We enable UK life science companies to develop their drug discovery projects. And through networks of expert labs and CROs our Virtual R&D team can access and provide: > industrially rigorous advice in drug discovery > clinical and commercial insight > expertise in delivery and project management If you are an SME with a drug discovery project, or a CRO with expertise to provide, attend this event and find out how we can help you.

1. Accelerating innovative drug discovery
Connecting the UK drug discovery community
Transforming ideas into better medicines
for patients, faster
A NEW MODEL FOR
COLLABORATIVE R&D

2. National facility connecting the UK community
to accelerate innovative drug discovery
Medicines Discovery Catapult
WHO WHY HOW WHAT WHERE WORK WITH US
• Independent not-for-profit
• Part of the UK’s Catapult network
• Helping to deliver the UK’s Industrial Strategy
• Funded by Innovate UK, part of UK Research and Innovation,
reporting to the Department for Business, Energy & Industrial
Strategy

3. Pharma is facing unprecedented commercial pressure that force them to
reduce risk in their R&D pipelines1
In the UK:
SMEs account for more than 90% of our life sciences community2
But 42% of biotech SMEs have less than 5 employees3 and struggle to access:
WHO WHY HOW WHAT WHERE WORK WITH US
The industry is increasingly reliant on SMEs to innovate
Funding Expertise Technology
1. Deloitte (2017) A new future for R&D? Measuring the return from pharmaceutical innovation 2017.
2. HMG (2016) Strength and opportunity 2016: The landscape of the medical and biopharmaceutical sectors in the UK.
3. BIS (2015) Strength and opportunity 2015: The landscape of the medical technology and biopharmaceutical sectors in the UK.
SMEs are the agile risk-taking testers of new ideas, for innovation, discovery and
validation of new drugs and technologies

4. • Target specific industry-led challenges that limit today’s discovery process
• Industrialise, and drive the adoption of new scientific tools and modern techniques for discovering
medicines
• Deploy scarce expertise and know-how to connect a fragmented community and leverage current UK
infrastructure
• Support the UK life sciences industry, SMEs and innovators to deliver growth for the UK bio-economy
• Maintain the UK’s heritage position as a global leader in drug discovery
The enabling products and services that we diffuse into the sector will help UK SMEs fill their pipelines
with targeted medicines tested in patients, faster
WHO WHY HOW WHAT WHERE WORK WITH US
Our aims
Our vision

5. Providing hard-to-access
scientific capabilities
Gateway to UK resources
& expertise
By providing access to technologies and expertise not readily accessible we will support UK SMEs to
drive the development and widespread use of new approaches for the discovery of new medicines
WHO WHY HOW WHAT WHERE WORK WITH US
We are focussed on enabling UK drug discovery

6. We will not
• Compete with pharmaceutical or service companies
• Perform independent medicines discovery
• Replicate readily available UK infrastructure
We will prioritise and exemplify our efforts
• Outside of areas already covered
• In areas where UK science is perceived as ‘strong’
• In areas where SME needs are not met by others
• On development and promotion of cross-cutting discovery approaches
WHO WHY HOW WHAT WHERE WORK WITH US
We are focussed on enabling UK drug discovery

7. DISCOVERY SCIENCE
& TECHNOLOGY
INFORMATICS SAMPLES & DATA VIRTUAL R&D
DISCOVERY
SYNDICATES
Providing UK SMEs with access to technologies and expertise not readily accessible to them
WHO WHY HOW WHAT WHERE WORK WITH US
Our unique capabilities
Designed to address the needs of our UK drug discovery community and change the shape of R&D:
BETTER DATA FASTER
ACCESS TO RESOURCES & EXPERTISE
Underpinned by our skills & expertise in:
 Funding Legal & IP Project management & delivery Business development

