Taking Stem Cell Therapies into the clinic – a case study Stem Cell Summit 2012 15 February, 2012

Michael Hunt

Chief Executive Officer

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Disclaimer

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Who we are

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Clinical stage stem cell therapy business Established 1997 - King’s College London (IOP) Based Guildford, Surrey Quoted on AIM (market cap approx £30m) Focused on development of novel stem cell therapies for poorly met medical needs

Presentation

What we do

Our cells and how we make and test them Our ReN001 stroke therapy - getting cells into the clinic Future clinical and commercial challenges

What we do

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Develop therapeutic products from human stem cell lines Proprietary cell expansion and cell selection technology Allogeneic approach – scalable, non-immunogenic cells Very extensive pre-clinical efficacy/safety data package with lead CTX neural cell line – developed over 7 years Multiple cGMP cell banks for future clinical and commercial Drug Product

Market stem cell products as research and drug discovery tools

ReNcell® range of neural cell lines, marketed worldwide through Millipore Corp (US) – academic and commercial research

Core stem cell assets

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CTX human neural stem cell line

In clinical development (ReN001 therapy for stroke) In pre-clinical development (ReN009 for CLI, and other indications)

hRPC human retinal stem cells

In pre-clinical development for retinal diseases (initially ReN003 for RP)

ReNcell® neural stem cell lines

For non-therapeutic research use (licensed to Merck Millipore)

Comprehensive and multi-layered IP position:-

Extensive patent portfolio covering composition, method of production, delivery and use of cell lines (CTX, CTXcryoTM, hRPCs, ReNcell® and other non-core cell lines)

Exclusive license to patents covering cell expansion technologies

Other IP license arrangements with commercial and academic organisations in UK and US to further broaden neural and retinal stem cell IP portfolio

Over 80 issued patents (50 in US/Europe) with over 75 further patent applications in key worldwide territories Trade secret protection for elements of cell formulation, manufacturing and scale-up

Intellectual Property portfolio

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The cell manufacturing challenge

Purity

Identity of cell line Potency - biological characteristics Stability of cell line in culture and in frozen state Formulation of cell product Product delivery/compatibility Process development (automation, etc) cGMP standards

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“We’ve got a few problems going from lab

scale up to full-scale commercial”

ReNeuron’s CTX cell product

Conditionally immortal human neural progenitor cell line - patented expansion technology, chemically controlled Unlimited supply from single tissue sample

Multiple cGMP cell banks laid down (CMOs in UK and US) – seed stock for future clinical and commercial product Cell characteristics maintained throughout cell expansion process Capable of scale-up in robotic cell culture systems Competitive advantage – biopharma manufacturing model

Frozen product – very long shelf-life; thawed product – 96 hour shelf-life Consistent efficacy in stroke and ischaemic limb models Potential in other neurological/vascular indications Clean acute and chronic toxicology, tumorigenicity and carcinogenicity

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CTX cell line selection

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CTX cell banking

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MCB = Master Cell Bank

P = Passage

WCB = Working Cell Bank

EOPCB = End of Production Cell Bank

CTX cell bank testing

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MCB/ WCB ( including EOPCB) testing includes: Sterility, Mycoplasma 16 different tests for human and animal viruses In vivo assay for tumorigenicity Transgene sequencing, integration site and gene copy number Karyology Viability and growth post thaw Phenotype

Drug substance Lots

Sterility, Mycoplasma, Endotoxin RCR, adventitious viruses Transgene sequencing, integration site and gene copy number Phenotype post growth factor removal Karyology Stability program supporting shelf life up to 3 years

CTX Drug Product testing

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Drug Product manufacture for individual treatments from drug substance lots In process testing for sterility, mycoplasma and endotoxin Full GMP process validation completed for sterility assurance of the process

Release specifications at point of formulation

Interim sterility Rapid mycoplasma Rapid Endotoxin Cell concentration and viability

Shelf life established for formulated product QP release on day of treatment

CTX product development - CTXcryo

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Stroke - third largest killer and single largest cause of adult disability in developed world

