Investor Presentation4965zs3ha2l125fk78zkozo3.wpengine.netdna-cdn.com/... · Woodford Investment...

37
Investor Presentation Preliminary results for the year ended 31 March 2017 Olav Hellebø, Chief Executive Officer Michael Hunt, Chief Financial Officer Dr Julian Howell, Chief Medical Officer

Transcript of Investor Presentation4965zs3ha2l125fk78zkozo3.wpengine.netdna-cdn.com/... · Woodford Investment...

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Investor Presentation

Preliminary results

for the year ended 31 March 2017

Olav Hellebø, Chief Executive Officer

Michael Hunt, Chief Financial Officer

Dr Julian Howell, Chief Medical Officer

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Disclaimer

THIS PRESENTATION IS CONFIDENTIAL AND IS BEING SUPPLIED TO YOU SOLELY FOR YOUR INFORMATION AND MAY NOT BE REPRODUCED,

FURTHER DISTRIBUTED TO ANY OTHER PERSON OR PUBLISHED, IN WHOLE OR IN PART, FOR ANY PURPOSE.

Neither this presentation, nor the information contained in it constitutes or forms part of an admission document or a prospectus and does not form any part of (and

should not be construed as constituting or forming any part of) an offer of, or invitation to apply for, securities nor shall this document or any part of it, or the fact of its

distribution, form the basis of or be relied on in connection with any investment decision, contract or commitment whatsoever. This presentation should not be

considered a recommendation by ReNeuron Group Plc (the “Company”) or any of its respective directors, members, officers, employees, agents or advisers in relation

to any purchase of the Company’s securities, including any purchase of or subscription for any ordinary shares in the capital of the Company. Accordingly, information

and opinions contained in this presentation are being supplied to you solely for your information only.

Although reasonable care has been taken to ensure that the facts stated in this presentation are accurate and that the opinions expressed are fair and reasonable, the

contents of this presentation have not been verified by the Company or any other person. Accordingly, no representation or warranty, express or implied, is made as to

the fairness, accuracy, completeness or correctness of the information and opinions contained in this presentation, and no reliance should be placed on such

information or opinions. Further, the information in this presentation is not complete and may be changed. Neither the Company nor any of its respective members,

directors, officers or employees nor any other person accepts any liability whatsoever for any loss howsoever arising from any use of such information or opinions or

otherwise arising in connection with this presentation.

In the UK this presentation is being provided only to investment professional and high net worth companies, as described in articles 19 and 49(2), respectively, of the

Financial Services and Markets Act 2000 (Financial Promotions) Order 2005 and persons otherwise exempt under such Order. Securities in the Company have not

been, and will not be, registered under the United States Securities Act of 1933, as amended (the “Securities Act”), or qualified for sale under the law of any state or

other jurisdiction of the United States of America and may not be offered or sold in the United States except pursuant to an exemption from, or in a transaction not

subject to, the registration requirements of the Securities Act. The Company does not presently intend to register any securities under the Securities Act, and no public

offering of securities in the United States will be made. In the United States, this presentation is directed only at, and may be communicated only to, persons that are

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This presentation may contain forward-looking statements that reflect the Company's current expectations regarding future events, its liquidity and results of operations

and its future working capital requirements and capital raising activities. Forward-looking statements involve risks and uncertainties. Actual events could differ

materially from those projected herein and depend on a number of factors, including the success of the Company's development strategies, the successful and timely

completion of clinical studies, the ability of the Company to obtain additional financing for its operations and the market conditions affecting the availability and terms of

such financing.

By participating in and/or accepting delivery of this presentation you agree to be bound by the foregoing restrictions and the other terms of this disclaimer.

