Catapult is a Technology Strategy Board programme

The Cell Therapy Catapult

Keith Thompson CEO January 29 2013 info@ct.catapult.org.uk

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

• > £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

2

The Catapults

• High Value Manufacturing

• Offshore renewable Energy

• Satellite Applications

• Digital Connected Economy

• Transport systems

• Future Cities • And

3

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

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

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

Core Purpose

Growing a UK cell therapy industry

delivering health and wealth

7

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

Mission

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

9

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

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

Suitabili

ty

• 0.5 People

• 3 Months

• £20k

• 36 pa

Projects

Proof of Principle

• 4 people, 12 Months, £0.5m, 2-6 ongoing

• Scientific, clinical, regulatory, commercial

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

12

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

Create an environment that increases clinical trials & clinical activity by 50-100%

13

Catapult is a Technology Strategy Board programme

Building The Catapult

14

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.

15

Facilities and Teams

• Facilities • 1200 sq m on 12th floor

• Capacity for 80-100 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

Catapult is a Technology Strategy Board programme

Building a Project Portfolio 17

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…

18

Projects: Successful Path to Commercialisation -start with the end product in mind and deliver an integrated plan

19

• Efficacy and safety hypothesis and evidence • Definition and characterisation of cellular product

• 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

• Pricing and reimbursement plan • Defensibility (IP, know-how…) • Commercialisation partner

Science

Clinical

Manufacturing

Business

Re

gu

lato

ry

ag

en

cy

dia

log

ue

on

p

lan

s a

t e

ac

h s

tag

e

Projects and Products

UK companies

SMEs

Big Healthcare

Inward investors

Trial initiation

Manufacturing and distribution

Academic pull through

Accelerate innovation

Create investible propositions

20

• Current UK and international activity

• Cell platform

• Technology barrier

• Disease

• Market size

• Market access

• Tractability and deliverability

• Early wins

21 Drivers in Portfolio Construction

Diverse cell therapies in clinical trial in the UK; but few are company sponsored

22

Category Number Comment

Cell therapies currently in

clinical trial in the UK* ~25

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

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

23

Field is growing beyond the traditional transplant and oncology indications

24

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

25

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

Communication Channels

• Pre-clinical and clinical databases

• Technology transfer offices

• Direct contact

• Intermediaries • Grant Funders

• Industry Groups

• Charities

• Investors

Gateway Questions

Project D: Investigate Further

Project E: Return to tracking

A

B

C

Blood

Bone and cartilage

Cardiovascular

Dermatology/wound healing

Diabetes

Gastroenterology

Immunology

Liver

Metabolic

Neurological

Other

Ophthalmology

Respiratory

Oncology

hESC iPS MSC Immune Cell

Other Somatic

Building the Portfolio 36

Manufacturability

Building the Portfolio

37

Blood

Bone and cartilage

Cardiovascular

Dermatology/wound healing

Diabetes

Gastroenterology

Immunology

Liver

Metabolic

Neurological

Other

Ophthalmology

Respiratory

Oncology

hESC iPS MSC Immune Cell

Other Somatic

Manufacturability

Catapult is a Technology Strategy Board programme

What does success look like? 38

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

Catapult is a Technology Strategy Board programme

Industry

Cell Therapy Catapult

Investment

Researchers

NHS

Need

• Patient

• Clinician

• Payer

• Seller

Population

• Identity

• Quantity

• Diagnostic

Product

• Substance

• Process

• Regulation

• Information

Tasks

• Innovation

• Quality

• Data

Start

Commercial Development Plan (start with the end in mind)

41

Commercial

Development

Plan

Investors in Cell Therapy in the UK (Examples of actual investment or declared interest)

Govt.

Support

Medical Charities

Financial Investment

Corporate Venturing

42

Commercial

Development

Plan

Working models 43

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

Intellectual Property Ownership

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

45