150,000 stroke patients annually in UK; 750,000 in the US. One third die Approx one half of all stroke survivors left permanently disabled 25% of long term hospital beds estimated to be occupied by stroke patients Annual health/social costs of caring for stroke survivors: >£5 billion in UK; >US$70 billion in US

No existing treatments beyond the acute phase, other than preventative Stroke disabilities potentially responsive to cellular intervention (and in earlier sub-acute phase of condition)

Sources: UK Stroke Association; American Stroke Association 14

ReN001 for disabled ischaemic stroke patients

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ReN001 – pre-clinical development (efficacy)

Source: Smith et al: Stem Cells. 2011 Dec 29. doi: 10.1002/stem.1024.

Bilateral Asymmetry Foot fault

Replicated pre-clinical studies showing that CTX cells reduce stable sensory/motor deficits in the rat in a dose-dependent way (three publications)

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ReN001 – pre-clinical development (MOA)

CTX cells react to their environment and are multi-modal in terms of mechanisms of action:

Pro-angiogenic – promotes new blood vessel growth Neurogenic – stimulates endogenous cellular response to damage Anti-inflammatory Immuno-modulatory

Continuing collaborative work on CTX cells with leading academic institutions Ten CTX-related publications so far

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ReN001 – pre-clinical development (safety)

Type of study Species and dose Route of administration and

study duration

Number of animals

injected with cells

Single dose GLP toxicology MCAo rat model of ischemic stroke.

450,000 cells

Intracerebral into the putamen – 3 and 12

months

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Single dose GLP toxicology

with/without tamoxifen

MCAo rat model of ischemic stroke.

900,000 cells

Intracerebral into the putamen in both

hemispheres – 7 days, 6 months and 9

months

94

Pathology from study GFi9 MCAo rat model of ischemic stroke.

450,000 cells

Intracerebral into the putamen – 1, 3 and 6

months

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Pathology from GFi10,

including measurement of

CTX0E03 cell proliferation

MCAo rat model of ischemic stroke.

4,500, 45,000 or 450,000

Intracerebral into the putamen - 3 months 27

GFi9 CTX0E03 and host cell

proliferation

MCAo rat model of ischemic stroke.

450,000 cells

Intracerebral into the putamen – 1, 3 and 6

months

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GFi10 host cell proliferation MCAo rat model of ischemic stroke.

4,500, 45,000 or 450,000

Intracerebral into the putamen – 3 months 27

GFi10 cell phenotype MCAo rat model of ischemic stroke.

4,500, 45,000 or 450,000

Intracerebral into the putamen – 3 months 27

Cell survival and

differentiation

MCAo rat model of ischemic stroke.

450,000 cells bilaterally

Intracerebral into the putamen in both

hemispheres – 2, 7, 14, 28, 56 and 183 days

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Gene silencing of cell

expansion system in vivo

MCAo rat model of ischemic stroke

450,000 cells

Intracerebral into the putamen – 7 and 28 days 12

The regulatory challenge

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National agencies involved in stem cell regulation:

British Standards Institute Department of Health European Medicines Agency Gene Therapy Advisory Committee Health & Safety Executive Home Office Animal Licensing Human Fertilisation & Embryology Authority Human Tissue Authority Medicines and Healthcare products Regulatory Agency Medical Research Council National Research Ethics Service UK Stem Cell Bank

ATMP Guidelines

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Centralised marketing authorisation procedure, to benefit from the pooling of expertise at European level and direct access to the EU market Multidisciplinary expert Committee (Committee for Advanced Therapies), within the European Medicines Agency (EMA), to assess advanced therapy products and follow scientific developments in the field Technical requirements adapted to the particular characteristics of these products

Traceability from product (unit) to patient and vice-versa Records kept for 30 years Risk-based programme for long-term follow up for safety and efficacy

Special incentives for small and medium-sized enterprises

Getting ReN001 into the clinic – what took so long?

