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China Rare Disease Market: Large and Untapped Opportunity

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China Rare Disease Market: Large and Untapped Opportunity Delivering Life-Changing Therapies to Patients June 2019 Mission and Vision To be a global biopharmaceutical company delivering life-changing therapeutics built upon a foundation in China CANbridge Pharmaceuticals 2 Investment Highlights Pioneer in Balanced Leverage Comprehensive Deep, Advanced China’s Orphan Business Technology Global Solution Portfolio Drug Market Model Partnerships Provider Leadership: Foundation: Leverage Portfolio: Strategy: Mission: Sole Deputy Director near term revenue from 1 marketed, 2 near- Partner with leading To be a global General from industry oncology to support commercial, 5 rare academic institutions biopharmaceutical for China’s Alliance for orphan drug disease and 2 and biopharma company delivering Rare Disease (CARD) development oncology clinical companies life-changing candidates therapeutics built upon Experience: Future: Execution: a foundation in China CANbridge Team The premier partner for De-risked: Partnership with Wuxi launched first rare China and global rare Most candidates target and GC Pharma to disease drug in China disease drug commercially validated prosecute product development pathways candidates in China Visionary Management Team Thought leaders and political influencers directly involved in shaping the future of China’s orphan drug market CANbridge Pharmaceuticals 3 World Class Leadership Team with Deep Experience James Xue, Ph.D., M.B.A Ben Wu, M.B.A. • Brings significant orphan drug experience from across Asia Founder, Chairman, Head of Rare • Joined Genzyme in 2008 to pioneer the company’s efforts to expand rare Disease Business Chief Executive Officer disease business into Hong Kong; held various management positions covering Southeast Asia, Taiwan and China; Spent 14 years at JNJ where he held various leadership positions Fangzhou Cheng • Brings 18 years of China MNC oncology sales and marketing and commercial VP of China BU Head management experience • Previously Senior Director and China Oncology BU Head of Celgene; Sales Director at Pfizer China; and member of Iressa marketing team at AstraZeneca China A veteran entrepreneur with extensive experience from small biotech to multi-national • More than 15 years experience in the healthcare sector globally, as an Glenn Hassan investor, investment banker, and strategy consultant. biopharmaceutical companies across the US CFO and CBO and China • Previously, Director of Investment Banking at China Renaissance; Portfolio Manager and Senior Analyst at Leerink Capital; Senior Therapeutic Analyst at Managed the launch of several life-saving drugs Citadel’s Surveyor Capital; Senior Healthcare Specialist at Fidelity for the treatment of hematologic cancer and rare Management & Research Company metabolic diseases in China including ® ® Thymoglobuline and Cerezyme Gerald Cox, M.D., Ph.D.• Previously Chief Medical Officer at Editas Medicine; Vice President of Clinical Chief Development Development, Rare Disease at Sanofi Genzyme Founding member and sole Deputy Director Strategist and Acting General of China’s Alliance for Rare Disease • Staff Physician in Genetics at Boston Children’s Hospital and Instructor in Chief Medical Officer Pediatrics at Harvard Medical School since 1994 (CARD) • Led the global clinical development of new treatments and world-wide approvals R&D Committee Member of China of Aldurazyme ® (laronidase), Cerdelga ® (eliglustat), and Elaprase ® (idursulfase) Pharmaceutical Innovation and Research in Japan Development Association (PHIRDA) James Geraghty, Ph.D. • Industry leader with 30 years of strategic and leadership experience Member of Advisory Committee of Joint Institute Board Director • Previously, North America Strategy and BD at Sanofi; 20 years at Genzyme, of Peking University Health Science Center and where his roles included SVP of International Development, President of University of Michigan Medical School Genzyme Europe, and General Manager of Genzyme's cardiovascular business • Board member of Orchard, Idera, etc CANbridge Pharmaceuticals 4 Pipeline Overview Estimated License License China Milestones/ Field Compound Indication Pre-clinical Ph I Ph II Ph III Marketed Partner Country Prevalence4 Development Path 2H 2019 – NDA CAN 1012 Greater 1 per MPS II1 Submission (China) / 1H (Hunterase) China3 166,000 2020 NDA Approval Complement Greater 1H 2020 – CAN 106 Varies Disorders China3 IND (China) Application Lysosomal 1 per 2H 2019 – CAN 103 Storage Global 100,000 IND (China) Application Disorder Greater 3 per 1H 2021 – CAN 105 Hemophilia China3 100,000 IND (China) Application Rare Disease Lysosomal 1 per 2H 2020 – CAN 104 Storage Global 40,000 IND (China) Application Disorder Metabolic Greater 4 per 