Canbridge Pharmaceuticals Inc. · HKEX:1228
Canbridge Pharmaceuticals — H1 2022 Earnings Presentation
39 slides from Canbridge Pharmaceuticals's earnings presentation, Aug 25, 2022
Slide 1: 2022 Interim Results
2022 Interim Results Aug 2022 CANBRIDGE, 1228.HK www.canbridgepharma.com
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 SUBSCRIPTION, PURHCASE OR SALE OF ANY SECURITIES, NOR SHALL ANY PART OF THIS DOCUMENT FORM THE BASIS OF OR BE RE
Slide 3: Business Overview
Slide Title 01 Business Overview 3
Slide 4: Our Vision
Our Vision To be a Global Biopharmaceutical Company Delivering Life-changing Therapies to Patients Built Upon a Foundation in China 4
Slide 5: Key Investment Highlights
Key Investment Highlights 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 therapies potential
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 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 Disease preva
Slide 7: Innovative Industry Pioneers Maintain Dominant Position
Innovative Industry Pioneers Maintain Dominant Position The CANbridge has the opportunity to pioneer rare disease industry in China and disrupt the global rare disease market Technology $2,479 B Rare Disease Retail (China/ Global) $1,844 B $20 B Rare Disease mRNA $140 B (US) Automotive Healthcare $6
Slide 8: CANbridge at a Glance
CANbridge at a Glance We are a leading developer of rare disease treatments for the Chinese and global markets, committed to the research, development and commercialization of innovative therapies with massive market potential Comprehensive Pipeline with Significant Revenue A Pioneer in the China Ra
Slide 9: Experienced Management Team
Experienced Management Team Strong global management team with deep industry experience and a track record of commercializing rare disease treatments • Veteran entrepreneur with 22+ years of experience • 21 years of biotechnology executive management • 15+ years of extensive banking, investment, and
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 Prevalence In China MPS II 8k ALGS 14k PFIC 7k BA 50k GBM 55k for China Hunterase® 666 CAN108 80* CAN108 24* CAN108 CAN0081 Global Sales Four I
Slide 11: 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 Overview of MPS II Hunterase Commercial Updates MPS II is a rare, disabling and life- threatening genetic disease MPS MPSII Patient II Patient Reimburs
Slide 12: Highlights for Ongoing Clinical Programs in China
Highlights for Ongoing Clinical Programs in China Paroxysmal Alagille Syndrome Progressive Familial Glioblastoma Nocturnal Biliary Atresia Gaucher Disease Intrahepatic Cholestasis Hemoglobinuria 14,000 Patients 7,000 Patients 50,000 Patients 55,000 Patients 24,000 Patients 3,000 Patients $ > 100 M*
Slide 13: 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 oncology indications with significant market potential. CANbridge owns global rights for 7
Slide 14: Business Highlights in 1H 2022
Business Highlights in 1H 2022 Hunterase ✓ Identification of new patients accelerates (539 patients*) and commercial insurance coverage expands (47 insurance programs*) CAN108 ✓ Approved under the Early and Pilot Implementation Policy in China (Boao) Corporate and Business ✓ NDA/ODR accepted by NMPA
Slide 15: Pipeline Update
Slide Title 02 Pipeline Update 15
Slide 16: 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 ALGS, PFIC and BA Obtained an exclusive license to develop, manufacture and Mechanism of Action c
Slide 17: CAN108 – Clinical Development Plan
CAN108 – Clinical Development Plan Large and robust safety dataset provides strong support for further studies in PFIC and BA 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 China and Ta
Slide 18: 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 Mechanism of Action Obtained global rights to develop, manufacture and commercialize CAN106 1 CAN106 binds to the α chain CAN106 Hig
Slide 19: CAN106 – Phase 1 SAD Topline Results
CAN106 – Phase 1 SAD Topline Results Complete blockade of complement function encourages further studies in patients with PNH 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 (SAEs) Pharmac
Slide 20: CAN106 Phase 1b Design for PNH in China
CAN106 Phase1b Design for PNH in China Phase 1b Open-label, Multiple Ascending Dose Study to Evaluate CAN106 in Complement Inhibitor Treatment-Naive Patients with PNH Study population • Complement inhibitor treatment-naive 1H 2022 Screening Induction and Maintenance Period Extension period patients