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CTX cell product - R&D/GMP manufacture 2003-2010 First advisory meeting with MHRA – May 2008 CTA submission to MHRA – September 2008 Ethics submission to GTAC – December 2008 CTA approval from MHRA – Jan 2009 Full HTA therapeutic licence (post-inspection) – Oct 2009 Ethics approval from GTAC – February 2010 R&D Approval from Glasgow NHS August 2010 Injection device – CE marked Class III – October 2010 First patient recruited September 2010/ treated November 2010

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ReN001 – clinical development

World’s first fully regulated clinical trial using neural (brain) stem cells in a major neurological condition Places ReNeuron in forefront of stem cell therapy development for disabled ischaemic stroke patients

PISCES clinical trial – “Pilot Investigation of Stem Cells in Stroke”

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ReN001- Phase I trial

Phase I single administration dose escalation safety study, but with several efficacy evaluation measures (brain imaging, sensori-motor function) Open label study at Glasgow Southern General Hospital, Scotland

Prof Keith Muir – Institute of Neurosciences & Psychology, University of Glasgow

12 moderate to severely disabled ischaemic stroke patients – four dose cohorts with 24 month follow-up – stringent inclusion/exclusion criteria One-off, straightforward neurosurgical procedure (1 or 2 night hospital stay) No immunosuppressive drug treatments required

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ReN001- Phase I progress

Cohort Patient Age Interval Since

Stroke

NIHSS

Pre-

NIHSS 1m

Post

1

2 Million cells

1 67 14m 8, 6 5

2 82 21m 9, 9 7

3 78 51m 6, 6 5

2

5 Million cells

4 75 32m 6, 6 5

5 68 33m 10, 10 9

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ReN001- Phase I progress

First patient synopsis:

Male

In 60’s

Large left middle cerebral artery infarct (16 months ago)

Severe right hemiparesis – very little movement in arm and leg

Dependent on wife for many aspects of day-to-day living

Safely discharged two days after treatment – no complications or AEs at 14 months follow-up

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ReN001 – Phase I progress No Neurological Deterioration After Surgery and Implantation NIHSS Scores Over Time

0

2

4

6

8

10

12

14

16

-60 0 60 120 180 240 300 360 420

Study Day

NIH

SS

Sco

re 1

2

3

4

5

Surgery

Worse

Better

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ReN001 – Phase I progress No Deterioration in Spasticity After Surgery and Implantation: Summed Ashworth Scores Over Time

0

10

20

30

40

50

60

-60 0 60 120 180 240 300 360 420

Study Day

Su

mm

ed

As

hw

ort

h S

co

re

1

2

3

4

5

Surgery

Affected side Arm and Leg Scores Worse

Better

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ReN001- the clinical challenge

Indication: ischaemic sub-cortical stroke with paresis of an arm and limited recovery after 30 days

Clinically and statistically significant increase in the patient’s ability to use the affected arm for activities of daily living, including feeding, 6 months post-treatment and persisting at 12 months

Increases in the percentage of those able to use affected arm for feeding a minimum 20 percentage points over placebo (i.e. if % without ReN001 is 5%, percentage with ReN001 is 25%)

Statistically significant increase in the number of patients capable of assisted independent living

Efficacy sufficient to justify a cost of treatment of approx. £30,000 per patient in EU

Minimum target product profile:

The commercial challenge - partnering

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Stem cells an increasing focus of attention of large pharma looking to fill dwindling product pipelines:

Company Stage Indication

Takeda/Fate Therapeutics Early clinical Leukaemia/lymphoma

Cephalon/Mesoblast Mid clinical Cardiac, central nervous system

Astellas/Cytori Pre-clinical Liver disease

Genzyme/Osiris Mid/late clinical GvHD, Crohn’s, COPD,Type 1 diabetes, osteoarthritis