2H 2020 – CAN 107 Disorder China3 100,000 IND (China) Application 22.30% CAN002 Oral China Of Cancer Marketed (CaphosolTM) Mucositis Patients HER2+ Breast Cancer Extended 20.30% CAN030 Adjuvant Greater Of Breast 2H 2019 / 1H 2020 (Neratinib, China3 Cancer NDA (China) Approval Nerlynx®) HER2+ Metastatic Patients Breast Cancer >=2nd line Oncology CAN008 Greater 2 per (CD95-Fc Glioblastoma China3 100,000 fusion protein) Esophageal CAN017 Global Squamous 22 per (anti-ErbB3 (exclude Cell 100,000 mAb) North America) Carcinoma 1 MPS II refers to Mucopolysaccharidosis type II; 2 CAN 101 is currently being marketed outside of China; 3 Greater China includes China, Hong Kong, Taiwan and Macau; 4 NCBI Website CANbridge Pharmaceuticals 5 Rare Disease Pipeline CANbridge Pharmaceuticals 6 China Rare Disease Market: Large and Untapped Opportunity The market potential for rare disease drugs in China is estimated to be 35-40% of the US market4 4X US Population1 1,423 USD$15.5b 1,423 Rare Diseases have been identified 2 1 40-50% Price Potential in China 5 Market 74 20% Access3 74 Patient Groups are Officially Registered in China1 Blue US China 1. China National Statistics 2016: Population: 1.3 Billion, New Births: 18 Mil / Yr. 2. As a reference: 17 oncology drug gain national reimbursement listing with 55% price reduction on average (Oct 2018) 3. Assume only 20% of China’s population have access to proper diagnosis & treatment access (compared to 58% Urban Population) 4. (400% * 45% * 20%) = 36% 5. 36% * US Orphan Drug Market $43 Billon in 2017 (Quintile IMS Orphan Drugs in US Oct 2018 Report) CANbridge Pharmaceuticals 7 8CANBRIDGE LIFE SCIENCES CONFIDENTIAL Some Cities and Provinces Have Reimbursed Rare Disease Products (Cerezyme Case Study) Panjin (2015) Shanxi (2019) Tianjin (2018) Ningxia (2013) Qingdao (2014) Jiaozuo (2015) Huaiyuan (2017) Shanghai (2012) Zhejiang (2016) Tongling (2017) Sanming (2015) Guangdong (2014) Kunming (2015) CANbridge Pharmaceuticals 8 Reaching Tipping Point for Rare Diseases in China 2018 2019 Disease Definition Drug Development Ecosystem Reimbursement May 11, 2018 May 23, 2018 Oct 24, 2018 March 13, 2019 First National Acceptance for overseas China Alliance for Establish annual Rare Disease List clinical data for treatment Rare Diseases (CARD) dynamic adjustment (121 diseases) of seriously life-threatening inauguration, the first mechanism for NRDL; and rare diseases cross disciplinary 2019 NRDL renewal to be alliance for policy completed by Sep 2019, July 10, 2018 advocacy in China and rare disease is Priority review Drugs for rare specifically highlighted for and pediatric diseases can be reimbursement change considered by CDE Nov 1, 2018 Expedited review NMPA releases a list of 48 much needed new orphan drugs marketed outside China 2018 is a breakthrough year in China for the acceleration and advancement of addressing treatment for rare disease, 2019 is shaping up to be the implementation year Source: NMPA / CFDA website CANbridge Pharmaceuticals 9 Unique Approach to Rare Diseases in China Leverage China-rooted Relationships to… • Access abbreviated regulatory development Referral pathways including Clinical Trial Waivers (CTW) Expertise Network Registries • Coordinate directly with China authorities to provide China government “one-stop shop” with predictable exposure to rare disease healthcare expenditures • Support development of Centers of Excellence Government CANbridge Pioneer China Orphan Drug Access • Establish strong provincial level market access team to drive reimbursement process on the local level • Reinvest profits into R&D research and development locally to build the first real Research Policy Funding innovative rare disease company in China and further strengthen government relationships CANbridge Pharmaceuticals 10 CAN 101 (Hunterase) Overview Enzyme Replacement Therapy (ERT) Indication: MPS II – lysosomal storage disease , X-linked recessive disorder Development Status: Filing China NDA by Q219, already marketed outside of China Significant Unmet Needs Potential Differentiation • MPS II is listed on National • Less anticipatedAEs Rare Disease List • Better potential efficacy • 5,000 – 6,000 patients in China1,2 Strategic Value First potential commercial asset for CANbridge Rare Disease Portfolio • High unmet need, No treatment available in China • ERT is standard of care for MPS II 1 Khan et cl, 2017 2 Orphanet CANbridge Pharmaceuticals 11 CAN 101 - Phase I/II Clinical Study Result * T-test (<0.05, Statisticallysignificant) † ANCOVA(<0.05, Statisticallysignificant) †† ANCOVA(<0.01, Statisticallysignificant) CANbridge Pharmaceuticals 12 CAN 101 Pre-Launch Preparation Focusing on Key Cities and Provinces Prioritization Criteria Financial Capabilities Beijing Socioeconomic Status Liaoning Tianjin Qingdao (C) Rare Disease Experts Ningxia Jiangsu Shanghai Patient
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