Slide 21: Complement Disease Scientific Advisory Board
Complement Disease Scientific Advisory Board Board will offer guidance on the CAN106 clinical development program, as well as explore the potential for CAN106 in other indications Brian Anthony Amato, Robert Colvin, Gerald Cox, MD, Richard Polisson, Sushrut Waikar, Jean Francis, MD Weinshenker, MD M
Slide 22: CAN008 – CD95-Fc Fusion Protein for Glioblastoma Multiforme
CAN008 – CD95-Fc Fusion Protein for Glioblastoma Multiforme (GBM) CAN008 is in clinical development as a first-line therapy for GBM in China Obtained exclusive rights to develop, manufacture and CD95L binds to its receptor, CD95, on the cell surface Mechanism of Action commercialize CAN008 (APG101/a
Slide 23: CAN008 – Phase 2/3 Patient Recruitment Ongoing
CAN008 – Phase 2 Study Patient Recruitment Ongoing Encouraging Phase 1 Data in newly diagnosed GBM1 PFS rates Cohort 1 Cohort 2 • No specific safety issues when CAN008 (200 or 400 mg) was combined with RT and TMZ. (200 mg; n=3) (400 mg; n=7) • Two patients in Cohort 2 experienced serious adverse eve
Slide 24: 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 Part A: Open-label, Within-subject, Dose escalation Phase 1 study Dose escalation: 3 infusions Continuous treatment: 16 infusions CAN10
Slide 25: 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 with industry innovators and working with academic experts In-house Research CANbridge Innovat
Slide 26: 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 Pathophysiology Illustration2 Epidemiology2 • Autosomal recessive genetic inheritance • 1 in 50 people are carriers • 1 in 6,000 to 1in 10,0
Slide 27: 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 Zolgensma®, demonstrated therapeutic advantages over the benchmark vector 2nd Gen Vector: Endogenous SMN1 promoter isolated and 2nd g
Slide 28: Financial Results
Slide Title 03 Financial Results 28
Slide 29: 1H 2022 Financial Highlights
1H 2022 Financial Highlights RMB in millions 604.6 34.7 274.8 + 36.8% - 42.4% 442.1 + 184% 158.3 + 43.2% 145.3 12.2 101.5 1H 2021 1H 2022 1H 2021 1H 2022 Including License Excluding License Fees Fees Cash Balance Revenue R&D Expenses YoY increase of RMB 385.0M, primarily YoY increase of RMB 22.5M ma
Slide 30: Outlook
Slide Title 04 Outlook 30
Slide 31: Upcoming Key Milestones
Upcoming Key Milestones 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 data analysi
Slide 32: Thank You
THANK YOU CANBRIDGE, 1228.HK Contact: www.canbridgepharma.com WeChat Official Account IR@canbridgepharma.com
Slide 33: Appendix
Slide Title 05 Appendix 33
Slide 34: Income Statement
Income Statement Six months ended 30 June RMB’000 2022 2021 Revenue 34,728 12,192 Revenue YoY increase of RMB 22.5M mainly attributable to the Cost of sales (12,561) (5,353) commercialization of Nerlynx® in Taiwan in Dec 2020 and the commercialization of Hunterase® in mainland China in May 2021 Gros
Slide 35: Balance Sheet
Balance Sheet Date ended RMB’000 30 June 2022 31 December 2021 Property, plant and equipment 7,601 9,564 Right-of-use assets 123,738 19,978 Intangible assets 50,185 51,269 Total Non-current Assets 188,331 80,811 Inventories 9,602 13,448 Trade receivables 14,553 9,141 Prepayments, other receivables a
Slide 36: CANbridge Innovative AAV Platform: AAV-GPS
CANbridge Innovative AAV Platform: AAV-GPS 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 programed to
Slide 37: Comparison of CANbridge AAV-GPS with Other Novel Capsid Approaches
Comparison of CANbridge AAV-GPS with Other Novel Capsid Approaches 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 • Inclusive of a
Slide 38: Development Collaboration & Track Record of Sourcing and Developing Therapies
Track Record of Sourcing and Developing Innovative and Validated Therapies 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 • Obtained ex
Slide 39: 1H 2022 Financial Highlights
1H 2022 Financial Highlights RMB in millions 34.7 274.8 - 43.4% + 184% Revenue R&D Expenses YoY increase of RMB 22.5M + 43.2% YoY decrease of RMB 116.5M mainly attributable to the 158.3 primarily attributable to decreased commercialization of 145.3 payments made to our licensing 12.2 Nerlynx® in Tai
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