Day 1:11:00am-12:00pm Panel Slides (Nov 18) Access to Innovation Conference 2019
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The document discusses genome-wide sequencing for diagnosis of rare diseases in Canada. It notes the current unmet clinical need, with specialists taking 3-5 years on average to diagnose rare diseases and 40% of patients receiving more than 3 misdiagnoses. Access to clinical exome sequencing varies across Canada. The goal of the Partnership discussed is to ensure access to clinical genome-wide sequencing as standard of care for Canadians at risk for a serious genetic condition who would benefit from timely diagnosis and intervention. It will include access to sequencing, data governance, a pan-Canadian data ecosystem for sharing to catalyze innovation, engagement of patients/communities and health professionals, and establishment of a rare disease cohort and diagnostic ecosystem to scale data.
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Day 1:11:00am-12:00pm Panel Slides (Nov 18) Access to Innovation Conference 2019
- 1. Genome-Wide Sequencing for Diagnosis of Rare Diseases Marc LePage President and CEO, Genome Canada
- 3. 800/yr 0/yr 50/yr 200/yr 100/yr 30/yr 50/yr 2018 site reported data Canada: 37,242,571 3 30/yr Access to clinical exome sequencing for rare disease is variable across Canada
- 5. access to genome-wide sequencing data governance to tackle social, economic and policy issues a pan-Canadian data ecosystem for sharing to catalyze innovation a rare disease cohort to scale data patient and community engagement to align genomic solutions with relevant problems and health-care professional engagement and education to optimize precision health delivery and patient care
- 6. Pan-Canadian Rare Disease Diagnostic Ecosystem• The ecosystem will allow data sharing between institutions, across jurisdictional boundaries and between clinical and research settings • Three tiers are envisaged • local clinical sites storing raw data • provincial staging areas for data harmonization and • a web portal to query provincial data for coded or aggregated results • An overarching goal is the passive collection of data generated during clinical care to be leveraged for secondary use in research 6
- 10. The ECOGENENE-21 Clinical Research toolbox Daniel Gaudet MD PhD ECOGENE 21 and Clinical Lipidology and Rare Genetic Lipid Disorders Unit Community Gene Medicine Center Département of Médecine, Université de Montréal Chicoutimi, Qc
- 11. Healtcare providers Industry/ biotechs -Health decision makers -payers Public agencies Research centers
- 13. Ecogene-21 Highlights n ECOGENE-21 is a non-for-profit organization dedicated to developing connected clinical research capacity for rare diseases prevention and gene/oligonucleotide-based Therapies; n EOGENE-21 operates as a biocluster having different, complementary platforms n Goal: Access
- 14. E-21 Main Platforms n Network of clinical and translational research centers; n Niche Clinical research Organisation (CRO) for rare or severe diseases; n Shareable experience with gene/oligonucleotide-based therapies; n A clinical research concierge service for patients associations and other stakeholders; n A world class Biobank and Biorepository (operated by Genome Quebec); n An innovation platform/lab
- 15. Integrating Concierge Clinical Research in the ECOGENE-21 Portfolio for Rare Diseases n Patients perspective n Healthcare providers perspective n Health decision makers perspective n Payers perspective n Industry perspective n Public agencies / research groups perspective
- 16. E-21 Concierge Service key words n Driven by unmet patients needs; n Provides connected solutions to complex issues; n Flexibility; n Creative approaches; n ACCESS;
- 17. ADigital Solution to Empower Patients to Manage their Health chronicallysimple.com/CORD
- 18. 11/21/19 CONFIDENTIAL2 Patient Experience § Built by a rare disease patient, every aspect of the tool was created from the patient/caregiver lens § Chronically Simple is truly a one-stop, centralized hub for every important facet of the patient’s administrative burden that comes with living with a chronic disease. § Patients have to be informed to be their own best advocates. Having all medical information in one secure location empowers patients to manage their health. chronicallysimple.com/CORD
