Canbridge Pharmaceuticals Inc. · HKEX:1228
Canbridge Pharmaceuticals — FY2022 Earnings Presentation
44 slides from Canbridge Pharmaceuticals's earnings presentation, Mar 31, 2023
Slide 1: FY2022 Annual Results Presentation
CANbridge Pharmaceuticals FY2022 Annual Results Presentation March 2023 0
Slide 2: Disclaimer
Disclaimer This document contains strictly confidential and proprietary information in relation to CANbridge Pharmaceuticals Inc. (the “Company”) and is only being made available on a confidential basis for the exclusive use of the person to whom it is addressed (the “Recipient”) and may not be repr
Slide 3: Our Vision
Our Vison To be a Global Delivering Life- Built Upon Biopharmaceutical changing Therapies a Foundation Company to Patients in China 2
Slide 4: Key Investment Highlights
Key Investment Highlights Positioned to drive rapid and comprehensive product 1 development and market access in China and globally 2 A comprehensive portfolio of rare disease-focused therapies with significant revenue potential Track record of sourcing and developing innovative and 3 validated ther
Slide 5: Experienced Management Team
Experienced Management Team • Veteran entrepreneur with 22+ years of experience in medical and pharmaceutical companies Strong global management • Former Founding General Manager of team with deep industry Genzyme China experience and a track record Dr. James • Deputy Director General at CHARD, Vice
Slide 6: Experienced Management Team
Experienced Management Team Chris Chen Pauline Li Bettie Li Qian Ma Vice President of Senior Vice Senior Director & Head of Legal and Human Resources President of Clinical Head of Finance Compliance, Development and Operation and Joint Company Operations Controller Secretaries and Board Secretary St
Slide 7: Business Overview
Part 01 Business Overview
Slide 8: A Rapidly Evolving Market for Developing Rare Diseases Products
A Rapidly Evolving Market for Developing Rare Diseases Products Emerging Favorable Regulatory Framework Hunterase launch Potential Livmarli Special rare disease funding Potential Commercial Path in self-pay market launch in China Global Expansion to Further Improve ASP 1983 2000 2018 2020 2021 2023
Slide 9: A Rapidly Evolving Market for Developing Rare Diseases Products
A Rapidly Evolving Market for Developing Rare Diseases Products Rare Disease Drug Development Incentives Faster approval Financial Increased timelines incentives awareness • Drugs approvable under certain conditions with • New China plan to slash the Value Added • National Network of Rare Diseases (
Slide 10: 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 8
Slide 11: Pipeline Targets Diseases with $15 Billion Potential
Pipeline Targets Diseases with $15 Billion Potential De-risked global pipeline with multiple programs in therapeutics with clinically validated MoAs Commercial Rights Pipeline Indications Prevalence Global Sales 2022 Global Sales (US$) Hunterase® MPS II 8k >500 M 2022 Global / China Prevalence Alagi
Slide 12: CANbridge Today
CANbridge Today Well-positioned to Deliver Multiple Commercial and Development Milestones in Rare Diseases Marketed Rare 1 Disease Product Hunterase Biliary Atresia PNH Gaucher Disease Glioblastoma Late-stage 4 Clinical Programs LIVMARLI CAN106 CAN103 CAN008 ALGS PFIC Anticipated Registration 2 Fili
Slide 13: Business Highlights in 2022
Business Highlights in 2022 Corporate and Business Development FY2022 net revenue RMB 79.0M, mainly attributable RMB 79.0M 01 to sales from Hunterase in mainland China and Nerlynx in HK/TW Announced license from UMass Chan Medical for the 02 global development and commercialization rights to a novel
