms of action, targeting some of the most prevalent rare diseases and rare Slide Titlewith significant market potential. CANbridge owns global rights for 7 of the 13
Slide 9: Developing a Gene Therapy Portfolio with Potential Best-in-Class Global Assets
Developing a Gene Therapy Portfolio with Potential Best-in-Class Global Assets Gene therapy holds the promise to transform treatments for LSDs and neuromuscular diseases from ameliorative to curative Slide Title In-licensed Gene Therapy Programs and In-house Tech Platform Pipeline Candidate Discover
Slide 10: Pipeline Targets Diseases with Significant Revenue Potential
Pipeline Targets Diseases with Significant Revenue Potential De-risked global pipeline with multiple programs in therapeutics with clinically validated MoAs Slide Title Prevalence In China MPS II 8k ALGS 10k PFIC 5k BA 50k GBM 55k for China Hunterase® 666 CAN108 70* CAN108 24* CAN108 CAN0081 Global
Slide 11: Experienced Management Team
Experienced Management Team Strong global management team with deep industry experience and a track record of commercializing rare disease treatments Slide Title • 21 years of biotechnology executive management • 15+ years of extensive banking, investment, and experience strategy consulting experien
Slide 12: Pipeline Update
Slide Title 02 Pipeline Update 12
Slide 13: Highlights for Ongoing Clinical Programs in China
Highlights for Ongoing Clinical Programs in China Slide Title Alagille Syndrome Progressive Familial Biliary Atresia Glioblastoma Paroxysmal Nocturnal Gaucher Disease Intrahepatic Cholestasis Hemoglobinuria 10,000 Patients 5,000 Patients 50,000 Patients 55,000 Patients 24,000 Patients 3,000 Patients
Slide 14: Hunterase – The Only ERT Approved for MPS II Launched in China
Hunterase® – The Only ERT Approved for MPS II Launched in China Identification of new patients accelerates and commercial insurance coverage expands Slide Title Overview of MPS II Hunterase Commercial Updates MPS II is a rare, disabling and life- threatening genetic disease MPS MPSII Patient II Pati
Slide 15: CAN108 – IBAT Inhibitor for Rare Cholestatic Liver Diseases
CAN108 – IBAT Inhibitor for Rare Cholestatic Liver Diseases A novel, oral, minimally-absorbed agent designed to selectively inhibit IBAT in the ileum and treat rare cholestatic liver diseases, including Slide ALGS, PFIC Title and BA Obtained an exclusive license to develop, manufacture and Mechanism
Slide 16: CAN108 – Clinical Development Plan
CAN108 – Clinical Development Plan Large and robust safety dataset provides strong support for further studies in PFIC and BA Slide Title CAN108 Development Status in China >70,000 patients with ALGS, PFIC, and BA in China Pivotal Studies NDA Filing ALGS BA ALGS PFIC BA ALGS ✓ NDA filed in mainland
Slide 17: CAN106 – Long-acting Anti-C5 Therapy for Complement Disorders
CAN106 – Long-acting Anti-C5 Therapy for Complement Disorders Significant unmet need in treating patients with complement-related disease in China and across the globe Slide Title Mechanism of Action FDA Granted Orphan Drug Designation to CAN 106 for the Treatment of 1 CAN106 binds to the α chain CA
Slide 18: CAN106 – Phase 1 SAD Topline Results
CAN106 – Phase 1 SAD Topline Results Complete blockade of complement function encourages further studies in patients with PNH Slide Title Mean concentration-time curves of CAN106 by cohort SAD Topline Results Safety • CAN106 was safe and well-tolerated with no drug- related serious adverse events (S
Slide 19: CAN106 Phase 1b/2 Design for PNH in China
CAN106 Phase1b/2 Design for PNH in China Phase 1b/2 Open-label, Multiple Ascending Dose Study to Evaluate CAN106 in Complement Inhibitor Treatment-Naive Patients with PNH Slide Title Study population • Complement inhibitor treatment-naive 1H 2022 Screening Induction and Maintenance Period Extension
Slide 20: CAN106 – Long-acting Anti-C5 Therapy for Complement Disorders
CAN106 – Long-acting Anti-C5 Therapy for Complement Disorders Board will offer guidance on the CAN106 clinical development program, as well as explore the potential for CAN106 in other indications Slide Title Brian Anthony Amato, Robert Colvin, Gerald Cox, MD, Richard Polisson, Sushrut Waikar, Jean
