Keith Thompson CEO January 29 2013 info@ct.catapult.org.uk - - PowerPoint PPT Presentation

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

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The Catapults

• High Value Manufacturing
• Offshore renewable Energy
• Satellite Applications
• Digital Connected Economy
• Transport systems
• Future Cities
• And

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Cell Therapy Catapult

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• 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

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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

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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

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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.

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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

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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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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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Suitability

• 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

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

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Catapult is a Technology Strategy Board programme

Building The Catapult

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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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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

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Catapult is a Technology Strategy Board programme

Building a Project Portfolio

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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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Projects: Successful Path to Commercialisation

• start with the end product in mind and deliver an integrated plan

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• 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 Regulatory agency dialogue on plans at each stage

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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• Current UK and international activity
• Cell platform
• Technology barrier
• Disease
• Market size
• Market access
• Tractability and deliverability
• Early wins

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Drivers in Portfolio Construction

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

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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

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Field is growing beyond the traditional transplant and oncology indications

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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

hESC iPS MSC Immune Cell Other Somatic

Building the Portfolio

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Manufacturability

Building the Portfolio

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hESC iPS MSC Immune Cell Other Somatic

Manufacturability

Catapult is a Technology Strategy Board programme

What does success look like?

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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

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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)

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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

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Commercial Development Plan

Working models

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Catapult

• wned

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

• f 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

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Catapult is a Technology Strategy Board programme

Industry Cell Therapy Catapult Investment Researchers NHS