Canbridge Pharmaceuticals Inc. · HKEX:1228
Canbridge Pharmaceuticals — H1 2023 Earnings Presentation
38 slides from Canbridge Pharmaceuticals's earnings presentation, Aug 31, 2023
Slide 1: 2023 Interim Result Presentation
CANbridge Pharmaceuticals 2023 Interim Result Presentation Aug 2023
Slide 2: Disclaimer
Disclaimer This document contains strictly conf idential and proprietary inf ormation in relation to CANbridge Pharmaceuticals Inc. (the “Company ”) and is only being made av ailable on a conf idential basis f or the exclusiv e use of the person to whom it is addressed (the “Recipient”) and may not
Slide 3: Our Vision
Our Vision 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 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 5: CANbridge Today
CANbridge Today Well-positioned to Deliver Multiple Commercial and Development Milestones in Rare Diseases MPS II ALGS in Mainland China* Marketed Rare 2 Disease Product Hunterase LIVMARLI Biliary Atresia PNH Gaucher Disease Glioblastoma Late-stage 4 Clinical Programs LIVMARLI CAN106 CAN103 CAN008 P
Slide 6: Well-established Commercialization Infrastructure
Well-established Commercialization Infrastructure Launched Products Established Commercial Infrastructure in Greater China • 20+ years of experience in leading • Responsible f or the commercial emerging market teams, operations in Mainland China establishing marketing and • 17+ years of commercial e
Slide 7: Access to GMP Manufacturing Capability for Multiple Modalities
Access to GMP Manufacturing Capability for Multiple Modalities Secured manufacturing capacity 7 CMC staff with third-party collaboration In-house process development Well-established quality control in China and the U.S. partners lab in the U.S. team in China and the U.S. Abundant manufacturing capa
Slide 8: Our Comprehensive and Diversified Pipeline
Our Comprehensive and Diversified Pipeline CANbridge holds global rights to 8 out of 14 assets, spanning biologics, small molecules, and gene therapy, targeting most prevalent rare diseases and oncology indications, with proven mechanisms and significantt market potential Candidate Mechanism Discove
Slide 9: Pipeline Targets Diseases with $15 Billion Potential
Pipeline Targets Diseases with $15 Billion Potential Commercial Rights Pipeline Indications Prevalence Global Sales 2022 Global Sales (US$) Hunterase® MPS II 8k >500 M 2022 Global / China Prevalence Alagille Syndrome 10k 75 M China Livmarli PFIC 5k >25 M (CAN108) Biliary Atresia 6.5k* NA Abbreviatio
Slide 10: Past and Upcoming Milestones
Past and Upcoming Milestones Two products planned for NRDL negotiation in 2023 and Multiple NDA filings expected by 4Q2024 Livmarli Hunterase CAN106 CAN008 CAN103 CAN203 Phase 2 Enrollment Data Presented at completed in Q1 ASGCT2 shows 1H 2023 Identified 739 IDMC1 completed CAN203 is able to Approve
Slide 11: Pipeline Portfolio Update
Pipeline Portfolio Update
Slide 12: Livmarli (CAN108): Near-term Launch in Rare Cholestatic Liver Diseases in China
Livmarli® (CAN108): Near-term Launch in Rare Cholestatic Liver Diseases in China 11
Slide 13: Livmarli – IBAT Inhibitor for Rare Cholestatic Liver Diseases
Livmarli – 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 • Received China NMPA marketing approva
Slide 14: Statistically Significant, Clinically Meaningful, and Sustained Improvement in Pruritis
Statistically Significant, Clinically Meaningful, and Sustained Improvement in Pruritis Safety Data of LIVMARLI in ALGS Includes 5 Years of Follow-up Events observed over 5% patients Number of events per 100 person-years Diarrhea 41.6 Abdominal pain 38.6 Vomiting 19.8 Nausea 2.9 Fat-soluble vitamin
Slide 15: Long-term Data Emerging Suggests Improvement in Event-Free Survival and Growth
Long-term Data Emerging Suggests Improvement in Event-Free Survival and Growth • Cohort of 84 patients treated with maralixibat compared with an external matched control cohort of 469 patients from the GALA* Database • Events defined as: liver transplantation; biliary diversion surgery; liver decomp
Slide 16: Commercial Launch of LIVMARLI in Reimbursed Market in Jan 2024
Commercial Launch of LIVMARLI in Reimbursed Market in Jan 2024 China market potential: $100-150 million in ALGS and $200-250 million for three indications combined Feb 2022 May 2023 2H 2023 Jan 2024 Early Access Program NRDL Submission & Potential NRDL NDA Approval in China in Bo’Ao Negotiation Reim
Slide 17: 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 18: Hunterase – Early Commercialization in Non-reimbursed Market
Hunterase® – Early Hunterase® Commercialization The Only In Non-reimbursed ERT Approved for MPS II LaunchedMarket in China Identification of new patients accelerates, and commercial insurance coverage expands Hunterase Commercial Updates Overview of MPS II MPS II is a rare, disabling and life- MPS M
Slide 19: CAN106