8. It is vital that SMEs developing new drug candidates access:
• Robust, well-characterised, sophisticated human models for pre-clinical testing
• Complex bioanalytical technologies to appropriately interrogate and advance
only the promising candidates faster
The creation of human models and bioanalytical technologies requires substantial
capital investment and specialist expertise
Discovery Science & Technology
Establishing patient-relevant testing models and
new technologies to advance drug discovery
Our strategy is built around four interconnected focus areas, supported
by innovative technology
BIOMARKERS FOR
PRECISION MEDICINE
COMPLEX CELL
MODELS
TARGET ENGAGEMENT
& VALIDATION
DRUG DELIVERY &
PRE-CLINICAL IMAGING
TECHNOLOGY
INNOVATION
WHO WHY HOW WHAT WHERE WORK WITH US

9. Who do we help?
Unique
Biology
Models
Advanced
Technologies
Drug Discovery
Expertise
Collaborative
Relationships
Real world evaluation
and application to
cutting edge science
Characterisation of lead molecules
in translatable models, using
cutting-edge technologies
Real world testing of models with
drug molecules and a suite of
advanced technologies
Drug Discovery
Companies
Technology
Companies
Biology
Companies
WHO WHY HOW WHAT WHERE WORK WITH US

10. BBB Spheroid
Pancreatic
Organoids on
chip
Vascularised
Cardiac
microtissue
Hepatocyte
Spheroids
Colorectal
Spheroids
Neurite
Dynamics
Kidney models
Disease relevant
iPSC
Cardiomyocytes
OOAC - Tissuse
OOAC – Second
platform
Scaffold
Supports
Drug Delivery
systems into
Cells
Strategic
Agreements
UK OOAC
network
Fibrosis
Neuro-
inflammation
Organ on a Chip (OOAC)
Connected Microphysiology
2D
3D spheroid
monolayer
DAPI
GFAP
NG2
DAPI
GFAP
CD31
50 um
Confocal microscopy images of a
blood brain barrier spheroid model
Complex cell models
WHO WHY HOW WHAT WHERE WORK WITH US
Replicating human physiology and pathophysiology
in vitro to enable better translation
Human Relevant
Predictive.

11. Biomarkers for precision medicine
WHO WHY HOW WHAT WHERE WORK WITH US
Transcriptomics
• RNA
• DNA
• Protein
Droplet digital PCR
Mass spec imaging
Mass Spectroscopy
• Lipidomics
• Metabolomics
• (peptides/proteins)
In vivo imaging
Biomarker Discovery
Biomarker Assay
Development
Biomarker Validation
Biomarker
prevalence
Early disease
linkage
studies
Human tissue

12. Profiling of complex biology and biomarkers
WHO WHY HOW WHAT WHERE WORK WITH US
Multiplexing quantification of up to 800 DNA, RNA and Protein molecules within a sample
• DNA
• RNA – mRNA, miRNA, fusion transcripts
• Protein expression

13. What canitdo?
Uniqueimagingsolutionsenablesquantificationof:
• Organ accumulation(PK,biodistribution)
• Targetexpressionorengagement(stratification)
• Biologicalactivity(PD,optimalbiologicaldose)
• Toxicitiesordrug-druginteractions(safety,drugcombinationstudy)
• Surrogacyforclinicalendpoint(efficacy)
What canwedo?
Imaging Expertiseinmulti-parametricimagingmodalities:
• PositronEmissionTomography(PET)
• ComputedTomography(CT)
• HighFrequencyUltrasound(HFUS)
• Gamma countingex-vivoanalysis
• Nearinfraredopticalimaging
Targeted Drug Delivery
WHO WHY HOW WHAT WHERE WORK WITH US
Using Imaging technologies to visualise biological
functions to accelerate disease understanding and drug
development

14. Target engagement & validation
WHO WHY HOW WHAT WHERE WORK WITH US
Strategy
• Does my compound reach its desired site of action?
• Does my compound engage with its molecular target in cells?
• What are the downstream consequences of compound/target binding?
In translatable
human cell models
At single-cell
resolution

