Business-led Translation A presentation by CEO, Keith Thompson, about growing a UK cell therapy industry. Oct 2013


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Business-led Translation
A presentation by CEO, Keith Thompson, about growing a UK cell therapy industry. Oct 2013

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Business-led Translation A presentation by CEO, Keith Thompson, about growing a UK cell therapy industry. Oct 2013

  1. 1. Business-led Translation Growing a UK cell therapy industry delivering health and wealth Keith Thompson CEO Terrapin Stem Cell, Cord Blood and Cell Culture Conference, Cambridge MA. October 2nd 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” Technology Strategy Board •  £440m for innovation in 2013/14 •  £250m+ assigned to TSB for 7 Catapults 2012 •  High Value Manufacturing, Cell Therapy, Offshore Renewables, CDE, Satellite Applications, Transport Systems, future Cities •  2 new ones announced August 2013 •  Stratified Diagnostics, Energy Storage •  Mandated to bridge the gap between research and commercialisation through industry collaboration 2
  3. 3. Cell Therapy -Mind the (translational funding) gap 3 •  Limited 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 mostly watching and waiting •  Operational SME’s lack finance and breadth of resources for rapid advance Catapult
  4. 4. Industry Barriers Business Manufacturing and Supply Chain Clinical And Regulatory •  Health Economics •  Business Models •  Reimbursement •  Partnering •  Robustness & Reliability •  COGS & Scale up •  Characterisation & Analytical •  GMP •  CMC •  Delivery •  Complex Regulatory Landscape •  Pre Clinical Packages •  Clinical trial design •  NHS partnering 4
  5. 5. Approach Project led •  Pathfind therapies into and through the Clinic and out and into the market Ø  Core projects with direct investment Ø  Rounded phase II data package Ø  Investible propositions •  Assist industry to progress to commercialisation Ø  Collaboration Ø  Contract research •  Technology platforms Ø  Develop novel technologies and license to industry •  Train professionals though immersion 5
  6. 6. Stage Gated 6 Proof of Principle •  Scientific, clinical, regulatory, commercial Non-clinical •  Safety, toxicology, GMP proving, assays Suitability •  Commercial Development Plan Clinical •  Safety and efficacy, investible data Platform •  Generic issues and large collaborations
  7. 7. Projects: Successful Path to Commercialisation 7 Regulatory agency dialogue on plans at each stage -start with the end product in mind and deliver an integrated plan Science •  •  Efficacy and safety hypothesis and evidence Definition and characterisation of cellular product Clinical •  •  •  •  Patient population, unmet medical need, differentiation Safety Robust evidence of efficacy Dose and dosing regimen •  •  •  GMP manufacturing process; release; comparability assays Supply logistics Scale-up / scale –out; Control of cost •  •  •  Pricing and reimbursement plan Defensibility (IP, know-how…) Commercialisation partner Manufacturing Business
  8. 8. Working models and finance 8 Nature of project Paid for by: Carried out by: Ownership of outputs Finance Core Project Catapult Catapult Catapult £70m/ 5 years Contracted Development Client Catapult Client £10m/ Per year Industry Collaboration Grant or Catapult Catapult and and partner partner Outputs shared £10m/ Per year
  9. 9. Funding for Cell Therapy in the UK Govt. Support Medical Charities Commercial Development Plan Financial Investment Corporate Venturing 9
  10. 10. Assets 10 -Facilities and Teams Facilities •  •  •  •  Central London location 1200 sq m on 12th floor Guys Tower Clinical research cluster Capacity for 100 people Teams •  Business •  •  Business development Business models •  Health economics •  Manufacturing and Supply •  Process development •  Analytical development •  •  GMP process proving Supply Chain •  Clinical Trial and Regulatory •  •  •  Regulatory Clinical trial sponsor Clinical operations
  11. 11. Manufacturing and Supply Chain Development Catapult is a Technology Strategy Board programme 11
  12. 12. Lab - Pilot - Scale 12 Catapult Multi Functional development Pod Commercial Scale Catapult GMP Proving Lab T e c h t r a n s f e r
  13. 13. Product release criteria Non clinical development Phase I 13 Phase II Market Approval Phase III •  Cell therapy characterisation requirements change depending upon where a product is in the commercialisation process •  Goal is to define and quantify the critical quality attributes for a particular product (release criteria) •  Need to develop appropriate assays for product release which can be incorporated into the manufacturing process (rapid, simple) Pilot GMP Process Development Process Commercial Process Scale Up/ Out
  14. 14. Cell therapy characterisation Non clinical development Phase I Phase II 14 Phase III Market Approval Products in pre-clinical development often require more thorough/complex characterisation using more advanced analytical approaches to understand the product MOA Cell markers Potential potency markers Identification markers Direct Surrogate Purity assay process qualification Pilot GMP Process Process Comparability Development Process Commercial Process Scale Up/ Out
  15. 15. Cell therapy characterisation Non clinical development Phase I Phase II 15 Phase III Market Approval MOA Cell markers Raw Materials Stability Potential potency markers Identification markers Direct Surrogate Purity assay Sterility testing Mycoplasma Virus testing Endotoxins Shelf life Genetic stability Viability the aim is to develop assays that are reliable, reproducible with low variance, Pilot GMP Commercial Scale Up/ Development inexpensive, simple, andProcess with appropriate reference standards — that rapid, Process Process Out also have relevance to the intended clinical activity.
