The Cell Therapy Catapult

Transcription

1 The Cell Therapy Catapult Keith Thompson CEO January Catapult is a Technology Strategy Board programme

2 The Launch of Catapults Hauser 2 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 Translational activity to bridge investment gap Learn from other countries with successful Technology Institutes Persistence of funding against a long term perspective and low priority on becoming self-financing

3 The Catapults 3 High Value Manufacturing Offshore renewable Energy Satellite Applications Digital Connected Economy Transport systems Future Cities And

4 Cell Therapy Catapult 4 Significant unmet medical needs World class science base supported by forward looking investments from TSB, MRC; Wellcome Trust, NHS, charities Development and production of cell therapies is complex NHS as large stakeholder for both research and exploitation Early enough for UK to establish a strong market position

5 Mind the (translational funding) gap 5 Little evidence yet that new cell therapies can be developed, licensed and adopted successfully Limited investment from commercial sector Catapult Limited precedents for valuable exits via IPO or acquisition Most large corporates are observing and waiting Operational SME s lack finance and breadth of resources for rapid advance

6 Addressing current industry barriers to commercialisation 6 Key barriers to commercialisation Pertinent issues for cell therapies Regulatory affairs Regulatory pathways still emerging and can be complex Pre-clinical science Investor avoidance of preclinical phase programmes Clinical development Hard to manage demonstration of clinical safety and efficacy Manufacturing and GMP readiness Move from bench/small scale GMP process to large scale manufacture is complex Supply chain management New model of delivery required Investment readiness Difficult for companies and technologies to attract investment

7 Core Purpose 7 Growing a UK cell therapy industry delivering health and wealth

8 Vision 8 The Cell Therapy Catapult vision is for the UK to be a global leader in the development, delivery and commercialisation of cell therapy. Where businesses can start, grow and confidently develop cell therapies, delivering them to patients rapidly, efficiently and effectively.

9 Mission 9 The Cell Therapy Catapult will grow the industry in the UK to substantial and sustainable levels by : Taking products into clinical trial, de-risking them for further investment Providing clinical expertise and access to NHS clinical partners Providing technical expertise and infrastructure to ensure products can be made to GMP and delivered cost effectively Providing regulatory expertise to ensure that products can get to the clinic safely in the shortest time Providing opportunities for collaboration, nationally and globally Providing access to business expertise, grants and investment finance so that commercially viable products are progressed and investable propositions generated

10 Strategic Goals 10 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

11 Cell Therapy Catapult Outputs 11 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 Suitability Projects 12 Proof of Principle 4 people, 12 Months, 0.5m, 2-6 ongoing Scientific, clinical, regulatory, commercial Suitability 0.5 People 3 Months 20k 36 pa Non-clinical 6 people, 24 Months, 2.2m, 2-6 ongoing Safety, toxicology, GMP proving, assays Clinical 5 People, 36 Months, 4.6m, 4-8 ongoing Safety and efficacy, investible data Platform 3 People, 12 Months, 0.5m, 1-3 ongoing Generic issues and large collaborations

13 Project activity 13 Over the Next 5 Years, complete short term suitability and assistance projects proof of principle projects 5-9 non-clinical projects 4-6 clinical projects Participate in the creation of 2-4 significant investible propositions Create an environment that increases clinical trials & clinical activity by %

14 14 Building The Catapult Catapult is a Technology Strategy Board programme

15 Assets 15 Finance 70m core grant from TSB to March m pa from other grant funders 10m pa from industry contracts We use our assets to accelerate innovation. Catapults do not give grants.

16 Facilities and Teams Facilities 1200 sq m on 12 th floor Capacity for people Adjacent to complementary activities Business Team Business development Health economics Business models Translational Labs Process development Analytical development GMP process proving Clinical Trial and Regulatory Team Regulatory Clinical operations 16

17 Building a Project Portfolio 17 Catapult is a Technology Strategy Board programme

18 Global cell therapy industry: Cell-able targets 18 Established medical practice: e.g. haematopoietic stem cell transplantation Commercial products: Rapidly growing global market Global turnover of $1bn in 2011; rising to $5bn in 2014; driven by diversity of small products (Apligraf, Dermagraft, Provenge made >$100m each in 2011) Therapies in development: Rapidly growing ~ 250 products in clinical development Global pharma involvement: Already Teva, Shire, Sanofi, Pfizer