15. Target engagement & validation
WHO WHY HOW WHAT WHERE WORK WITH US
Compound-Target Interaction with
Purified Protein
Direct Measurement of
Compound-Target
Interaction in Cells
Target Conformational
Change in Cells
Proximal pathway events
following target engagement
Distal pathway events following
target engagement
Super-Resolution Microscopy
Fluorescence Polarisation & FCS
BRET/FRET
Protein Stability
Dimerisation
Localisation
Cellular Mass-Spectrometry +/- Target
Knockdown
Cellular Mass-Spectrometry +/- Target Knockdown
Transcriptomics +/- Target Knockdown
Microscopy
build from
4Q’18
Target
Engagement
Target
Validation
Establishing
assay methods to
interrogate
TE/TV at all
levels
Solid-state NMR
Mass-Spectrometry

16. Mass spec imaging capabilities
WHO WHY HOW WHAT WHERE WORK WITH US
DESI-MSimaging;CD1micetumourxenografts
• Smallmoleculeexpressiondifferences
• Noveltargeteddeliverymethod
Instrumentation
Q-TOF
TQ-XS
DESI
Image visualisation
software
Application /
exploitation

17. WHO WHY HOW WHAT WHERE WORK WITH US
Drug discovery generates a huge quantity of complex biological, chemical,
clinical and safety information
To make the best decisions on which assets to further optimise or progress, and to
create the best data assets, secure sharing and associated data analytics are critical
Informatics
Using data science to discover new diagnostics,
biomarkers and drugs
Using our informatics skills, extensive proprietary databases and algorithms
we can help validate and drive adoption of new data-driven approaches
NOVEL DRUG COMBINATIONS
In silico TARGET VALIDATION
USING REAL WORLD DATA
DRUG REPURPOSING
EFFICACY AND SAFETY
ANALYSIS
UNDERSTANDING
CHEMICAL SPACE
DRUGGABILITY
We have deep expertise in scientific data analysis, information extraction,
natural language processing and machine learning

18. Core themes
WHO WHY HOW WHAT WHERE WORK WITH US
Collaborative Intelligence
• Mapping the UKs life science capability
Interoperable Data
• Inter-organization ELN data transfer
• Connected Data
• ‘Data on demand’ – better coverage of known facts
• Drug repositioning & reformulation – maximise asset use
• Synergistic drug combinations – chimeras, blends, cocktails
• Drug resistance – AMR, cancer, antivirals
• Leads for new target classes – not PPIs – channels, transporters, viruses…
Target Prioritization
• Variant Effect Prediction – ‘multiscale’ approach
• Target Validation - druggability & efficacy
e.g., small molecule protein kinase inhibitor
benchmark set. N=746
IT
Academic
Large
Pharma
Therapeutic
SME
CRO
Single Drug Multiple Drugs
SingleTargetMultipleTargets
Combination
Therapy
Classic Drug
Discovery,
Ehrlich’s ‘Magic
Bullet’
Drug Blending
Designed/Serendipitous
Polypharmacology
Drug
Target
Biochemic
al assay
Cell-
based
screen
Function
al assay
Animal
disease
model
Human
clinical
trial
ChEMBL MDC Clinicaltrials.gov

19. IT
Academic
Large Pharma
Therapeutic
SME
CRO SME
Mapping capability
WHO WHY HOW WHAT WHERE WORK WITH US
Challenges for the community
• Finding the best collaborators
• ‘Entry-level’ e.g., one-off experiments, running a few
compounds through an assay…
• ‘Discoverability’ and ‘commercial inertia’ are big factors
UK inventory and map of drug discovery capability
• Companies House, Charities Commission, Patents,
Published Papers, Grant Applications, UK Universities,
Theses, Professional Societies, Consultant Networks…..
• Text-mining/Semantic Web, technical focus on
name/organization ‘grounding’