Slide 14: Business Highlights in 2022
Business Highlights in 2022 CAN008 CAN108 CAN106 CAN103 CAN203 • Completed patient • First patient dosed in Phase • Dosed the first PNH • Initiated Phase 1/2 • Presented pre-clinical enrollment of the Ph2 2 EMBARK study in BA in patient in Phase 1b/2 trial in adult and data showing potential trial i
Slide 15: Pipeline Update
Part 02 Pipeline Update
Slide 16: Hunterase: The First and Only Approved ERT for MPS II in China
Hunterase: The First and the Only Approved Enzyme Replacement Therapy for MPS II in China
Slide 17: Hunterase – Early Commercialization In Non-reimbursed Market
Hunterase® – Early Commercialization In Non-reimbursed Market Identification of new patients accelerates and commercial insurance coverage expands Mucopolysaccharidosis Type II (MPS II) MPS II is a rare, disabling In East Asian countries, MPS II Chinese government has Life expectancy of patients Dea
Slide 18: Hunterase – MPS II Patients Identified in China as of Dec 2022
Hunterase® – 667 MPS II Patients Identified in China as of Dec 2022 Hunterase Commercial Update MPS II Patient Reimbursement Identification Campaign Total 667 identified patients • Hunterase has entered into 78 cities’ commercial insurance programme • 263 identified in 2022 (“Huiminbao”) as of end o
Slide 19: Livmarli (CAN108): Near-term Launch in Rare Cholestatic Liver Diseases
Livmarli®（CAN108）: Near-term Launch in Rare Cholestatic Liver Diseases in China 18
Slide 20: 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 Recent Highlights Mechanism of Action 1. IBAT is primarily responsible for recy
Slide 21: CAN108 – Commercialization Strategy
CAN108 – Commercialization Strategy Large and robust safety dataset provides strong support for further studies in BA Key Development ~22,000 targeted patients with ALGS, PFIC, BA in China Timeline ALGS PFIC Next Potential ALGS CN approval Catalyst in H1 2023 Represent 500 patients Potential ALGS TW
Slide 22: CAN106: Pipeline in A Product
CAN106: Pipeline in A Product
Slide 23: 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 Recent Highlights Mechanism of Action CAN106 Obtained global Currently in Phase Data of Phase 1 Obtained Orphan rights to develop, 1
Slide 24: CAN106 – Long-acting Anti-C5 Therapy for Complement Disorders
CAN106 – Long-acting Anti-C5 Therapy for Complement Disorders Potential “Pipeline in a Product”. Initial development efforts focused on PNH treatment. Estimated global market revenue projections of >$9B in 20251 Potential Indications for Estimated Addressable Patient Complement Therapeutics Populati
Slide 25: CAN106 – Complement Advisory Board
CAN106 – Complement Advisory Board Board will offer guidance on the CAN106 global development program, as well as explore the potential for CAN106 in other indications Anthony Amato, Robert Colvin, Gerald Cox, MD, Jean Francis, Richard Polisson, Sushrut Waikar, Brian MD MD PhD MD MD, MHSc MD, MPH We
Slide 26: CAN106 – Optimizing Commercial Pathway in China
CAN106 – Optimizing Commercial Pathway in China Phase 1b/2 Open-label, Multiple Ascending Dose Study to Evaluate CAN106 in Complement Inhibitor Treatment-Naive Patients with PNH Key Development Ongoing Phase 1b Timeline Phase 1b FPD Study Population Primary Endpoint Region in March 2022 • Complement
Slide 27: CAN008: Development in Newly Diagnosed GBM
CAN008: Development in Newly Diagnosed GBM
Slide 28: 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 Recent Highlights Mechanism of Action CD95L binds to its receptor, CD95, on the cell surface and induces the CD95L 1 oligomerization of CD95, which triggers an
Slide 29: CAN008 – Phase 1 in Newly Diagnosed GBM