19 Regulatory agency dialogue on plans at each stage Projects: Successful Path to Commercialisation -start with the end product in mind and deliver an integrated plan 19 Science Efficacy and safety hypothesis and evidence Definition and characterisation of cellular product Clinical Manufacturing 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 Business Pricing and reimbursement plan Defensibility (IP, know-how ) Commercialisation partner

20 Projects and Products 20 UK companies SMEs Big Healthcare Inward investors Trial initiation Manufacturing and distribution Academic pull through Accelerate innovation Create investible propositions

21 Drivers in Portfolio Construction 21 Current UK and international activity Cell platform Technology barrier Disease Market size Market access Tractability and deliverability Early wins

22 Diverse cell therapies in clinical trial in the UK; but few are company sponsored 22 Category Number Comment Roughly equal numbers of autologous and allogeneic Cell therapies currently in clinical trial in the UK* ~25 Range of indications from cardiovascular to oncology Bone marrow derived cells still predominate but rising diversity Cell therapies currently in clinical trial in the UK sponsored by a commercial company 4 Majority of studies are Research Council funded and EU consortia studies *verified studies currently on-going; excludes gene therapy & haematopoietic transplantation

23 Bone marrow derived cells still predominate but T-cells are a rising class amongst greater diversity 23

24 Field is growing beyond the traditional transplant and oncology indications 24

25 Opportunities for the Catapult 25 (i) significant potential to grow the clinical translation of therapies originating in the UK and to attract inward investment for clinical development (ii) significant potential to grow the number of UK based cell therapy companies (iii) in order to accelerate towards more approved therapies in this space, more larger Phase 2 studies are required, statistically designed and powered to demonstrate efficacy Cell Therapy Catapult Confidential

26 Identifying projects Catapult widely understood The door is open to all, at all times Screen database against our criteria Initial engagement : suitability project Selected projects progress Board decision against published criteria Portfolio and rationale shown on website

27 Communication Channels Pre-clinical and clinical databases Technology transfer offices Direct contact Intermediaries Grant Funders Industry Groups Charities Investors

28 Gateway Questions

29 Project D: Investigate Further

30 Project E: Return to tracking

31 A

32 B

33 C

34

35

36 Building the Portfolio 36 hesc ips MSC Immune Cell Other Somatic Blood Bone and cartilage Cardiovascular Dermatology/wound healing Diabetes Gastroenterology Immunology Liver Metabolic Neurological Oncology Ophthalmology Respiratory Manufacturability Other

37 Building the Portfolio hesc ips MSC Immune Cell Other Somatic Blood Bone and cartilage Cardiovascular Dermatology/wound healing Diabetes Gastroenterology Immunology Liver Metabolic Neurological Oncology Ophthalmology Respiratory Manufacturability Other 37

38 What does success look like? 38 Catapult is a Technology Strategy Board programme

39 Success for the Cell Therapy Catapult means.. 39 Leadership in building an emerging industry, addressing barriers to commercial investment Increased numbers of cell therapies in UK trials and clinical use Investible propositions creating successful UK companies Demonstrating that the UK is the place to do this work Being a leader in building a 10bn industry

40 Industry Researchers Cell Therapy Catapult NHS Investment Catapult is a Technology Strategy Board programme

41 Commercial Development Plan (start with the end in mind) 41 Start Tasks Product Population Need Commercial Development Plan Innovation Quality Data Substance Process Regulation Information Identity Quantity Diagnostic Patient Clinician Payer Seller

42 Investors in Cell Therapy in the UK (Examples of actual investment or declared interest) 42 Govt. Support Commercial Development Plan Medical Charities Financial Investment Corporate Venturing

43 Working models 43 Nature of project Paid for by: Carried out by: Catapult owned Contracted Development Industry Collaboration Catapult Catapult Catapult Client Grant or Catapult and partner Catapult Client Catapult and partner Ownership of outputs Outputs shared

44 Collaboration 1+1 = 3 Partner Project Input Funding Ratio Client knowledge + Catapult capabilities = Better outcome Up to 80% Technology Strategy Board funding available for collaboration with SME TSB Grant SME 1m 60% 0.6m Catapult 1m 100% 1.0m Total 2m 1.6m 80%

45 Intellectual Property Ownership 45 Basic Principles: Catapult model does not rely upon income from IP Background IP stays with owner Arising IP shared in commercial transaction In proportion to inputs Example: 2m collaboration Urgent new manufacturing process 50/50 Catapult and partner Partner: exclusive rights with their product and business Catapult: rights outside partners business

46 Industry Researchers Cell Therapy Catapult NHS Investment Catapult is a Technology Strategy Board programme