The document discusses the launch and goals of the Cell Therapy Catapult. The Catapult was launched to address barriers to commercializing cell therapies in the UK and grow a substantial cell therapy industry. Its goals are to increase the number of cell therapies in clinical trials, create investable companies and propositions, and demonstrate that the UK is a leader in cell therapy development. It will provide various services to support companies and aims to build a £10 billion cell therapy industry in the UK.

1. The Cell Therapy Catapult
Keith Thompson
CEO
January 29 2013
info@ct.catapult.org.uk
Catapult is a Technology Strategy Board programme

2. The Launch of Catapults
Hauser
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
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> £10bn pa long term industrial revenue to UK
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Exploit a strong existing UK science base
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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
2

3. The Catapults
• High Value Manufacturing
• Offshore renewable Energy
• Satellite Applications
• Digital Connected Economy
• Transport systems
• Future Cities
• And
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4. 4
Cell Therapy Catapult
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Significant unmet medical needs
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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
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Early enough for UK to establish a
strong market position

5. Mind the (translational funding) gap
5
•
•
Catapult
Little evidence yet that new cell therapies
can be developed, licensed and adopted
successfully
Limited investment from commercial
sector
•
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
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
6

7. Core Purpose
Growing a UK cell therapy industry
delivering health and wealth
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8. Vision
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.
8

9. Mission
The Cell Therapy Catapult will grow the industry in the UK to
substantial and sustainable levels by :
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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
9

10. 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
10

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
11

12. Projects
12
Proof of Principle
• 4 people, 12 Months, £0.5m, 2-6 ongoing
• Scientific, clinical, regulatory, commercial
Suitability
Non-clinical
Suitability
• 0.5
People
• 3
Months
• £20k
• 36 pa
• 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
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
Create an environment that increases clinical trials & clinical activity
by 50-100%
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14. 14
Building The Catapult
Catapult is a Technology Strategy Board programme

15. Assets
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. Catapults do not give
grants.
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16. Facilities and Teams
Facilities
•
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1200 sq m on 12th floor
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Capacity for 80-100 people
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Adjacent to complementary activities
Business Team
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Business development
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Health economics
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Business models
Translational Labs
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Process development
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Analytical development
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GMP process proving
Clinical Trial and Regulatory Team
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Regulatory
•
Clinical operations
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17. Building a Project Portfolio
Catapult is a Technology Strategy Board programme
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18. Global cell therapy industry: ‘Cell-able targets’
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…
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19. Projects: Successful Path to Commercialisation
19
Regulatory agency dialogue on
plans at each stage
-start with the end product in mind and deliver an integrated plan
Science
•
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Efficacy and safety hypothesis and evidence
Definition and characterisation of cellular product
Clinical
•
•
•
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Patient population, unmet medical need, differentiation
Safety
Robust evidence of efficacy
Dose and dosing regimen
•
•
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GMP manufacturing process; release; comparability assays
Supply logistics
Scale-up / scale –out; Control of cost
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•
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Pricing and reimbursement plan
Defensibility (IP, know-how…)
Commercialisation partner
Manufacturing
Business

20. Projects and Products
UK companies
SMEs
Big Healthcare
Inward investors
Trial initiation
Manufacturing and distribution
Academic pull through
Accelerate innovation
Create investible propositions
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21. Drivers in Portfolio Construction
• Current UK and international activity
• Cell platform
• Technology barrier
• Disease
• Market size
• Market access
• Tractability and deliverability
• Early wins
21

22. Diverse cell therapies in clinical trial in the
UK; but few are company sponsored
Category
Cell therapies currently in
clinical trial in the UK*
Number
~25
Comment
Roughly equal numbers of
autologous and allogeneic
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
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23. Bone marrow derived cells still predominate
but T-cells are a rising class amongst greater
diversity
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24. Field is growing beyond the traditional
transplant and oncology indications
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25. Opportunities for the Catapult
(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
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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
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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

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

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?
Catapult is a Technology Strategy Board programme
38

39. Success for the Cell Therapy Catapult means..
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
39

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

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

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

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

44. 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%

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