20. WHO WHY HOW WHAT WHERE WORK WITH US
The UK has millions of samples and billions of data points collected from
patients who have agreed their samples and data can be used for
research
However, small UK medical research companies struggle to access them
“Over 90% of SMEs said that access to high quality clinical samples was important,
however 80% found accessing samples unexpectedly difficult.”1
Samples & Data
Brokering easier access to consented patient
data and samples
We are working with key organisations supporting access to
consented tissues & data:
DIRECTORIES
RESEARCH
SERVICES
PROCUREMENT SERVICES

21. Access to samples is required by the sector
WHO WHY HOW WHAT WHERE WORK WITH US
Objectives
• Support UK SMEs in the supply of consented human tissue
• Set up collaborations with UK biobanks
• Provide access to expertise – Tissue Solutions
• A world leading provider of quality human tissue samples
• Benefit from connections and expertise
• Provide SMEs with required samples in a timely, cost
effective manner
• Provide vital cost recovery to biobanks to ensure that high
quality collections can be maintained
-100% 0% 100%
Access to biosamples for commercial
development is hugely important
It is easy to access high quality NHS biosamples
for commercial research
Access to the right health data such as registries
and activity is hugely important for innovative
companies
It is easy to access the right health data such as
registries and activity
The UK has a clear framework and process for the
commercialisation of NHS health data
Please could you indicate how much you agree / disagree with these
statements:
Neither agree nor disagree Somewhat agree Strongly agree
Somewhat disagree Strongly disagree

22. The ecosystem surrounding medicines discovery is highly
fragmented and complex
42% of biotech SMEs have less than 5 employees1 and are unable to efficiently
access the UK’s rich resources in translational research
Virtual R&D
Leveraging the UK’s renowned leadership
in drug discovery – a modern approach to R&D
Our VR&D platform uses a unique national network of up-to-date industry-
skilled consultants and service providers to deliver three essential elements
VR&D will deliver value to finance, biotech, academia and charities
1. BIS (2015) Strength and opportunity 2015: The landscape of the medical technology and
biopharmaceutical sectors in the UK.
WHO WHY HOW WHAT WHERE WORK WITH US
‘Pop-up’ Advisory teams
Virtual programme
management
UK capability map

23. Drug discovery is complex, costly and resource intensive
WHO WHY HOW WHAT WHERE WORK WITH US
Complex web of service providers, enablers and funders is challenging to navigate
Successful projects require:
• Enablers; CROs, specialist academic institutes, etc
• Knowledge; Decision making advice and expertise in drug discovery
• Delivery; Experienced drug discovery project management and due diligence
TI/TV
Hit
finding
Med.
Chem.
Pharmacology
Biomarkers
IND
enabling
Cell
models
Assay
generation
Comp.
Chem.
Library
design
Synth.
Chem.
Analyt.
Chem.
Process
Chem.
Formulation
In vivo
models
In vivo
efficacy
ADME
PK
PD
Dose to
man
Safety
GLP
tox
Idea
Drugcandidate

24. The UK: strength in basic science and a vibrant biopharma sector > 650 businesses
• BUT 11x less follow-on investment than US hubs
• 42% businesses <5 employees and the sector is fragmented and hard-to-navigate
9th Annual Report Office for Life Sciences, May 2018
Understand future market
Identify untapped opportunities
Best practice concept testing
Navigating the NHS to set up high
quality single centre trials
Advice on appropriate
models of efficacy and
safety
Biomarker and/or personalised
medicine strategy
Project
management
Advice on appropriate research
options – quality and price
Target ID &
validation
Lead
generation
Optimisation
Candidate
selection
First-in-
human trials
Exploratory
clinical trials
Proof of
concept
Launch
Secure
capital
WHO WHY HOW WHAT WHERE WORK WITH US
Why companies may want to seek expert support