- 19. 11/21/19 CONFIDENTIAL3 Caregiver Experience § Partnered with Brenda in her early years and of managing the dreaded “binder” of test results and imaging § Brenda had taken her son to over 200 appointments in his first year and was looking for a solution to manage the many appointments, notes for the doctors, results and expenses § She was also worried about the continuity of her son’s care should something happen to her. How could all of her knowledge be transferred to another care team member, without something being missed? chronicallysimple.com/CORD
- 20. 11/21/19 CONFIDENTIAL4 Our Healthcare Solution § Built on a solid understanding of the Canadian healthcare system: the patient journey, security & privacy requirements and information storage § Ability to scale globally with Innomar and ABC § Web-based and mobile application allows user to access all their medical information from any where and to update information & share in real time chronicallysimple.com/CORD
- 21. 11/21/19 CONFIDENTIAL5 Day at a Glance Healthcare Providers Medical Documents This feature shows you what’s in store for the day ahead on one screen. Store all your healthcare providers’ details and associate them with appointments, prescriptions and medical records. Keep all your medical records, diagnostic images and test results in one secure place. Take your past results to new appointments to ensure continuity of care. Appointment Reminders Expenses Medication Tracker Don’t forget your appointments ever again! Chronically Simple will send you reminders. Never lose a parking receipt again! Keeping track of all your expenses will make doing your taxes much less stressful. Store all your current and previous medications. Receive reminders when it’s time for a refill, ensuring you never run out again. chronicallysimple.com/CORD
- 23. Regulatory Oversight of Cell and Gene Therapies in Canada Jian Wang, MD, PhD Division Manager, Clinical Evaluation Division – Haematology/ Oncology Biologics and Genetic Therapies Directorate Health Canada CORD Conference on Personalized Healthcare & Medicine 18-19 November, 2019, Toronto 1
- 24. No Conflict of Interest to Declare 2
- 25. Canadian Legislation Pertinent to the Regulation of Gene and Cell Therapies Food and Drugs Act Gene Therapy is regulated under Food and Drug Regulations § Gene therapy is the introduction, removal, or change in the content of a person’s genetic code with the goal of treating or curing a disease. § Chimeric antigen receptor T cells (CAR T cells) are T cells that have been genetically engineered to produce an artificial T-cell receptor for use in immunotherapy. Once the receptor binds to a tumor antigen, the T-cell is stimulated to attack the malignant cells. Food and Drug Regulations CTO Regulations Medical Device Regulations Drug / Medical Device Combination Products Policy 3
- 26. Regulations Governing Cell Therapies in Canada 4
- 27. Regulatory Challenges: Advanced Therapeutic Products (2019) § The speed at which innovative products can be developed, the method with which they are made or distributed, and how data can be collected, has resulted in a shift away from the traditional product development model for which the current regulations are based § Some health products are so novel and distinct that it is difficult for them to meet the current regulatory requirements § Lack of appropriate regulatory oversight for continuously changing products and innovative business practices 5 3D PrintingGene Editing Big Data Artificial Intelligence Advanced Cell Therapies Novel Drug Delivery
- 28. Remove product from market (or Schedule) Health Canada to decide the product can be regulated in existing framework Iterative consultation with partners to design rules for market access and address uncertainties. Item is on Schedule G and tailored requirements are published Market Access: 1. Individual license OR 2. Order of permission The Regulatory Sandbox Follow regulatory requirements of Food and Drugs Act Ye s Requirements may be adjusted based on evidence generated from market access and ongoing consultations $ No Amend or create new regulations A potential candidate is identified Cells, Tissues & Organs Blood Drugs Medical Devices 6 Advanced Therapeutic Products and The Regulatory Sandbox
- 29. Clinical Trial Application • Screening – 7 days • Review – 30 days (default) • Pre-filing meeting is “Optional” Pre-Market Filling Regular Review Pathway • Screening – 45 days • Review – 300 days • Pre-filing meeting is “Optional” Notice of Compliance (NOC) with Conditions (NOC/c) Pathway • Screening – 25 days • Review – 200 days • Pre-filing meeting is “Required” Priority Review Pathway • Screening – 25 days • Review – 180 days • Pre-filing meeting is “Recommended” Submission Pathways and Performance Timelines
