for personal use only - asx · ecoquest (asx: ecq) profile ecoquest limited is an environmental and...

Investor update: Cynata November 2012

For

per

sona

l use

onl

y

Important information

This presentation has been prepared by EcoQuest Limited. (“EcoQuest” or the “Company”) based on information available to it as at the date of this presentation. The information in this presentation is provided in summary form and does not contain all information necessary to make an investment decision.

This presentation does not constitute an offer, invitation, solicitation or recommendation with respect to the purchase or sale of any security in EcoQuest, nor does it constitute financial product advice or take into account any individual’s investment objectives, taxation situation, financial situation or needs. An investor must not act on the basis of any matter contained in this presentation but must make its own assessment of EcoQuest and conduct its own investigations. Before making an investment decision, investors should consider the appropriateness of the information having regard to their own objectives, financial situation and needs, and seek legal, taxation and financial advice appropriate to their jurisdiction and circumstances. EcoQuest is not licensed to provide financial product advice in respect of its securities or any other financial products. Cooling off rights do not apply to the acquisition of EcoQuest securities.

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, no representation or warranty, express or implied, is made as to the fairness, accuracy, completeness or correctness of the information, opinions and conclusions contained in this presentation. To the maximum extent permitted by law, none of EcoQuest, its officers, directors, employees and agents, nor any other person, accepts any responsibility and liability for the content of this presentation including, without limitation, any liability arising from fault or negligence, for any loss arising from the use of or reliance on any of the information contained in this presentation or otherwise arising in connection with it.

The information presented in this presentation is subject to change without notice and EcoQuest does not have any responsibility or obligation to inform you of any matter arising or coming to their notice, after the date of this presentation, which may affect any matter referred to in this presentation.

The distribution of this presentation may be restricted by law and you should observe any such restrictions.

Forward looking statements

This presentation contains certain forward looking statements that are based on the Company’s management’s beliefs, assumptions and expectations and on information currently available to management. Such forward looking statements involve known and unknown risks, uncertainties, and other factors which may cause the actual results or performance of EcoQuest to be materially different from the results or performance expressed or implied by such forward looking statements. Such forward looking statements are based on numerous assumptions regarding the Company’s present and future business strategies and the political and economic environment in which EcoQuest will operate in the future, which are subject to change without notice. Past performance is not necessarily a guide to future performance and no representation or warranty is made as to the likelihood of achievement or reasonableness of any forward looking statements or other forecast. To the full extent permitted by law, EcoQuest and its directors, officers, employees, advisers, agents and intermediaries disclaim any obligation or undertaking to release any updates or revisions to information to reflect any change in any of the information contained in this presentation (including, but not limited to, any assumptions or expectations set out in the presentation). F

or p

erso

nal u

se o

nly

EcoQuest (ASX: ECQ)

Profile EcoQuest Limited is an environmental and life sciences technology business based in Perth, Western Australia.

Board Darren Olney-Fraser: Executive Chairman Howard Digby: Executive Director Peter Webse: Non Executive Director and Company Secretary

Capital Structure 505 million ordinary shares (ECQ) 290 million 1 cent options (ECQO)

Investments •  11% stake in US based stem cell company Cynata •  Biodegradable technology investments.

For

per

sona

l use

onl

y

EcoQuest Transformation

•  Restructured in March 2012 with the aim of expanding

biodegradable hygiene portfolio to other technology based assets

•  Business expansion focus: biotechnology

•  Existing operations around core biodegradable hygiene technology to focus on licensing IP to third parties

•  Extension of IP portfolio by acquiring attractive life sciences assets

•  11% interest in Cynata Inc. announced September 2012

For

per

sona

l use

onl

y

The healthcare boom: all signs point to growth

-40

-30

-20

-10

0

10

20

30

40

50

60

IBB IXIC XHJ XMM XAO

% change

Relative performance of major healthcare and resources indices

2Y 5Y

IBB: Nasdaq Biotech Index IXIC: Nasdaq Composite XHJ: ASX Healthcare 200 XMM: ASX300 Metals and Mining XAO: ASX All Ordinaries

Market factors influencing the attractiveness of life sciences

Venture-oriented mining and resources finance is slowing Life Sciences has outperformed globally

