Catapult is a Technology Strategy Board programme

Growing a UK Cell Therapy Industry Delivering

Health & Wealth

Matthew Durdy, Chief Business Officer December 2013 ct.catapult.org.uk

The Cell Therapy (and Regenerative Medicine) Opportunity

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*US Market data •  http://www.cancer.org/cancer/cancerbasics/economic-impact-of-cancer •  http://www.cdc.gov/diabetes/pubs/pdf/ndfs_2011.pdf •  http://www.cdc.gov/heartdisease/facts.htm •  http://www.pdf.org/en/parkinson_statistics •  http://www.provengehcp.com/FAQs.aspx •  http://www.dermagraft.com/about/efficacy/

Why a Catapult?

•  Learning

•  Excellence

•  Leadership

•  Efficiency

•  Risk taking

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Assets -Facilities and Teams

•  Facilities •  1200 sq m on 12th floor •  Capacity for 80-100 people •  Clinical research cluster •  Global location

•  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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Management Team and Board

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Keith Thompson Chief Executive (Director, Scottish National Blood transfusion Service, Serologicals)

Dr Natalie Mount Chief Clinical Officer (Director Pfizer, Neusentis. Cambridge, UCL)

Dr Stephen Ward Chief Operating Officer (Director, Stabilitech, Onyvax. Southampton, Imperial)

Matthew Durdy Chief Business Officer (Director, Aqix, Biotec, CDC. Oxford, Chicago)

Dr John Brown, CBE, FRSE Chairman BTG plc, Acambis, CAT

Professor Marc Turner Non-executive Director Edinburgh, SNBTS

Tim Edwards Non-executive Director Chairman, Bio Industry Association

Professor Michael Whitaker Non-executive Director NE Stem Cell Institute

Dr Zahid Latif Non-executive Director Technology Strategy Board

Nick Higgins Non-executive Director Consort Medical, Intercytec

Professor Johan Hyllner Chief Scientific Officer (Director, Cellectis, Cellartis, Vitrolife)

Headcount: 65 22/11/2013

Catapult is a Technology Strategy Board programme

Catapult Themes Risk

Management of Perceived Risk

Solution Nature Example

Risk?

EU Regulation

Not Understood

Expert Advice

Economics Not Clear Prepare Data

GMP Not done Expert partner

Therapy Not proven Share

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Catapult is a Technology Strategy Board programme

Catapult Themes Funding

Need

•  Patient •  Clinician •  Payer •  Seller

Group •  Identity • Quantity • Diagnostic

Product •  Substance •  Process •  Regulation •  Information

Tasks •  Innovation • Quality • Data

Commercial Development Plan

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Start Commercial

Development Plan

?

Govt. Support

Medical Charities

Financial Investment

Corporate Venturing

Funding for cell therapy in the UK

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Commercial Development Plan

Successful commercialization depends on both regulatory approval and optimal market access

Quality Safety Efficacy Comparative Clinical & Cost-Effectiveness; Budget Impact

REGULATORY APPROVAL MARKET ACCESS

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Lab - Pilot - Scale

Commercial Scale

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Catapult GMP Proving Lab

Catapult Multi Functional development Pod

Tech t ransfer

Collaboration

1+1 = 3

Client knowledge + Expert 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% UK Technology Strategy Board Funding available for collaboration with SME

Sharing the outputs and opportunities

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Partners

• Cooperation on manufacturing platforms

• Project sourcing and funding

•  International collaboration

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Project Examples

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Large Cap Company

•  New delivery device to reduce injection pain

•  Share of expertise •  Support for in house projects

•  Phase 2 clinical trials •  Scale up, Assays, Freezing and distribution

of cells

•  Manufacturing partner, Regulatory, Clinical trial design and delivery

•  Immunomodulation •  Regulatory, Clinical trial design, business

models

•  Commercial Development Plan

Activities

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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Working models and finance

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 and partner

Catapult and partner

Outputs shared

£10m/ Per year

Gateway Criteria

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

Outputs

Life changing therapies Investible propositions Industry changing innovation Skilled leaders UK leadership

Summary: The Cell Therapy Catapult

Strong first year of growth Laboratories and teams up and running, 60+ Large and small collaborations Global interaction Industry success is our success

Matthew Durdy info@ct.catapult.org.uk http://ct.catapult.org.uk Twitter: @CTCatapult

Catapult is a Technology Strategy Board programme

Industry

Cell Therapy Catapult

Investment

Researchers

NHS