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Company overview

3

Global leader in allogeneic cell-based therapeutics

Breadth of pipeline

Strong management team

Unique platform technologies

Well backed and well funded

Focus on high value indications

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Global leader in cell-based therapeutics

4

Regenerative Medicine

1. Leaders in stem cell field

2. Ahead of the competition in

our selected indications

3. Secure Intellectual Property

4. Manufacturing capability

Innovation

1. Targeting unmet needs

2. High demand indications

3. Viable production strategies

4. Emerging exosome-based

nanomedicine

5. Broad range of indications

High growth sector

Potential to broaden therapeutic pipeline

beyond cell-based programmes

Extraordinary patient impact

Life-changing improvements in vision and

quality of life

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Unique platform technologies

5

CTX clonal cell line

hRPCs

CTX platform

Clinical and pre-clinical pipeline in vascular

and neurological indications

Exosome platform

Potential to broaden therapeutic pipeline

beyond cell-based programmes

Retinal platform

Targeting retinal degenerative diseases

In-licensed technology (Harvard, Boston)

One single stem cell

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Breadth of the pipeline

6

Product/

Indication

Discovery Pre-clinical Phase I Phase II Phase III Market

approval

CTX Cells

CTX cell therapy

Stroke Disability

CTX cell therapy

Critical Limb Ischaemia

Exosomes (CTX-derived)

Cancer

Human Retinal

Progenitor Cells

hPRC

Retinitis Pigmentosa

hPRC

Cone-Rod Dystrophy

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Well backed and well funded

7

Backed by major generalist and specialist life science institutional investors:

35.5% Woodford Investment

Management

9.5% Wales Life

Science Fund

9.3% Invesco

5.7% Aviva

£53 million ($66 million)

Cash on balance sheet (as at 31 March 2017)

Cash runway into late 2018

Funds therapeutic programs into mid or late-stage clinical development

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Strong management team

8

Olav Hellebø Chief Executive Officer

(Schering-Plough,

Novartis, UCB, Clavis)

Board member

Michael Hunt

ACA Chief Financial Officer

(Biocompatibles,

Bunzl)

Board member

Dr. Randolph

Corteling Head of Research

Dr. John Sinden Chief Scientific Officer

and co-Founder

Sharon Grimster VP Development and

General Manager,

Wales

(F-Star, Antisoma,

Celltech)

Shaun Stapleton Head of Regulatory

Affairs

(Elly Lilly, Boehringer

Ingelheim, Ipsen,

RRG)

Dr. Julian Howell Chief Medical Officer

(Shield, ProStrakan,

Roche, Pharmion)

Senior Management

Non-executive Board

John Berriman Chairman

(Autolus, Algeta,

Heptares, Abingworth)

Simon Cartmell

OBE (Apatech, Celltech,

Glaxo)

Dr. Tim Corn (Jazz Pharma, EUSA

Pharma, Circassia,

Glaxo)

Prof. Sir Chris

Evans OBE (Arthurian, Excalibur)

Dr. Paul Harper

(Physiomics, Sareum,

CAT, Glaxo)

Dr. Michael Owen Chair - ReNeuron

Scientific Advisory Board

(Zealand Pharma,

Avacta, Kymab, GSK,

ICRF)

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Focused strategy helping patients without any treatment options

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1

2

3

Gain clinical validation for our therapeutic programmes via robust clinical trials in well regulated territories.

Develop best-in-class cell based therapies for life changing high value products

Realise value for our technologies and therapeutic programmes via direct sales or substantial license deals

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10

Indication Assumptions to 2026 Peak

Sales

($bn)

CTX for stroke 1.76 million strokes/year (total US/EU/Japan)

85% survival, 85 % ischaemic

Peak penetration 5% US/EU/Japan

Treatment cost $40,000 EU to $60,000 US/Japan

1.1 - 3.9

hRPC for RP Prevalence 1:4000, ~244,000 cases (total US/EU/Japan)

Peak penetration 7.5% US/ EU

Per-eye treatment cost $50,000 EU to $75,000 US/Japan

0.5 - 1.8

Market potential according to Analysts’ estimates*

• Applicability of hRPCs in other hard-to-treat ophthalmic diseases could

provide upside potential

• Longer-term upside from exosome platform

*Stifel July 2016, N+1 Singer April 2017, Edison May 2017

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11 CTX cell product

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CTX cell product

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CTX, an allogeneic, cryopreserved human neural stem cell product