Pfizer/UCL Pre-clinical Dry AMD

Pfizer/Eyecyte Pre-clinical Diabetic retinopathy

Pfizer/Athersys Pre-clinical IBD

Teijin/SanBio Early clinical Stroke

Novo Nordisk/Cellartis Research Type 1 diabetes

Novartis/Opexa Pre-clinical Type I diabetes

AstraZeneca/UCL Research Diabetic retinopathy

Partnering rationale

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Stem cell therapies have the potential to match pharma’s expected future needs:

novel therapies addressing diseases with significant patient populations or population sub-groups

targeting diseases that cannot be addressed by existing drugs/biologics -

significant new market opportunities But only if:

cell product is readily scalable with favourable cost of goods (therefore reimbursable and likely to be adopted at an acceptable profit margin for the development partner)

there is a broad and robust data package and identifiable mode of action

the product is protected by a comprehensive IP position

Partnering challenges

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Recent partnering deals disguises wider corporate reluctance to invest at this stage Lack of marketed therapies Degree of disruption to established therapeutic business models Scientific/reputational/regulatory/market risk New scientific and business competencies required Internal structure/support from key decision makers

Implications for stem cell businesses looking to partner:

Clinical proof-of-concept data required – demonstrable clinical benefit and mechanisms of action exclusive to a regenerative medicine (or cell therapy) approach

Possible earlier deals in certain therapeutic areas where perceived risks are lower Early engagement with potential partners – identify potential strategic and business

case matches (parallel markets, orphan indications, etc)

Keeping the shareholders happy

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ReNeuron’s investors: Institutional funds

Hedge funds

Sovereign wealth funds

Venture capital fund

High net worth individuals

Retail investors

Board

Bryan Morton Non-Executive Chairman (Merck, BMS, Protherics, Dechra, EUSA)

Michael Hunt ACA Chief Executive Officer (Biocompatibles, Bunzl)

Dr. John Sinden Founder & Chief Scientific Officer

John Berriman Non-Executive Director (Algeta, Heptares, Abingworth, Celltech)

Simon Cartmell Non-Executive Director (ApaTech, Celltech, Glaxo)

Mark Docherty ACA Non-Executive Director (Excalibur)

Dr. Paul Harper Non-Executive Director (GSK, CAT, CMS)

Professor Trevor Jones CBE Non-Executive Director (Wellcome, ABPI, Allergan)

Senior Management

Dr. Kenny Pollock Head of Cell Development

Dr. Paul Stroemer Head of Pre-clinical Research

Caroline McManamon Head of Regulatory Affairs

Dr Mike McDonald Clinical Development Consultant

Professor Jack Price Principal Scientific Consultant (King’s College London)

Pat Huggins FCCA Head of Finance & Company Secretary

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Board and Senior Management

Professor Philip Bath MD - Stroke Association Professor of Stroke Medicine, Univ. of Nottingham

Dr. Louis Caplan MD - Chief, Cerebrovascular and Stroke Division, Beth Israel Deaconess Medical Center & Professor

of Neurology, Harvard Medical School, Boston

Dr. John Cooke MD - Stanford Univ. – Professor, Division of Cardiovascular Medciine, Stanford Univ. School of

Medicine; Associate Director, Stanford Cardiovascular Institute

Dr. Sid Gilman MD - Univ. of Michigan – Professor, Dept of Neurology; past FDA CNS Advisory Committee member

Dr. William Hiatt MD - Univ. of Colorado Denver – Professor for Cardiovascular Research, Department of Medicine;

past Chairman of FDA Cardiovascular and Renal Advisory Committee

Dr. Douglas Kondziolka MD - Univ. of Pittsburgh - Professor and Vice Chairman, Dept of Neurological Surgery

Professor Paolo Madeddu MD - Chair of Experimental Cardiovascular Medicine, Bristol Heart Institute, Univ. of Bristol

Dr. Paul Sanberg - Univ. of South Florida - Associate VP & Associate Dean, Biotechnology Development Director,

Center for Ageing and Brain Repair

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Clinical Advisory Board

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ReNeuron Group plc

10 Nugent Road

Surrey Research Park

Guildford

Surrey

GU2 7AF

UK

(t) +44 (0) 1483 302560

(f) +44 (0) 1483 534864

(e) info@reneuron.com

www.reneuron.com

Ticker: RENE.L

Thank you!