World stem cell and regenerative medicine conference, london – may 2013


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World stem cell and regenerative medicine conference, london – may 2013

  1. 1. The Cell Therapy Catapult Growing a UK cell therapy industry delivering health and wealth Keith Thompson CEO World Stem Cell and Regenerative Medicine Conference, London. May 21st 2013 Catapult is a Technology Strategy Board programme
  2. 2. The Launch of Catapults Hauser Report • Creating new manufacturing industries for the UK • Better exploiting the UK science base • “Grow and stick” • £200m+ assigned to TSB for 7 Catapults Hauser Criteria • > £10bn pa long term industrial revenue to UK • Exploit a strong existing UK science base • UK absorptive capacity, to capture and retain value • Learn from other countries with successful Technology Institutes • Translational activity to bridge investment gap • Persistence of funding against a long term perspective and low priority on becoming self-financing 2
  3. 3. 3 Cell Therapy Catapult • Significant unmet medical needs • World class science base • Complex development and production • NHS potential for both research and exploitation • Early enough for UK to establish a strong market position
  4. 4. Mind the (translational funding) gap 4 • Little evidence yet that new cell therapies can be developed, licensed and adopted successfully • Limited commercial investment • Limited precedents for valuable exits via IPO or acquisition • Large corporates are watching and waiting • Operational SME’s lack finance and breadth of resources for rapid advance Catapult
  5. 5. 6 Addressing Barriers Business • • • • Health Economics Business Models Reimbursement Partnering Manufacturing and Supply Chain • • • • • • Robustness & Reliability COGS & Scale up Characterisation & Analytical GMP CMC Delivery Clinical And Regulatory • • • • Complex Regulatory Landscape Pre Clinical Packages Clinical trial design NHS partnering Business Development Process Development Clinical Operations & Regulatory Affairs
  6. 6. Cell Therapy Catapult -2012 objectives • Identify premises, acquire and complete lease • Write strategic and detailed business plan, secure 5 year GFA • Establish company resource systems • Design organisation, recruit a world-class management team and Board • Design Facilities • Review UK GMP manufacturing capability • Build stakeholder relationships and project the Catapult Image • (corporates/SMEs/medical and academic researchers/technologists/DH/NHS/RCs/charities/patient groups) • Establish database of projects and initiate dialogue • Open for business November 2012 7
  7. 7. 8 Building The Catapult Catapult is a Technology Strategy Board programme
  8. 8. Assets -Money Finance • £70m core grant from TSB to March 2018 • £10m pa from other grant funders • £10m pa from industry contracts We use our assets to accelerate innovation. Not reliant upon IP for future funding 9
  9. 9. Assets -Facilities and Teams • Facilities • 1200 sq m on 12th floor • Capacity for 80-100 people • Clinical research cluster 10 • Business Team • Business development • Health economics • Business models • Translational Labs and Team • Process development • Analytical development • GMP process proving • Clinical Trial and Regulatory Team • Regulatory • Clinical operations
  10. 10. Cell Therapy Catapult Board Members and Executive Team Dr John Brown, CBE, FRSE Chairman of the Board Keith Thompson Chief Executive Officer Tim Edwards Non Executive Board Director Matthew Durdy Chief Business Officer Nick Higgins Non Executive Board Director Professor Marc Turner Non Executive Board Director Professor Michael Whitaker Non Executive Board Director Dr Zahid Latif Non Executive Board Director Dr Natalie Mount Chief Clinical Officer Dr Stephen Ward Chief Operating Officer Professor Johan Hyllner Chief Scientific Officer 11
  11. 11. Cell Therapy Catapult Outputs Investible therapies (Rounded Phase 2 data package) Industry assisted over barriers Novel technologies licenced to cell therapy companies Contract research which enhances capability of the industry Standards, quality systems, guidance & co-ordinated framework Skilled, trained and experienced professionals 12
  12. 12. 13 The UK ATMP Manufacturing Landscape Catapult is a Technology Strategy Board programme
  13. 13. ATMP GMP capability study To document and analyse • Physical Capacity • Regulatory status • Human capability • Process capability To allow • Catapult to place work with appropriate facilities • To direct inward investors • To form the basis of a UK Strategy 14
  14. 14. • 21 licensed facilities, mostly academic or public sector •12 sites analysed • 51 manufacturing cleanrooms • 55 parallel processes • 94 full time staff • 42 part time staff • 44% availability 2013 Grade of cleanroom Grade B Grade C Grade B/C/D with isolator Number 24 10 17 Facility Process Experience gene modification viral vector cell banking cells from tissue IPS HESC 3D 2D adherent suspension 0 2 4 6 8 Number of Facilities 10 12
  15. 15. ATMP GMP Manufacturing Capability study • Future capability • Expansion planned in 4 academic facilities • Licenses planned in 8 facilities • 2 commercial CMOs expressing interest in expanding into Cell therapy • Strategic planning for Phase 3 and in market supply • Catapult to collaborate on establishment of CMO 16