• GMP production, full set of release testing with a 6 month shelf life

• Product to be readily ordered, shipped and stored at the hospital

• Commercial scale of manufacturing allows reasonable cost of goods

• No generation of immune response after administration

CTX delivered in

cryo-shipper

Controlled thawing by

validated methods

Administer to

patient ‘on demand’

Similar to a conventional ‘off-the-shelf’ pharmaceutical /biologic drug

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Autologous vs Allogeneic cells

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• Autologous Cells

• Tissues harvested from a specific patient for his/her treatment

• Cell potency dependent upon age and health of each patient

• Usually limited shelf life with fresh cells

• Cost of cell preparation per preparation

• Unknown reimbursement scenario

Allogeneic cells offer commercial advantages over autologous preparation

• Allogeneic Cells

• One tissue harvest for many patients with scaled production

• Characterisation of product provides consistency across lots

• CTX established six month shelf life

• Costs spread over larger production base

• Realistic reimbursement projections

• No evidence of CTX generating an immune response in any patients treated

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CTX for Stroke disability: unmet medical need

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• Stroke is the single largest cause of adult disability

• Annual health/social costs: >$70 billion in the US

• Only one pharmaceutical treatment option available within 4 hours of stroke onset

• No treatment options available for stroke patients months to years later

• CTX administration promotes repair in the damaged brain

Lifetime risk of stroke:

1 in 5 women

1 in 6 men

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Two distinct cell therapy strategies for stroke

Str

oke D

am

ag

e

Bra

in R

eco

ve

ry

Tre

atm

en

ts

Thrombolysis;

Thrombectomy

Brain

Protection Brain Repair

Neuroplasticity: • Neurogenesis

• Angiogenesis

• Scar remodelling

Stem Cells

Minutes Days Hours Months Years

Inflammation

Neuron death

Neuroplasticity

Neuroprotective

Intravascular route

Regenerative

Intracerebral route

Acute Stroke Chronic Stroke

ReNeuron are pioneers in treating chronic stroke patients

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Implanted CTX cells

modulate the immune

system to promote repair by

Formation of new blood

vessels (angiogenesis)

Formation of new

neurons (neurogenesis)

Formation of new

connections between

neurons (synaptogenesis)

CTX promotes anatomical plasticity in the brain

Images are for illustration only 16

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Strong pre-clinical proof of concept

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• Rat stroke model used that affected similar regions of the brain as seen in patients

• Panel of behavioural tests to characterise dysfunction and recovery

• Injection of CTX into same region of brain as in the patients

• Improvements in “permanent” disabilities

• Restoration of function weeks after CTX administration

• Demonstrated dose response – unique in field of cell therapy

Well validated pre-clinical models predict efficacy in chronic stroke

Improvement in permanent dysfunction

CTX administration led to recovery of tape

removal from the affected forelimb in a

dose dependent manner

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CTX for Stroke disability: Phase I data published

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• Phase I dose escalation safety study published with 24 months follow up

• 11 disabled, stable stroke patients, 6 months to 5 years post stroke

• Single, straightforward neurosurgical procedure, Doses at 2, 5, 10, 20 million cells

• No cell-related or immunological adverse events

• Significant improvement in NIH Stroke Scale, 3 patients improved in modified Rankin Score,

Encouraging results across multiple efficacy measures of disability

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CTX for stroke disability – Phase II study design

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• Aim of the PISCES II study:

• To demonstrate effect of CTX cells on improving outcome of patients during rehabilitation phase following an ischaemic stroke

• To provide further safety data in a larger group of patients

• Inclusion Criteria

• Male and female patients; aged 40-89; 2-12 months after a stroke

• Upper limb dysfunction (Inability to pick up a 1” cube and place on a shelf)

• Study Procedures

• CTX 20 million cells injected into brain (putamen) on affected side, Follow up for 12 months

• Outcome measures

• Modified Rankin Score, Barthel Index, ARAT, Fugl-Meyer

• Treated 23 patients in 8 centres across the UK

• Median Age: 62 yrs (41-79), Median time from stroke to treatment: 7 months (2-13)

Robust trial of the capabilities of CTX in stroke patients

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Modified Rankin Score

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Improvement by one category is a significant change in a patient’s life

