Partnering for Impact Human Health Therapeutics

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1 Partnering for Impact Human Health Therapeutics

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3 Bernard Massie General Manager Human Health Therapeutics Partnering for Impact in Biologics and Vaccines NRC s Human Health Therapeutics (HHT) portfolio helps firms advance their biologics and vaccine pipelines. Our competitive advantage rests upon our unique biotechnology expertise, know-how and infrastructure, covering the entire value chain from design to the Investigational New Drug (IND) submission required for clinical trials. Our data allows clients to generate confidence, attract financing, and increase valuation on the path to market. And by fostering biomanufacturing in Canada, we re helping to increase returns on national innovation investments. On behalf of the HHT management team, I invite you to discover the impact we can generate by working together!

4 Management Team Lynne LeSauteur Director of R&D and Program Leader Biologics and Biomanufacturing Danica Stanimirovic Director of R&D and Program Leader Therapeutics Beyond Brain Barriers Lakshmi Krishnan Director of R&D and Program Leader Vaccines and Immunotherapeutics Frank Van Lier Director of R&D The National Research Council (NRC) is the Government of Canada s premier organization for research and development. Working with clients and partners, we provide innovation support, strategic research, scientific and technical services. NRC s Human Health Therapeutics (HHT) portfolio works hand in hand with industry to help firms achieve success and save costs. As an R&D partner of choice, NRC de-risks critical steps in the development of biologics and vaccines. We add value to partners products and accelerate their progress to market by overcoming challenges all along the value chain. Through R&D services, collaborations, and licences, our clients can access à la carte expertise or partner with us for co-development opportunities. We transfer IP assets and enabling technologies to our industrial clients to reinforce a strong, prosperous Canadian biomanufacturing sector with global impact. Meet with our experts and discover our technologies today!

5 Business Team Eileen Raymond Portfolio Business Advisor Tel.: cnrc-nrc.gc.ca April Luong Client Relationship Leader Tel.: cnrc-nrc.gc.ca Daniel Desmarteaux Client Relationship Leader Tel.: cnrc-nrc.gc.ca Stacey Nunes Client Relationship Leader Tel.: nrc-cnrc.gc.ca Our focus 1 Fundamental 2 Target Selection 3 Research and Validation Candidate Design and Optimization Our role in the value chain Product Co-development R&D Services Strategic R&D Licensing Clinical Phase I & II Milestone Proof of Concept (PoC) 5 6 Clinical Phase III 7 Milestone New Drug Application (NDA) Preclinical Development Milestone Investigational New Drug (IND) Market 4

6 86% are repeat customers Focus on large molecules In 2016, 81% of the revenue generated by the top 10 selling pharmaceuticals came from biologics, compared to 7% in Source: Genetic Engineering and Biotechnology News, 2016 Programs Biologics and Biomanufacturing Developing biotherapeutics and bioprocesses Indications Cancer Inflammatory diseases Auto-immune diseases Technologies Novel targets Antibodies: monoclonal, bi-specific, ADCs Humanization and molecular modelling High-throughput cell-based assays Protein and vector production Process development and scale-up Purification Biophysical charvterisation Biomarkers, preclinical safety and efficacy Vaccines and Immunotherapeutics Developing vaccines and immune-modulating biologics Indications Targeting immuno-oncology and (re)emerging infections Cancer vaccines Cell therapy Infections in high risk populations Nosocomial infections and anti-microbial resistance pathogens Regulatory compliance and quality assurance (QA)

7 Our strategy is to leverage our strength in biotechnology, our ability to partner, and the high productivity and commitment of our staff to deliver impact for our partners and for Canada. Bernard Massie, General Manager, Human Health Therapeutics, NRC Technologies Carbohydrate, protein and lipid antigen targets Immunomodulation: carriers, adjuvants and delivery vectors Human immunology: high throughput, multiplex assays Flow cytometry: 18-color analysis and 6-way multimodal sorting Virus, virus-like particle, vector, protein, and bacteria production, purification and scale-up Biomarkers and preclinical safety, efficacy, bio-distribution, and mechanism of action Therapeutics Beyond Brain Barriers Enabling therapeutics to cross the blood brain barrier Indications Brain / central nervous system diseases Alzheimer s Parkinson s Brain cancer Epilepsy Technologies Portfolio of carrier molecules to shuttle biotherapeutics into the brain Chemical conjugation or fusion to couple NRC carriers with your antibodies, peptides, growth factors, imaging agents, enzymes and nanocarriers Preclinical testing and imaging of blood-brain barrier penetration and efficacy Target engagement and vascular biomarkers In vitro rodent and human blood-brain barrier models