Forecasts of resources boom peak in 2014 (e.g. Deloitte's latest Investment Monitor report)

Aging global population; greater focus on healthcare economics driving patient care

Productivity and cost issues, commodity prices, $A Signs of new biotech boom forming

Change in Chinese economy Developing countries have more healthcare spend

For

per

sona

l use

onl

y

Why biotech is a compelling sector

Three year NASDAQ Biotech Index: up 63%

•  Addresses the issues resulting from rapidly aging populations globally

•  Massive chronic disease burden on healthcare systems

•  Big pharma is experiencing:

  Major drugs going off patent

  Narrowing internal pipelines

•  Cashed up generic drug makers are looking to own their own IP

•  Sector rotation - markets looking for opportunities outside of mining

•  Rapidly maturing sector that contends with largely outdated investor perceptions

•  Less dependant on global commodity prices For

per

sona

l use

onl

y

Major growth segment: regenerative medicine

•  Biopharma product development involves considerable risk:

  Biologicals represent lower risk than small molecule drugs

  Stem cell technology is beginning to mature

  Local and international examples of success rewarded by the market

  2012 Nobel Prizes recognise the potential of Regenerative Medicine

For

per

sona

l use

onl

y

Cynata: EcoQuest has an 11% equity position

•  Incorporated in the USA to commercialise unique and versatile platform stem cell technology coming out of the University of Wisconsin (UW) in Madison, USA.

•  Stem cell technologies hold immense potential for regenerative therapies. Certain stem cells can be induced to become any type of cell in the body.

•  Founders and key executives include;

  Professor Igor Slukvin, pioneer of stem cell technology at UW (along with James Thomson with whom he co-founded Cellular Dynamics)

  Dr Allen Bollands, former CEO of Genera Biosystems (GBI) and Repromed

  Ian Dixon, Executive Director, experienced biotechnology executive, with particular interest in stem cells, director of Cell Therapies Pty Ltd and Trobio Pty Ltd

  Dr Roger Aston, previous positions former CEO of Peptech Ltd (PTD), former Chairman and CEO of Mayne Pharma (MYX)

For

per

sona

l use

onl

y

•  Complications of Diabetes ($3 billion market)

  Tragically growing levels of diabetes leading to vascular complication

  Critical Limb Ischaemia (CLI)

  Diabetic ulceration

  Peripheral neuropathy

•  Other opportunities ($10 billion market)

  Cardiovascular disease: heart failure, heart attack, stroke

  Lung disease

  Orthopedic applications

Cynata: A compelling market

For

per

sona

l use

onl

y

•  Stem cell technology originates from the laboratory where stem cells were pioneered

•  Platform technology with exciting early applications in diabetes

•  Patent protection (USA) until 2028

•  Ethically safe: stem cells are non-embryonic •  Pilot animal studies in Critical Limb Ischaemia (CLI) under way

•  Low development risk

•  Access to world class FDA-approved laboratory in USA

Why Cynata? Investment highlights

For

per

sona

l use

onl

y

CONFIDENTIAL – NOT FOR ONWARD DISTRIBUTION

Stem Cells tor the treatment of Vascular Disease

Stem Cells for the treatment of Vascular Disease

Introduc)on to Cynata

Allen Bollands

Founder and Chief Execu5ve Officer For

per

sona

l use

onl

y



The Vision

Build value by developing highly effec5ve and compe55ve, off-‐the-‐shelf, stem cell-‐based medica5ons, using our patented cell plaBorm technology

2

For

per

sona

l use

onl

y



Key strategies

1.  Build an experienced, high-‐performing team

2.  DifferenMate the Cynata plaNorm from alternaMve technologies

3.  Exploit outstanding benefits of Cynata technology: 1.  MulMpotent, primiMve, pericyte-‐like cells 2.  Pure and reproducible cellular product 3.  Massive expansion capability 4.  Safe

4.  Pick low-‐hanging fruit

5.  Leverage investor funds using non-‐diluMve means

6.  PosiMon the company for public ownership or M&A 3

For

per

sona

l use

onl

y



World’s first manufactured stem cell product approved

•  Osiris TherapeuMcs Incorporated –  Prochymal: adult bone marrow-‐derived mesenchymal stem cells –  Indicated for paediatric Gra^ versus Host Disease (GvHD) –  Approved in Canada (05/12) and New Zealand (06/12)