Algorithm from Bruno et al, 2010

Category

6 Death

5 Bedridden, completely dependent on others

4 Needing help to walk, use toilet, bathe

3 Can walk, but need still need help at home

2 Mostly recovered, but still has limitations

1 Slower than before, but no limitations

0 Back to pre-stroke life

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Bedridden, requires constant help from others

Needing help to walk, use toilet, bathe

Can walk with appliance,

Needs some help at home

Slight to no disability

from Lekander et al 2017, 42,114 patients from 2007-2012

Costs from Sweden, translated into pounds

Costs of disability – mRS scale

21

Improvements in disability result in substantial reductions in patient care costs

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PISCES II interim efficacy – (mRS)

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n= 21 21 10 3

33 percent of subjects (7 of 21) had a significant improvement in their quality of life

Most within 1 month of cell administration

Life changing results seen with CTX administration

Baseline 3 months 6 months 12 months

1 - no signficant disability

2 - slight disability

3 - moderate disability

4 - moderately severe

5 - severe

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PISCES II efficacy – Barthel Index

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• Measure of Activities of Daily Living

• mobility, self care, dressing

• 38% of patients had significant improvement (8 of 21)

• 100 point range of measure

• >9 point change is clinically significant

• 6 of 21 patients had BI >90 at baseline (therefore unable to reach ‘responder’ status)

• 53% of evaluable subjects had significant improvement (8 of 15)

0

2

4

6

8

1 month 3 months 6 months

Barthel Index Responders

Improvements in everyday activities after CTX administration

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PISCES II – Conclusions

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• Rate of patient improvement in patients with established disability due to stroke has greatly exceeded what we expected

• 15 of 21 patients responded to one or more of the four efficacy measures

• Response rate on mRS was greater than expected – use as primary measure in future studies

• Patients ready to enter study from 6 months post-stroke

• CTX intracerebral injection was well tolerated

• Adverse Events were attributed to surgical procedure

or stroke complications

• 1 death due to sepsis, 7 months after CTX treatment.

Assessed as not attributable to treatment.

• 33% response rate in mRS outcome measure

• 53% response rate in evaluable patients for BI measure

Potential of CTX in stroke warrants moving into a Phase III study

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Phase III Study - PISCES III

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• Randomised, controlled study with placebo surgery

• Entry criteria: mRS of 3 or 4 Primary endpoint will be a change in modified Rankin Score category

• Examine patients at 6 months post cell administration

• US and European sites use “Hub and Spoke” – reduced number of surgical sites

• Improves blinding by eliminating interaction of surgical staff and assessors

• 220 patients, 1 to 1 randomisation, CTX 20 million cell dose as used in PISCES II

• Discussions held with FDA / EMA, received favourable responses

• Commencing in early 2018 – Data expected early 2020

• A similar response rate in mRS to that seen in PISCES II would provide basis for a clinically and commercially viable cell therapy

• Presumed 20% increase over placebo response of 15%

• Alteplase had an 11% increase in favourable outcome to placebo response (n=333)

Discussions held with FDA on pivotal study design

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26 Human Retinal Progenitor Cells

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Retinal platform

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Broad application across a range of retinal diseases

• The eye does not regenerate lost cells

• Small changes in the retina will have a great impact in vision

• Programme based on human retinal progenitor cells (hRPCs)

• Preclinical testing programme demonstrated:

• Rescue of photoreceptors to preserve vision

• Maturation of injected hRPCs

• Approval of hRPC frozen formulation

• Ship and thaw on demand

• Collaborations:

Schepens Eye Research Institute (Harvard Medical School)

• Targeting Retinitis Pigmentosa, Cone Rod Dystrophy

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Retinitis pigmentosa

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• RP is an inherited, degenerative eye disease

• Onset from 20s to middle age

• Initial damage in outer retina

• Results in tunnel vision then blindness

• Incidence of RP is 1:4000 in the US with an estimated treatment population of 275,000 in the US and EU