  16. 16. Regulatory Permissions • Catapult building strong clinical and regulatory team • Interact with regulators to improve clarity and speed • Clinical access to NHS • Clinical trial sponsor 17
  17. 17. HRA vision and ambition To make the UK a great place to do research, where more money invested in research goes into carrying out relevant, good quality research 18 • Greater numbers of people can and do take part in health research, and continue to feel safe when they do • Researchers find it easier to do highquality, ethical research • Less resource is invested in getting studies started • Clinical trials are registered and research gets published • The NHS appreciates the benefits of health research Protecting and promoting the interests of patients and the public in health research
  18. 18. GTAC – Improvements • Increased availability of review slots from 6 to 44 per year • Increased geographical spread – now four locations • Improved timelines – 2012–2013: • Pre-September 2012 – 8 studies: range 82–144 days • Post-September 2012 – 7 studies: range 19–76 days • KPI – HRA Business Plan – all studies < 60 days 19 Protecting and promoting the interests of patients and the public in health research
  19. 19. 20 Business Development and Project Financing Catapult is a Technology Strategy Board programme
  20. 20. Funding for Cell Therapy in the UK Govt. Support Medical Charities Commercial Development Plan Financial Investment Corporate Venturing 21
  21. 21. Working models Nature of project 22 Paid for by: Carried out by: Ownership of outputs Catapult owned Catapult Catapult Catapult Contracted Development Client Catapult Client Industry Grant or Collaboration Catapult and partner Catapult Outputs and shared partner
  22. 22. Collaboration 1+1 = 3 Client knowledge + Catapult capabilities = Better outcome Up to 80% Technology Strategy Board funding available for collaboration with SME Partner Project Input Funding Ratio TSB Grant SME £1m 60% £0.6m Catapult £1m 100% £1.0m Total £2m £1.6m 80%
  23. 23. 24 Building a Project Portfolio Catapult is a Technology Strategy Board programme
  24. 24. UK preclinical and clinical stage cell therapies Category Number Comparision More allogeneic therapies in preclinical stage Preclinical (<2 yrs from clinic) 37 Larger variety of cell types in preclinical stages Larger range of indications for preclinical projects Clinical (UK trial ongoing) 34 Total 71 Few commercially sponsored projects in both pre clinical and clinical stages 25
  25. 25. Larger variety of cell types in preclinical stage projects 26
  26. 26. Larger variety of cell types in preclinical projects 27
  27. 27. Opportunities for the Catapult • Significant potential to grow the clinical translation of therapies originating in the UK and to attract inward investment for clinical development • Significant potential to grow the number of UK based cell therapy companies • More and larger Phase 2 studies are required, statistically designed and powered to demonstrate efficacy Cell Therapy Catapult Confidential 28
  28. 28. Identifying projects • Pre-clinical and clinical databases • Technology transfer offices • Intermediaries • • • • Grant Funders Industry Groups Charities Investors • Direct contact • • Inward investors EU entrants
  29. 29. Gateway Criteria
  30. 30. Projects 31 Proof of Principle • Scientific, clinical, regulatory, commercial Suitability Non-clinical Suitability • Commercial Development plan • Safety, toxicology, GMP proving, assays Clinical • Safety and efficacy, investible data Platform • Generic issues and large collaborations
  31. 31. Project activity Over the Next 5 Years, complete • 150-200 short term suitability and assistance projects • 11-15 proof of principle projects • 5-9 non-clinical projects • 4-6 clinical projects Participate in the creation of 2-4 significant investible propositions 32
  32. 32. Building the portfolio hESC Blood Bone & Cartilage Cardiovascular Dermatology/Woun d Healing Diabetes Gastroenterology Immunology Liver Metabolic Neurological Oncology Ophthalmology Respiratory Other iPS MSC Immune Cell Other Somatic
  33. 33. Collaborations Loughborough University • Cooperation on manufacturing platforms UK Stem Cell Foundation • Project sourcing and funding 34
  34. 34. Project Examples GSK • Share of expertise • Support for in house projects Intercytex • New delivery device to reduce injection pain Reneuron • Phase 2 clinical trials • Scale up, Assays, Freezing and distribution of cells Inward investor • Manufacturing partner, Regulatory, Clinical trial design and delivery Kings • Immunomodulation • Regulatory, Clinical trial design, business models 35
  35. 35. 36 What does success look like? Catapult is a Technology Strategy Board programme
  36. 36. Strategic Goals Pipeline • Increased cell therapies in UK clinical trial and clinical use Value • Investible propositions created leading to cell therapy companies that succeed and stay in the UK Attractiveness • Demonstrating that the UK is the place to do this work, with increased inward investment Goals • Build a £10bn industry 37
  37. 37. Industry Researchers Cell Therapy Catapult NHS Catapult is a Technology Strategy Board programme Investment
  38. 38. Contact us Cell Therapy Catapult Limited Biomedical Research Centre, R&D 16th Floor Tower Wing, Guy’s Hospital Great Maze Pond, London, SE1 9RT Tel: +44 (0) 207 1883428 Catapult is a Technology Strategy Board programme 39