- 30. Stakeholders / Sponsors Pre-CTA/ Consultation Meetings – Clinical and/or Quality Pre-NDS – Clinical and/or Quality Pre-SNDS – Clinical and/or Quality Office of Regulatory Affairs Engagement with Sponsors (Pre-filing Meetings) CTA Reviewers Pre-Market Reviewers Schedule a meeting at least 3 months ahead hc.bgtd.ora.sc@canada.ca
- 32. Collaborative, Capital-Efficient & Scalable Translation of Advanced Therapies Nov 18th, 2019 Michael H. May PhD President & CEO
- 33. Commercializing Living Therapies 2 Advanced Therapies – One of the Hottest Sectors in Biotech • Capital Continues to Flow Into the Sector: 2018 was Record Year, 2019 Off to a Slow Start o $13B raised in 2018; $7.4B raised by end Q3 2019 • Mega Rounds Continue to Grow: 22 Company Deals ≥ $100M o Increasing trend to launch with large sized early stage rounds § Century Therapeutics: Raised $250M Series A § Allogene Therapeutics: Raised $411M Series A § Sana Biotechnology: Raised $215M Series A • IPOs: 17 RM Company IPOs in 2018, 6 in 2019 YTD o Over 50% are trading above initial share price o 2018 totals just short of 2014 highs (2014; 21 IPOs, $1.5B Raised) • Public Markets are Receptive to Cell & Gene Therapies – RM Index up 19.2% in 2018 • RM Company Valuations Continue to Rise – Currently 2.6x Higher than Biotech Overall (2019 YTD) • Big Pharma Continues to be Engaged – Increasing No. of Acquisitions to Fill Pipeline ADVANCED THERAPIES Cell Therapy Cellular Immunotherapy Gene Therapy Bio-moleculesBio-materials
- 34. Commercializing Living Therapies 3 Canadian Cell & Gene Therapy Landscape – Small but Growing Raised $20M in investment & co- development (Nov 2019) Raised $82.5M (Sep 2019) Raised $42M (Jan 2019) Source: Pitchbook Capital Invested Deal Count $14.33M $0.30M $6.43M $13.32M $17.18M $24.94M $53.09M $150.42M $120.85M $72.50M $162.60M 0 5 10 15 20 25 30 $0.00M $20.00M $40.00M $60.00M $80.00M $100.00M $120.00M $140.00M $160.00M $180.00M 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 YTD Capital Invested (CAN) Deal Count (CAN) $4.3B $162M 54% 20% 20% 4% 2% Ontario Quebec BC Alberta Manitoba Deals Are Dominated by Three Provinces US Fundraising Dwarfs Canadian Companies Capital Invested, 2019 YTD (all figures USD) Notable Recent Canadian Deals Cell Therapy trials in Canada Canadian academic sponsored International academic sponsored Industry sponsored ~80 cell therapy trials
- 35. Commercializing Living Therapies 4 Business Model: Collaborative, Capital-Efficient & Sustainable NETWORKS Academia Industry Investor Proprietary Development Stakeholder Engagement Investment BUSINESS UNITS VALUE CENTRES Deal Flow Capabilities Sustainability OUTCOMES IP Market Needs
- 37. THE PROBLEM • Science is outpacing our historical business models • Precision medicine -> monolithic diseases are now understood to be many sub diseases • Smaller patient populations • Large markets are now multiple small markets • Cost to develop therapies has not gone down in parallel • Historical business model solution • Smaller market à charge a higher price OR • Effective therapies not being developed for small markets Open Science for Children’s Health
- 38. THE HYPOTHESIS • Open science can be used to reduce the cost and risk of drug development and make small market drug development viable at affordable prices • Open Science: A commitment to rapid multilateral sharing of knowledge, results, data and materials without patent restrictions • Open science can bring in funds from foundations, granting agencies, academic institutions and corporate “donations” • Commercial opportunities will remain: • Discovery/development costs are reduced à sustainable pricing can be lower • Alternative IP assets (regulatory data and market exclusivities) can attract partners to take-up de-risked asset through registration, manufacturing, and distribution Open Science for Children’s Health
- 39. THE EXPERIMENT • Select a disease that: • Represents an urgent medical need • Has a limited commercial market or doesn’t fit historical business models • Known genetic target • Our scientific founders have insight into the target • Favourable clinical path • Objective: • Access grants, foundations and corporate partners • Share (give AND receive) scientific insights • Aggressively promote open science • Find motivated partners • Retain IND and NDA data to gain regulatory exclusivity for out-licencing Open Science for Children’s Health
- 40. A Genes to Affordable Medicines Corporate Structure Agora Open Science Trust (Charity) Open Science Drug Discovery and Development M4K Pharma Inc. M4ND Pharma Inc. M4ID Pharma Inc. M4?? Pharma Inc. Open Science for Children’s Health