• First therapeutic target for hRPCs

• Orphan Drug Designation in EU and the US & Fast Track Designation in US

• Phase I / II study ongoing in the US

• Phase I safety data readout in H2 2017

• Phase II commences H2 2017

• Phase II readout H2 2018

RP vision

There is no approved drug treatment for Retinitis Pigmentosa

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RP: IND-enabling pre-clinical efficacy data

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• RCS rat model of retinal degeneration

• Damage starts within weeks after birth

• hRPCs injected into the retina anticipating clinical administration

• Treatment significantly improved visual function

• Injected cells still present 12 weeks after dosing

• hRPC cells extend into the ganglion cell layer, demonstrating full integration

• Collaboration with UCL/Moorfields Eye Hospital

Dystrophic rats

hRPCs preserved visual acuity in degenerating retina

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RP: Clinical development – Phase I / II

30

• Dose escalation study in adult patients with established RP in the US

• Phase I - 3 dose groups of 3 subjects each

• Phase II - 20 additional patients at highest safe dose (to be expanded from 6 patients)

• Primary endpoint is safety, with visual acuity, visual field, retinal sensitivity and retinal structure as secondary efficacy measures

• Measurements in both treated and untreated eyes for comparison

• Phase I clinical site – Massachusetts Eye & Ear Infirmary, Boston (PI: Dr Eric Pierce)

Light guide

Infusion cannula

Subretinal cannula

delivering hRPC

Subretinal space

Choroid

Collaborating with elite institutions in ground breaking research

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Cone rod dystrophy

31

• CRD is an inherited, degenerative eye disease

• First noticed in childhood

• Initial damage to cones in central retina

• Loss of visual acuity and colour vision

• Progresses to blindness

• Incidence of CRD is 1:40,000 in the US

• Second therapeutic target for hRPCs

• Use safety data from RP Phase I study

• Conduct Phase II trial alongside RP study

• Commence in H1 2018

• Readout in H1 2019

CRD vision

image from MD Support

Working to prevent lifelong blindness

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32

Exosome Platform

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Exosome nanomedicine development

33

• Exosome therapeutic candidate selected (ExoPr0)

• Pre-clinical data

• ExoPr0 inhibits glioblastoma cell migration

• ExoPr0 reduces tumour volume in a CDX (cell-line derived xenograft) mouse model of glioblastoma

• Published data* identifies micro-RNAs contained within ExoPr0 responsible for regulating cell growth and apoptosis in cancer

• ExoPr0 crosses blood brain barrier allowing treatment of a number of conditions

• Development of platform technology for the delivery of therapeutic silencing RNA

• Target genes over-expressed in cancer indications

• £2.1m Innovate UK grant awarded to pursue ExoPr0 pre-clinical development

• Collaborators – Netherlands Cancer Institute, UCL, Cell & Gene Therapy Catapult

* Stevanato et al. PLOS ONE (2016)

ReNeuron are leaders in a new field of medicine

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34

Preliminary Results

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Preliminary results – highlights

35

(£’m)

Year ended March

31, 2017

(unaudited) Year ended March

31, 2016

Revenues and grant income 0.9 0.6

Research and development costs (16.7) (10.3)

General and administrative costs (4.1) (4.0)

Operating loss (19.9) (13.7)

Finance income 1.7 0.8

Tax credit 2.6 1.5

Loss for the year (15.6) (11.4)

Operating cash burn (12.6) (11.9)

Equity issues net of costs - 65.2

Net (decrease)/increase in cash (12.6) 53.3

Cash and deposits at start of year 65.7 12.4

Cash and deposits at year end 53.1 65.7

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Key clinical milestones – next 18 months

36

• H2 2017 CTX for Stroke disability – Phase II 12 month follow-up data

• H1 2018 CTX for Stroke disability – Phase III commencement

• H2 2017 hRPC for Retinitis pigmentosa – Phase I safety data

• H2 2017 hRPC for Retinitis pigmentosa – Phase II commencement

• H1 2018 hRPC for Cone-rod dystrophy – Phase II commencement

• H2 2018 hRPC for Retinitis pigmentosa – Phase II efficacy data

• H2 2018 Exosomes for cancer – Phase I commencement

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