8 Cell Culture Stainless 500L 50L 20L 7L 3L Cell Culture Single use 200L Facilities Advanced analytical platform Biophysical characterization Molecular weight and charge heterogeneity Post-translational modifications Purity Stability Solubility Aggregate formation Mass spectrometry (MS) Intact proteins Post-translational modifications Carbohydrates Lipids Other services Targeted biomarker quantification and mass spectrometry omics approaches Development of novel methods for bioanalysis Advanced nuclear magnetic resonance (NMR) structural analysis of proteins and carbohydrates Data packages to enable Investigational New Drug (IND) submissions

9 Microbial fermentation Stainless 1500L 750L 150L 75L 20L 14L 3.5L Cell culture pilot plant Production of antibodies, proteins, viruses, and virus-like particles Proprietary expression systems for transient and stable production: HEK293, CHO Process optimization and scale up of batch, fed-batch, and perfusion cultures up to 500 L In-process assay development Purification and analytical services Tech transfer to contract manufacturing organizations (CMOs) Microbial fermentation pilot plant Production of peptides, proteins, enzymes, nutraceuticals, organic acids, polymers, polysaccharides and probiotics Bioprocess optimization and scale up to 1,500 L Bacteria and yeast systems: E. coli, M. extorquens, Pichia, Saccharomyces Pathogen cultivation facilities up to BSL 3 Handling of methanoloxidizing microorganisms Testing of new monitoring/ control equipment and reagents Purification and analytical services Tech transfer to contract manufacturing organizations (CMOs) Preclinical in vivo facility Validation of therapeutic targets and biomarkers for peripheral and central diseases Pharmacokinetic and pharmacodynamics studies in animal models In vivo vaccine safety and efficacy studies for infectious diseases (level 2 & 3) Efficacy and immune mechanism of action testing in syngenic, xenogenic, orthotopic and transgenic cancer models Development of novel in vivo imaging methods and contrast agents for molecular imaging Data packages to enable Investigational New Drug (IND) submissions

10 On behalf of the entire Zymeworks team, we are delighted to let everybody know what a world-class group of researchers and infrastructure exists at NRC We have t remendously benefited from what NRC has to offer, and I am extremely confident about what we re about to do together: develop best-in-class biologics to treat cancer patients. NRC is a fantastic trendsetter. This is the place to be, this is the group to do your research with. Dr. Ali Tehrani, President and CEO, Zymeworks Impact Our clients 82% Found unique offerings at NRC 61% Submitting IND next 3 years 56% Pre-revenue 74% < 50 employees $16.4M Increased investments since interaction with NRC $108M Increased market valuation since interaction with NRC

11 Milestones Projects with NRC Year Milestone Company Molecule / Technology Indication / Application 2017 Phase 1 Formation Biologics Antibody-drug conjugate Solid tumours 2016 Phase 1 Zymeworks Bi-specific antibody Breast, gastric, and ovarian cancer 2016 Phase 1 VBI Vaccines Virus-like particle Cytomegalovirus 2016 Phase 1 Alethia Biotherapeutics Monoclonal antibody Metastatic carcinomas 2016 Phase 1-2 Cansino Vaccine Ebola 2015 Phase 1 PREVENT Vaccine Streptococcus Group A 2015 Market NPS Biopharmaceuticals Hormonal therapy Hypoparathyroidism 2012 Phase 1-2 Helix Biopharma Urease single-domain antibody conjugate Lung cancer 2012 Phase 3 Oncolytics Oncolytic virus Cancer HHT in numbers 350 Experts 3 Programs 72 Projects 4 Sites Candidates in the pipeline 3 Biosafety levels Licencing opportunities > $45M invested in human health by NRC each year

12 Ideal partner for small and medium- sized enterprises (SMEs) When you work with NRC, you can look forward to an experienced team backed by extensive technology and infrastructure assets. While we work with firms of all sizes, these resources make us an especially attractive partner for SMEs. Why partner with NRC? Recognized expertise in biotechnology Track record of industry collaborations State-of-the art infrastructure From target selection to GMP-like bioprocesses Partnering for Impact Enhanced ability to attract investment High-quality job creation Progression of products and technologies towards commercialization Increased business expenditures on R&D (BERD) ISBN Cat. no.: NR16-146/2017E