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 info@ct.catapult.org.uk

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

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

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

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

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

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

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

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

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

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

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

Gateway Criteria

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

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

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

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 info@ct.catapult.org.uk www.celltherapycatapult.org.uk

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