CAN008 – Phase 1 in Newly Diagnosed GBM CAN008 shows clear signs for clinical efficacy in newly diagnosed GBM patients Newly Diagnosed Glioblastoma Efficacy (n = 10) PFS rates 200 mg cohort 400 mg cohort PFS-3 months 33.33% 71.42% Low dose cohort Standard dose cohort PFS-6 months 33.33% 57.14% (n=3)
Slide 30: CAN008 – Ongoing Phase 2 Registrational Trial in Newly Diagnosed Glioblastoma
CAN008 – Ongoing Phase 2 Registrational Trial in Newly Diagnosed Glioblastoma Phase 2 Multi-center, randomized, double-blind, placebo-controlled study CAN008+RT+TMZ (n = 78) Follow-up until Key Development Surgery PD, intolerance, death, or Timeline Placebo+RT+TMZ withdrawal (n = 39) Enrollment comp
Slide 31: CAN103: The First Enzyme Replacement Therapy For Gaucher Disease Developed In China
CAN103: The First Enzyme Replacement Therapy For Gaucher Disease Developed In China
Slide 32: CAN103 – Enzyme Replacement Therapy for Gaucher Disease
CAN103 – Enzyme Replacement Therapy for Gaucher Disease Aspired to provide the first domestically manufactured ERT for Gaucher patients in China Key Development CAN103 Overview Timeline Phase 1 FPD in July 2022 Phase 2 FPD First ERT for Company holds GD inherited in One of the best Included in the i
Slide 33: CAN203: Next-gen Gene Therapy for SMA
CAN203: Next-gen Gene Therapy for SMA CANbridge’s Next-Generation Innovation and Process Development Facility in Burlington, Massachusetts
Slide 34: CAN203 – Gene Therapy for Spinal Muscular Atrophy
CAN203 – Gene Therapy for Spinal Muscular Atrophy Pathophysiology Illustration CAN203 Gene Therapy Design • SMA is characterized by dysfunction of ɑ‐motor neurons that under healthy • Second-Gen gene therapy to potentially treat SMA Type 1-3 conditions innervate skeletal muscles and are responsible
Slide 35: CAN203 – Preclinical Data Presented at 2022 ASGCT
CAN203 – Preclinical Data 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 gen
Slide 36: Financial Review
Part 03 Financial Review
Slide 37: 2022 Financial Highlights
2022 Financial Highlights RMB Million R&D expenses including R&D expenses excluding 79.0 milestones payment milestones payment R&D Expenses 745.8 Revenue 153.2% 427.7 -27.3% YoY decrease of RMB -37.9% 116.5M primarily YoY increase of 311.2 +17.7% attributable to RMB 47.8M 463.1 251.7 decreased licen
Slide 38: Outlook
Part 04 Outlook
Slide 39: Multiple Catalysts to Build Valuation into 2023 and Beyond
Multiple Catalysts to Build Valuation into 2023 and Beyond H1 2023 H2 2023 H1 2024 H2 2024 CAN108 H1 2023– ALGS H2 2023 – ALGS End of 2023 – Potential BA H2 2024 – Approval in CN Approval in TW Interim readout PFIC Approval in CN/TW CAN106 Mid 2023 2024 PNH Phase 1b data readout Initiate global tria
Slide 40: Q&A
Q&A CANBRIDGE-B 01228. HK www. canbridgepharma. com
Slide 41: Thank You
THANK YOU Contact IR@canbridgepharma.com CANBRIDGE-B 01228. HK www. canbridgepharma. com WeChat Official Account 40
Slide 42: Appendix
OCRMbridge 05 Appendix
Slide 43: Income Statement
Income Statement Year ended December 31 RMB’000 2022 2021 Revenue 78,972 31,161 Cost of sales (30,078) (12,385) Gross profit 48,894 18,776 Other income and gains 12,883 13,402 Selling and distribution expenses (86,782) (100,748) Administrative expenses (108,907) (145,517) Research and development ex
Slide 44: Balance Sheet
Balance Sheet December 31 RMB’000 2022 2021 Property, plant and equipment 15,003 9,564 Right-of-use assets 129,714 19,978 Intangible assets 49,011 51,269 Other non-current assets 3,157 - Total Non-current Assets 196,885 80,811 Inventories 9,824 13,448 Trade receivables 19,054 9,141 Prepayments, othe
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