  16. 16. Health Economics Catapult is a Technology Strategy Board programme
  17. 17. For healthcare products, price, reimbursement and demand are interlinked determinants of profit All these have to be effectively addressed to reach commercial goals… PRICE PROFIT REIMBURSEMENT 17 DEMAND
  18. 18. Successful commercialization depends on both regulatory approval and optimal market access Quality Safety Efficacy REGULATORY APPROVAL 18 Comparative Clinical & CostEffectiveness; Budget Impact MARKET ACCESS
  19. 19. Payers have a common aim: to achieve the greatest health care value for the money they spend Payers are key market access decision-makers (with input from clinical and economic advisors) Is it worth it? Does it add value over SOC? Cost-effectiveness Price comparison Can we afford it? Budget impact Should we control its use? Comparative clinical effectiveness Restrictions Do we need to fund it? Is the product needed? Unmet need Political imperative The emphasis on these elements differs across markets, most notably the use of cost-effectiveness in decision-making 19
  20. 20. Pricing approaches in healthcare are shifting towards value-based models Cost-based Competitor-based Value-based What is it? •  Price is set by assumptions on costs, expected sales volumes and margins •  Price is driven by the pricing of competitor products •  Price is based upon therapeutic / economic value to the customer Examples •  Cost-plus pricing •  ROI based pricing (e.g. PPRS in UK) •  Penetration pricing •  Reference group pricing •  Value-based pricing Comments 20 •  Enforced by many reimbursement •  Becoming obsolete; no longer resonates systems for “undifferentiated” with payers products •  Typical approach for differentiated products
  21. 21. Value-based pricing relies on the quantification of the added-value that a new technology delivers over SOC •  Reference Value of Standard of Care (SOC) o  Comparative data against the SOC is required: V = RV + PDV - NDV H2H comparative data demonstrating superiority or non-inferiority of Product X against the SOC is preferred Ø  Indirect comparisons of high methodological standards (NMA) sufficient for non-inferiority claims Ø  PDV Negative Differentiation Value (NDV) Positive Differentiation Value V RV 21 Reference Value (SOC) NDV •  Differentiating Value o  Clinical effectiveness o  Economic impact: budget impact, cost-minimization, cost-effectiveness, cost-utility •  Value (V) o  For a given indication “V” varies depending on the intervention’s positioning in the treatment algorithm & the target patient profile
  22. 22. In markets like UK, cost-effectiveness is the measure of value and the determinant of reimbursed price •  QALYs are the measure of clinical effectiveness Ø  QALYs = Life expectancy (life years) x Quality of life (utility) Ø  Ø  •  Utility ranges from 0 (death) to 1 (full health) Utility determined by HRQoL instruments; incorporation in clinical trials is key for market access Costs Ø  Direct (healthcare) and indirect (social care) costs o  Incremental Cost Effectiveness Ratio (ICER) Ø  QoL (utility score) •  Societal costs (e.g. time off work) not yet accounted by NICE 22 1.0 Cost B-Cost A / QALY B- QUALY A NICE ICER thresholds: •  Set at £20K/QALY; up to £30K justifiable if: Treatment A Ø  Treatment B 0.5 Ø  •  QALYs gained (B vs A) •  Orphans: No defined threshold Under VBP additional factors are likely to impact ICER threshold e.g. Ø  0 1 Life Years There is confidence in results Technology is highly innovative Ø  Ø  Disease burden Level of unmet need Size of target population
