Catapult is a Technology Strategy Board programme
The Cell Therapy Catapult
Keith Thompson CEO January 29 2013 [email protected]
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
Catapult is a Technology Strategy Board programme
Industry
Cell Therapy Catapult
Investment
Researchers
NHS
Top Related