25. An academy of excellence to guide SME drug discovery
WHO WHY HOW WHAT WHERE WORK WITH US
Benefits and desired outcomes
• Leveraging existing expertise for
the benefit of UK SMEs
irrespective of location
• Critical advice and due diligence
for drug discovery projects
• Identifying new opportunities for
interaction and collaboration
• Creating a connected eco-system
of expertiseCentres of excellence
existing networks and
consultants:
Connecting expertise
in drug discovery
and clinical science.
Timely, flexible support
for business critical decisions
+
UK strength in
drug discovery

26. Project Owner
Medicines Discovery
Catapult
VR&D
ProjectDelivery
“Status of the
Project”
Recommendation
(fee for service)
CDA
Project
Data
Package
MDC
Expertise to Progress your Drug Discovery Project?
WHO WHY HOW WHAT WHERE WORK WITH US

27. • Management of the virtual project team
• Alignment of a functional network of CROs, specialist
academic providers in addition to MDC capabilities to
deliver the project
• Provision of expertise to guide the project through
agreed milestones with associated Go/No Go
decisions for progression
WHO WHY HOW WHAT WHERE WORK WITH US
Medicines Discovery Catapult Virtual R&D Project Delivery

28. Clinical Development
Clinical
End to end services
Disease agnostic
Disease
specific
Niche
WHO WHY HOW WHAT WHERE WORK WITH US
Virtual R&D Project Delivery A Functional Network of CROs

29. • Existing relationships with CROs and knowledge of the
service/ expertise offered
• Medicines Discovery Catapult Informaticians identify
specialist & niche provision
• Development of expertise by Medicines Discovery
Catapult internal capability
• Management of relationships between CROs
• Facilitate administrative and contractual process
WHO WHY HOW WHAT WHERE WORK WITH US
Access to a Functional Network of CROs: The Advantages

30. Joining the Network of CROs
WHO WHY HOW WHAT WHERE WORK WITH US

31. What expertise or
capability is offered by
your CRO?
What expertise is needed
in our CRO Network?
Medicines Discovery
Catapult
Business Development and
Communications
Access to new and
larger markets
1. Qualitative due diligence
2. What is the Business Model?
• Revenue?
• Contractual Structure?
WHO WHY HOW WHAT WHERE WORK WITH US
Joining the Network of CROs

32. Who can we help?
WHO WHY HOW WHAT WHERE WORK WITH US
Real world evaluation and due diligence
Execution of project plans
New business for service providers
Opportunities for collaborative R&D
Start Ups,
Spin Outs
TTOs,
VCs
Drug Discovery
SMEs and CROs
Project management and drug discovery expertise
Access to drug discovery and disease area expertise
Relationships with CRO and specialist academic service providers

33. WHO WHY HOW WHAT WHERE WORK WITH US
As only patients know what it’s like to live with a disease, it’s crucial they
play a more central role in medical research
Talking to patients is often a better way to find out what medical products need to
developed, yet it doesn’t always happen
Discovery Syndicates
Spearheading patient involvement in early
drug discovery – a collective approach to R&D
Discovery Syndicates are a new, shared-resource model for disease focussed
consortia
Anchored by the leading medical research charity and centred
around patient needs
De-risk new promising assets
and drive commercialisation
Develop a patient centric
cohesive research agenda
Identify key barriers to
translation for a disease
Accelerate translation of
research to the clinic
Create new and more efficient
funding and investment models

34. WHO WHY HOW WHAT WHERE WORK WITH US
National facility and laboratory
Our office and laboratories are based at Alderley Park in Cheshire
Providing UK SMEs with access to the UK’s second largest life sciences cluster and unrivalled opportunities

35. md.catapult.org.uk
@meddisccat
info@md.catapult.org.uk
01625 238734
Visit our website for our latest partnerships and
funding opportunities.
Get in touch for more information, expert guidance,
strategic consultancy and access to our technology.
Or to discuss a potential project or service.
WHO WHY HOW WHAT WHERE WORK WITH US
Get in touch now