- 41. WHY USE A CORPORATE STRUCTURE Open Science for Children’s Health • Corporate structure allows us to apply for grants and foundation awards • Act as a hub to “outsource” work to collaboration partners • Working with industry CROs and academic institutions • “Virtual” biotech model lowers costs • Aggregating and aligning academic and industry collaborators to move along drug development path • Creates a vehicle which can enter into agreements to sell and/or partner its assets
- 42. Open Science for Children’s Health DIFFUSE INTRINSIC PONTINE GLIOMA (DIPG)
- 43. HOW IS THE EXPERIMENT GOING? • Started lead series development program December 2017 • Ontario Institute of Cancer Research (Med Chem, project management) • Charles River Laboratories (Med Chem, Assays) • Reaction Biology Corp (Assays) • Structural Genomics Consortium (Structural biology, assays) • University of Toronto • Oxford University • University of North Carolina • ICR UK (Med chem, in vivo PD model) • Sant Joan de Deu, Barcelona (in vivo PD model) • Tufts University (assays) • University of Pennsylvania (in vivo PD model) • The Brain Tumour Charity • Children’s National (in vivo PK) Open Science for Children’s Health
- 44. HOW IS THE EXPERIMENT GOING? • Monthly project meetings broadcast and available on YouTube • Attracted pharma to discuss ALK2 project they had abandoned • Scientific and clinician providing input • https://m4kpharma.com/blog/ • Approximately 400 compounds synthesized to date • 5 compounds met TPP and selected to enter preclinical candidate selection studies in 2020 -> Select one compound to enter IND enabling studies in 2021 Open Science for Children’s Health
- 45. PRACTICAL BENEFITS REALIZED FROM OPEN SCIENCE Open Science for Children’s Health q Partners have provided services at a reduced price or, in some cases, for free q Dramatically lowered costs q Allowed us to achieve much more than originally planned q Roughly 4x as many compounds synthesized q >300 versus 75 in original grant q Extra funds used for more assays and initial efficacy studies q Goal was to have one compound that met the Target Product Profile
- 46. BROADER OPEN SCIENCE ADVANTAGES Open Science for Children’s Health Micro advantages: • Attractive to granting institutions • Academic work is unencumbered • Facilitates collaboration Macro advantages: • Cost savings to the pharmaceutical industry by avoiding multiple companies running the same program in parallel or by avoiding previous failures. • Provides learnings about “dead ends” to the medical research community.
- 47. Contact Max Morgan, JD, LLM SGC Director of Policy & Legal max.morgan@mail.utoronto.ca FUNDING PARTNERS The SGC is a registered charity (number 1097737) that receives funds from AbbVie, Bayer Pharma AG, Boehringer Ingelheim, Canada Foundation for Innovation, Eshelman Institute for Innovation, Genome Canada through Ontario Genomics Institute [OGI-055], Innovative Medicines Initiative (EU/EFPIA) [ULTRA-DD grant no. 115766], Janssen, Merck KGaA, Darmstadt, Germany, MSD, Novartis Pharma AG, Ontario Ministry of Research, Innovation and Science (MRIS), Pfizer, São Paulo Research Foundation- FAPESP, Takeda, and Wellcome [106169/ZZ14/Z]. Open Science for Children’s Health
- 48. REGULATORY EXCLUSIVITY VS. PATENTS
- 49. REGULATORY EXCLUSIVITY VS. PATENTS Regulatory exclusivities: • Like patents, shield a product from generic competition • Consistent with open science: – Are not invalidated by prior disclosure/open sharing/ collaboration • Virtually costless to obtain and enforce • Not subject to challenge by competitors • Provide a period of market protection that is certain ex ante
- 50. POLICY RECOMMENDATIONS • Translational Funding Opportunities for Open Drug Discovery (‘Push’ Mechanisms) – Public translational funding programs should support open science drug discovery businesses – Public funding of open trials for clinical proof of concept • Enabling Digital Infrastructure for Open Science Projects – E.g. open drug development data repository • Voluntary Business Incentives for Open Drug Development (‘Pull’ Mechanisms) – ‘Open science” regulatory exclusivity extension • Quid pro quos: (1) open data, (2) no patents, (3) affordable pricing – Regulator support – e.g. fast-track designation, priority review for ‘open science’ programs
- 51. Commercializing Living Therapies 5 Impact of Strategic Investment in Ecosystem § Global Outreach & Presentations § Industry Boards & Committees § Industry Initiatives Academic & Industry Networks Academic Partnerships Global Hubs – Scaling the Model Specialized Infrastructure Industry Partnerships Company Creation and Scaling Ventures $150M RM Seed Fund