  23. 23. Main commercial preparations to optimize market access Enviromental Reviews Qualitative Research with Key Market Access Stakeholders Suitability assessment • Assess • Generate insights opportunity (e.g. on reaction to epidemiology, “Target Product disease burden, Profile”, key value unmet need, drivers, likely Objectives clinical /pricing positioning, benchmarks, pricing, funding & reimbursement, supporting data uptake, requirements, supporting data competition) requirements 23 Value Story Development Development Quantitative Pricing Research Value Dossier Development Out-licensing or Launch • Development of • Identify revenue clinical value maximising arguments and target price economic models • Inform asset • Test value story valuation and with key market market access access strategy stakeholders • Enhance market • Identify areas to strengthen story and data access potential and strategic partnering • Compile in a single document clinical and economic value proposition and corresponding evidence to support negotiations
  24. 24. Clinical Trials Catapult is a Technology Strategy Board programme 24
  25. 25. Databases of UK preclinical (<2 yrs from clinic) and clinical stage cell therapies Category   Preclinical (<2 yrs from clinic)   Number   Comparision   More allogeneic therapies in preclinical stage 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
  26. 26. Regulation……… •  Interact with regulators to improve clarity and speed •  Clinical access to NHS •  Clinical Trial Sponsor •  Use the existing flexibility 26
  27. 27. Clinical trials for cell therapies 27 Safety •  •  •  Risk mitigation Monitoring Duration follow-up Efficacy •  Patient population •  •  •  Risk vs benefit Disease stage Paediatrics Logistics •  Very close integration of clinical and manufacturing teams Dose selection •  •  Recognised endpoints •  Head to head comparison •  HRQoL Placebo and blinding •  In study vs •  historical /parallel Single vs multiple Dose escalation Feasibility •  •  Accompanying device / surgical technique Manufacturing success rate
  28. 28. Project Examples Catapult is a Technology Strategy Board programme 28
  29. 29. Identifying projects •  Pre-clinical and clinical databases •  Technology transfer offices •  Intermediaries •  Grant Funders •  Industry Groups •  Charities •  Investors •  Direct contact •  •  Inward investors EU entrants
  30. 30. Collaborations 30 •  Cooperation on manufacturing platforms •  Project sourcing and funding •  International collaboration
  31. 31. Project Examples •  Share of expertise •  Support for in house projects •  New delivery device to reduce injection pain •  Phase 2 clinical trials •  Scale up, Assays, Freezing and distribution of cells Large Cap Company •  Manufacturing partner, Regulatory, Clinical trial design and delivery •  Immunomodulation •  Regulatory, Clinical trial design, business models 31
  32. 32. Platform Project Example -Build a GMP iPS bank with Roslin Cells Biopsy Reprogramming Existing registry of volunteers for platelet and BM donation Known subpopulation of triple HLA-A, B and DR homozygotes Cell therapy GMP iPS cell bank Differentiated cells/ tissues of chosen types and origins Phase 1: Generate 6 cell lines: £2m •  •  Generate highly specified Master and Working Cell banks Generate Research grade equivalents and distribute to bona fide researcher Phase 2: Expand to 20 cell lines: £3m •  Milestone and success driven Phase 3: Expand to 100 cell lines: •  Self financing dependent upon early clinical trial results
  33. 33. INVESTABILITY Market  sen-ment  for  cell  therapy  investments  improving   •  Looking  for  rounded  data  package   Clinical  Data,  Health  Economics,  Business  Models,  Market  Access,  Reimbursement,  Defensibility   •  Pricing  target  and  COGS  drive  long  term  margin  towards  valuaHon   •  Clinical  Data  incorporaHng  H2H  and  HRQoL  improves  valuaHon   Health  economics,  pricing  and  value  story  built  to  support  commercialisaHon   Transac-ons   •  Financial  investment  –  Venture  Capital     •  Corporate    Venturing       •  Direct  Corporate  TransacHons  
  34. 34. 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 34
  35. 35. Researchers Industry Cell Therapy Catapult NHS Catapult is a Technology Strategy Board programme Investment