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Catapult is a Technology Strategy Board programme
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 [email protected]
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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
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Cell Therapy Catapult
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• 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
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Mind the (translational funding) gap 4
Catapult
• 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
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Addressing Barriers
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• Health Economics
• Business Models
• Reimbursement
• Partnering
Business
• Robustness & Reliability
• COGS & Scale up
• Characterisation & Analytical
• GMP
• CMC
• Delivery
Manufacturing
and
Supply Chain
• Complex Regulatory Landscape
• Pre Clinical Packages
• Clinical trial design
• NHS partnering
Clinical
And
Regulatory
Clinical Operations &
Regulatory Affairs
Process Development
Business Development
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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
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Catapult is a Technology Strategy Board programme
Building The Catapult
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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
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Assets -Facilities and Teams
• Facilities
• 1200 sq m on 12th floor
• Capacity for 80-100 people
• Clinical research cluster
• 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
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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
Dr Natalie Mount
Chief Clinical Officer
Professor Marc Turner
Non Executive Board Director
Dr Stephen Ward
Chief Operating Officer
Professor Michael Whitaker
Non Executive Board Director
Professor Johan Hyllner
Chief Scientific Officer
Dr Zahid Latif
Non Executive Board Director
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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
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Catapult is a Technology Strategy Board programme
The UK ATMP Manufacturing Landscape
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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
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Grade of
cleanroom
Grade B Grade C Grade B/C/D with
isolator
Number 24 10 17
• 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 0 2 4 6 8 10 12
suspension
adherent
2D
3D
HESC
IPS
cells from tissue
cell banking
viral vector
gene modification
Facility Process Experience
Number of Facilities
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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
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Regulatory Permissions
• Catapult building strong clinical and regulatory team
• Interact with regulators to improve clarity and speed
• Clinical access to NHS
• Clinical trial sponsor
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HRA vision and ambition
Protecting and promoting the interests of patients and the public in health 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 high-quality, 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
To make the UK a
great place to do
research, where
more money
invested in
research goes into
carrying out
relevant, good quality research
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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
Protecting and promoting the interests of patients and the public in health research 19
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Catapult is a Technology Strategy Board programme
Business Development and Project Financing
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Funding for Cell Therapy in the UK
Govt.
Support
Medical Charities
Financial Investment
Corporate Venturing
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Commercial
Development
Plan
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Working models 22
Catapult owned
Catapult Catapult Catapult
Industry Collaboration
Grant or Catapult and partner
Catapult and partner
Outputs shared
Contracted Development
Client Catapult Client
Nature of project Paid for by: Carried out by:
Ownership of outputs
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Collaboration
1+1 = 3
Client knowledge + Catapult capabilities = Better outcome
Partner Project
Input
Funding
Ratio
TSB Grant
SME £1m 60% £0.6m
Catapult £1m 100% £1.0m
Total £2m £1.6m
80%
Up to 80% Technology Strategy Board funding available for collaboration with SME
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Catapult is a Technology Strategy Board programme
Building a Project Portfolio
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UK preclinical and clinical stage cell therapies 25
Category Number Comparision
Preclinical
(<2 yrs from clinic) 37
More allogeneic therapies in
preclinical stage
Larger variety of cell types in
preclinical stages
Larger range of indications for
preclinical projects
Few commercially sponsored
projects in both pre clinical and
clinical stages
Clinical
(UK trial ongoing) 34
Total 71
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Larger variety of cell types in preclinical stage projects
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Larger variety of cell types in preclinical projects
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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
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Identifying projects
• Pre-clinical and clinical databases
• Technology transfer offices
• Intermediaries • Grant Funders
• Industry Groups
• Charities
• Investors
• Direct contact • Inward investors
• EU entrants
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Su
ita
bili
ty
• Commercial Development plan
Projects
Proof of Principle
• Scientific, clinical, regulatory, commercial
Non-clinical
• Safety, toxicology, GMP proving, assays
Clinical
• Safety and efficacy, investible data
Platform
• Generic issues and large collaborations
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Suitability
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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
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Blood
Bone & Cartilage
Cardiovascular
Dermatology/Wound Healing
Diabetes
Gastroenterology
Immunology
Liver
Metabolic
Neurological
Oncology
Ophthalmology
Respiratory
Other
hESC iPS MSC Immune
Cell Other
Somatic
Building the portfolio
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Collaborations
• Cooperation on manufacturing platforms
Loughborough University
• Project sourcing and funding
UK Stem Cell Foundation
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Project Examples
• Share of expertise
• Support for in house projects GSK
• New delivery device to reduce injection pain Intercytex
• Phase 2 clinical trials
• Scale up, Assays, Freezing and distribution of cells
Reneuron
• Manufacturing partner, Regulatory, Clinical trial design and delivery Inward investor
• Immunomodulation
• Regulatory, Clinical trial design, business models Kings
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Catapult is a Technology Strategy Board programme
What does success look like?
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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
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Catapult is a Technology Strategy Board programme
Industry
Cell Therapy Catapult
Investment
Researchers
NHS
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Catapult is a Technology Strategy Board programme
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 [email protected] www.celltherapycatapult.org.uk
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