CAN106 Clinically De-risked, Near Commercialization, Targeting Multiple Indications with Significant Market Potential
Slide 20: CAN106 – Long-acting Anti-C5 Therapy for Complement Disorders
CAN106 – Long-acting Anti-C5 Therapy for Complement Disorders Novel, long-acting monoclonal antibody for the treatment of complement-mediated diseases, including PNH, myasthenia gravis (MG) and various other complement-mediated diseases that are targeted by anti-C5 antibodies. Recent Highlights Mech
Slide 21: C5 Inhibition Remains the Current Standard of Care for PNH
C5 Iinhibition Remains the Current Standard of Care for PNH Recent studies raise concerns about potential risks associated with proximal inhibitors in anti -complement treatment. • Enhanced complement inhibition leads to heightened breakthrough hemolysis risk upon inhibition loss, due to increased s
Slide 22: Dose-dependent, Rapid and Substantial Effects on LDH and Hgb
Dose-dependent, Rapid and Substantial Effects on LDH and Hgb Cohort 3: LDH, a biomarker of hemolysis, was reduced by 81% at Week 13. All subjects showed rapid and sustained LDH reduction, with 88% (7/8) subjects in Cohort 3 achieving LDH＜1.5 x ULN at least once after Week 3 Mean LDH (+/- SD) over ti
Slide 23: CAN106 – Potential for Other Complement Disorders
CAN106 – Potential for Other 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 Population in China
Slide 24: CAN106 – Differentiated Follow-On Indication Strategy
CAN106 – Differentiated Follow-On Indication Strategy CAN106’s multi-indication potential, coupled with the limited access to the current available anti-C5 therapies in most parts of the world, allows for versatile indication expansion and go-to-market strategies to maximize its global commercial va
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, Gerald Cox, MD, Jean Francis, Richard Polisson, Sushrut Waikar, Brian MD PhD MD MD, MHSc MD, MPH Weinshenker, MD • Br
Slide 26: CAN008: Development in Newly Diagnosed GBM
CAN008: Development in Newly Diagnosed GBM
Slide 27: 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 • An independent data monitoring committee completed an interim analysis and CD95L binds to its receptor, CD95, on the ce
Slide 28: CAN008 – Ongoing Phase 2 Registrational Trial in Newly Diagnosed GBM
CAN008 – Ongoing Phase 2 Registrational Trial in Newly Diagnosed GBM 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 completion in
Slide 29: Next-gen Gene Therapy Pipeline
Next-gen Gene Therapy Pipeline CANbridge’s Next-Generation Innovation and Process Development Facility in Burlington, Massachusetts
Slide 30: Proprietary Gene Therapy Technology Platform & Powerful Discovery Engine
Proprietary Gene Therapy Technology Platform & Powerful Discovery Engine We continue to invest in gene therapy as our next-generation technology development and expand our in-house R&D activity to generate promising leads for treatment of rare diseases Collaborations in Neuromuscular Disorders Poten
Slide 31: CAN203 – Second Generation Gene Therapy for Spinal Muscular Atrophy
CAN203 – Second Generation Gene Therapy for Spinal Muscular Atrophy SMA Pathophysiology Illustration CAN203 • Second-gen gene therapy to potentially treat SMA type 1-3 (from infants to adult patients) • Endogenous promoter: regulated, targeted tissue expression to avoid unwanted toxicity in liver an
Slide 32: CAN203 - Preclinical Data Presented at 2022 and 2023 ASGCT
CAN203 - Preclinical Data Presented at 2022 and 2023 ASGCT Head-to-head comparison between the 2nd-generation vector (CAN203) and the reference vector (designed similar to Zolgensma®): both IV* and ICV* injections demonstrate therapeutic advantages 2nd gen vector: Endogenous SMN1 promoter isolated a
Slide 33: CANbridge Innovative AAV Capsid Platform: BEST2AAV
CANbridge Innovative AAV Capsid Platform: BEST2AAV Addressing limitations of current AAV technology CANbridge BEST2 Tissue Specific Delivery Platform PoC of BEST 2AAV in vitro in Myotubes and Non-Muscle Cells Programmed AAV AAV with BEST2 AAV Control AAV w/o BEST2 AAV-BEST2 AAV-BEST2 GPS1 Capsid BES
Slide 34: Comparison of CANbridge BEST2 with Other Novel Capsid Approaches
Comparison of CANbridge BEST2 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 all
Slide 35: Financial Review
Financial Review
Slide 36: 1H 2023 Financial Highlights
1H 2023 Financial Highlights RMB Million R&D expenses including 24.2% 43.1 Revenue milestones payment R&D Expenses 463.1 34.7 158.3 -9.7% YoY decrease of RMB 15.3M. YoY increase of RMB 8.4M from Due to lower upfront and 143.0 milestone payments made increase of sales 283.6 of Hunterase® to our licen
Slide 37: Q&A
Q&A CANBRIDGE-B 01228. HK www. canbridgepharma. com
Slide 38: THANK YOU
THANK YOU Contact IR@canbridgepharma.com CANBRIDGE-B 01228. HK www. canbridgepharma. com WeChat Official Account 37
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