Slide 21: CAN008 – CD95-Fc Fusion Protein for Glioblastoma Multiforme (GBM)
CAN008 – CD95-Fc Fusion Protein for Glioblastoma Multiforme (GBM) CAN008 is in clinical development as a first-line therapy for GBM in China Slide Title Obtained exclusive rights to develop, manufacture and CD95L binds to its receptor, CD95, on the cell surface Mechanism of Action commercialize CAN0
Slide 22: CAN008 – Phase 1 Data and Phase 2/3 Design
CAN008 – Phase 1 Data and Phase 2 Design Encouraging Phase 1 Data in newly diagnosed GBM1 Slide Title • No specific safety issues when CAN008 (200 or 400 mg) was combined with RT and TMZ. PFS rates Cohort 1 Cohort 2 (200 mg; n=3) (400 mg; n=7) • Two patients in Cohort 2 experienced serious adverse e
Slide 23: CAN103 First Patient Dosed in Phase 1/2 Trial
CAN103 First Patient Dosed in Phase 1/2 Trial Multi-center Phase 1/2 trial consists of two parts and will recruit in total 40 adolescent and adult patients in China Slide Title Part A: Open-label, Within-subject, Dose escalation Phase 1 study Dose escalation: 3 infusions Continuous treatment: 16 inf
Slide 24: Three-Pronged Gene Therapy Research Strategy
Three-Pronged Gene Therapy Research Strategy In-house gene therapy research to build AAV platform for specific tissue targeting; accelerate development of cutting-edge gene therapy technology by partnering Slide with industryTitle innovators and working with academic experts In-house Research CANbri
Slide 25: UMass Collaboration: Gene Therapy for Spinal Muscular Atrophy
UMass Collaboration: Gene Therapy for Spinal Muscular Atrophy The first and only gene therapy approved for SMA expected to hit peak global sales of $3 billion1 Slide Title Pathophysiology Illustration2 Epidemiology2 • Autosomal recessive genetic inheritance • 1 in 50 people are carriers • 1 in 6,000
Slide 26: CANbridge-UMass SMA Program Presented at 2022 ASGCT
CANbridge-UMass SMA Program Presented at 2022 ASGCT Head-to-head comparison with our 2nd gen vector and a benchmark vector, whose design is similar to the one used in Slide Zolgensma®Title , demonstrated therapeutic advantages over the benchmark vector 2nd Gen Vector: Endogenous SMN1 promoter isolat
Slide 27: CANbridge Innovative AAV Platform: AAV-GPS
CANbridge Innovative AAV Platform: AAV-GPS Slide Title CANbridge AAV-GPS Tissue Specific Delivery Platform PoC of AAV-GPS in vitro in Myotubes and Non-Muscle Cells Programmed AAV AAV with GPS AAV9 Control AAV9 w/o GPS AAV9 w/ GPS AAV-GPS GPS1 Capsid GPS Engineering Programming Human myotube AAV-GPS
Slide 28: Comparison of CANbridge AAV-GPS with Other Novel Capsid Approaches
Comparison of CANbridge AAV-GPS with Other Novel Capsid Approaches Slide Title Natural Cap Discovery Rational Design Directed Evolution In-Silico Design • 100s identified • Based on understanding of • High throughput • As for Directed evolution Pros • Nature made from evolution receptor biology • In
Slide 29: Developing Gene Therapies to Treat LSDs
Developing Gene Therapies to Treat LSDs Gene therapy holds the promise to transform treatments for LSDs such as Fabry disease / Pompe disease from chronic to curative Slide Title Application to Lysosomal Storage Diseases (LSDs) LSDs are a group of over 70 diseases that are characterized by lysosomal
Slide 30: Outlook
Slide Title 03 Outlook 30
Slide 31: Upcoming Key Milestones
Upcoming Key Milestones Slide Title We expect in 2H 2022 and 2023: ➢ CAN108 – Potential approval for ALGS ➢ CAN008 –Phase 2 interim readout in 2023 As of 30 June, 2022, we have cash balance of $604.6M RMB* ➢ Multiple pre-clinical data on gene therapy programs 2022 2023 ➢ CAN106 – Phase 1b/2 interim
Slide 32: Appendix
Slide Title 04 Appendix 32
Slide 33: Track Record of Sourcing and Developing Innovative and Validated Therapies
Track Record of Sourcing and Developing Innovative and Validated Therapies Slide Title CANbridge established Shifts focus to rare diseases from oncology 2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 • Obtained an exclusive • Obtained exclusive license rights • Obtained exclusive worldwide •
CANbridge Pharmaceuticals — Investor Presentation (November 2022) on 6ix