Canbridge Pharmaceuticals Inc. · HKEX:1228
Canbridge Pharmaceuticals — Investor Presentation (August 2023)
51 slides from Canbridge Pharmaceuticals's investor presentation, August 2023
Slide 1: Corporate Presentation Aug 2023
CANbridge Pharmaceuticals Corporate 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: Strong Investor Base
Strong Investor Base Raised total capital of $357m* since 2015 2015: Series A $13m 2017: Series B $54m 2019: Series C $46m 2020: Series D $98m 2021: Series E $58m 2021: HKEX IPO $88m *Currency: $ USD 4
Slide 6: Experienced Management Team
Experienced ExperiencedManagement Management Team 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 in medical and pharmaceutical companies • Former Founding General Manag
Slide 7: 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 8: Track Record of Sourcing and Developing Innovative Therapies
Track Record of Sourcing and Developing Innovative Therapies ✓ Agile execution capabilities on Hunterase ® with 6 months from licensing to NDA submission, then 14 months to approval ✓ Solid clinical progress made on CAN108 with 9 months from licensing to NDA submission, then 17 months to approval ✓
Slide 9: 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 10: 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 11: Next Generation Gene Therapy Portfolio in Development
Next Generation Gene Therapy Portfolio in Development Gene therapy holds the promise to transform treatments for LSDs and neuromuscular diseases from ameliorative to curative CANbridge In-house Tech Platform Pipeline In-licensed Gene Therapy Programs US R&D Center. Burlington, MA Candidate Discovery
Slide 12: Business Overview & Opportunities
Business Overview & Opportunities
Slide 13: Proven Large Global Rare Disease Market Opportunity
Proven Large Global Rare Disease Market Opportunity Global Rare Disease Drug Market1 (By Region) Rare Disease Prevalence in U.S., EU & China (est.) (US$ billion) 383 China Rare Disease Prevalence > 100 million (est)2 106.6-128.0 ✓ Favorable regulatory reform ✓ Innovative medicines 112 29.2% (Million
Slide 14: A Rapidly Evolving Market for Developing Rare Diseases Products
A Rapidly Evolving Market for Developing Rare Diseases Products Emerging Favorable Regulatory Framework Hunterase launch Livmarli NDA Special rare disease funding Potential Commercial Path in self-pay market approved in China Global Expansion to Further Improve ASP 1983 2000 2018 2020 2021 2023 Near
Slide 15: 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 16: 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 17: Recent Business Highlights
Recent Business Highlights Livmarli CAN106 CAN008 CAN103 CAN203 • IDMC 1 conducted a mid- • Received China NMPA • Reported positive study analysis of the Phase • Initiated dosing in • Presented ICV data that approval for marketing in proof-of-concept data 2 trial in Chinese patients Phase 2 trial in
Slide 18: Pipeline Portfolio Update
Pipeline Portfolio Update
Slide 19: 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 20: 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 21: Livmarli (CAN108): Near-term Launch in Rare Cholestatic Liver Diseases in China
Livmarli® (CAN108): Near-term Launch in Rare Cholestatic Liver Diseases in China 20
Slide 22: 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 23: 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 24: Long-term Data 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 25: 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 & NRDL Reimbursement in NDA Approval in China in Bo’Ao Negotiati
Slide 26: LIVMARLI Key Launch Initiatives
LIVMARLI Key Launch Initiatives LIVMARLI as New Disease Awareness Patient Diagnosis and Standard of Care and Identification ‐ Leverage maralixibat efficacy in ‐ Increase physician attention to pruritus and ‐ Boost patient registration in key pruritus relief and sBA reduction the impact on patients’
Slide 27: CAN106
CAN106 Clinically De-risked, Near Commercialization, Targeting Multiple Indications with Significant Market Potential
Slide 28: 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 29: 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 30: Positive Preliminary Results From Phase 1b/2 Trial In PNH Patients
CANbridge Reported Positive Preliminary Results From Phase 1b/2 Trial In PNH Patients Safety & Tolerability • CAN106 was well-tolerated and safe. 6 subjects (37.5%), up until the data cutoff, experienced 18 drug-related AEs, with all AEs being mild and moderate. No drug-related SAE, and no drug-rela
Slide 31: 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 32: 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 33: 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 34: 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 35: CAN008: Development in Newly Diagnosed GBM
CAN008: Development in Newly Diagnosed GBM
Slide 36: 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 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 37: 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 38: 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 39: Next-gen Gene Therapy Pipeline
Next-gen Gene Therapy Pipeline CANbridge’s Next-Generation Innovation and Process Development Facility in Burlington, Massachusetts
Slide 40: 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 41: 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 42: 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 43: 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 44: 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 45: Gene Therapies to Treat LSDs
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 Application to Lysosomal Storage Diseases (LSDs) LSDs are a group of over 70 diseases that are characterized by lysosomal dysfunction, most of w
Slide 46: Financial Review
Financial Review
Slide 47: 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 milestone payments made increase of sales 283.6 of Hunterase® to our licensi
Slide 48: Outlook
Outlook
Slide 49: 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 50: Q&A
Q&A CANBRIDGE-B 01228. HK www. canbridgepharma. com
Slide 51: Thank You
THANK YOU Contact IR@canbridgepharma.com CANBRIDGE-B 01228. HK www. canbridgepharma. com WeChat Official Account 50
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