•  GvHD –  Bone marrow transplant recipients –  Immune cells in gra^ aeack recipient as “foreign”

•  FDA status –  Granted orphan drug status in 2010 –  Canadian submission based upon data-‐mining – FDA don’t accept this –  Resubmission in 2013

4

For

per

sona

l use

onl

y

Business plan overview

5

For

per

sona

l use

onl

y



Twelve month milestones

6

•  Human resources: –  Chief ScienMfic Officer –  RA Consultant

•  Research and Development: –  Comparison of compeMng cells –  DiabeMc Ulcers and Gra^ versus Host Disease proof of concept –  Preclinical design meeMng with FDA

•  Manufacturing: –  30-‐50% through manufacturing development process

•  Intellectual property: –  Conclude agreement to access MCAs via Wisconsin –  In-‐licenced iPSC rights

For

per

sona

l use

onl

y



12-‐36 month milestones

•  Corporate: –  Adequate funding to take the company through Phase 2 human clinical

trials ($40m)

•  Research and Development: –  Finalised lead indicaMon –  Completed preclinical programme –  Submieed IND

•  Manufacturing: –  CGMP product available –  Tech transfer to volume manufacturer underway

•  Commercial: –  At least 1 large non-‐diluMve grant –  At least 1 major commercial partnership

7

For

per

sona

l use

onl

y



RelaMve risk of development failure

0%

5%

10%

15%

20%

25%

30%

Biologics NMEs

8 Bethan Hughes, Nature Biotechnology, April 2011

For

per

sona

l use

onl

y



Management team

•  Allen Bollands, Managing Director –  Highly experienced life science industry execuMve. Over 25 years in major pharmaceuMcal

companies (SmithKline Beecham and NovarMs) and biotech startups. –  Former Chief ExecuMve Officer of Repromed Pty and ASX-‐listed Genera Biosystems

•  Ian Dixon, ExecuMve Director –  Experienced biotechnology execuMve, with parMcular interest in stem cells –  Director of Cell Therapies Pty Ltd and Trobio Pty Ltd

•  Roger Aston, Chairman –  Highly experienced execuMve with thirty years in a range of biotechnology business around the

globe. –  MulMple successful startups –  Outstanding capital-‐raising connecMons

•  Igor Sluvkin, Founder and MCA inventor –  Professor, Dept. Pathology and Laboratory Medicine, University of Wisconsin –  Founder of Wisconsin-‐based Cellular Dynamics – world’s largest producer of fully-‐funcMonal

human cells derived from iPSCs. –  Author of almost 70 peer-‐reviewed papers.

9

For

per

sona

l use

onl

y



CriMcal success factors

•  IdenMficaMon and recruitment of appropriate experMse

•  Maintenance of adequate funding via a relentless programme of investor relaMons

•  Intensive investment on high quality science

•  Enhance awareness of MCAs as a differenMated, versaMle and high value technology plaNorm

•  Full and rapid exploitaMon of MCA technology, where possible using partners and non-‐diluMve funding sources

•  Low-‐cost “virtual” model

10

For

per

sona

l use

onl

y



Summary

•  Cynata will develop MCAs into an allogeneic stem cell therapy to treat condiMons requiring revascularisaMon.

•  MCAs are outstanding therapeuMc stem cell candidates. Risk of development failure is low.

•  Strategic focus on building an experienced team, differenMaMng the plaNorm, and targeMng less challenging indicaMons

•  Cynata will mature as stem cell interest is peaking

11

For

per

sona

l use

onl

y



Stem Cells for the treatment of Vascular Disease

Allen Bollands [email protected]

+61 423 943 600

www.cynata.com

12

For

per

sona

l use

onl

y

Igor Slukvin MD, PhD Founder and Chief Scien�fic Advisor

1

For

per

sona

l use

onl

y

Human Embryonic Stem Cells

  Embryonic Stem Cells are Pluripotent Stem Cells   Self-renewal and large-scale expansion   Differentiation into all types of cells found in human body

James Thomson (University of Wisconsin)

For

per

sona

l use

onl

y

Pluripotent Stem Cells

Red Blood Cells

Heart Cells

For

per

sona

l use

onl

y

Induced Pluripotent Stem Cells

  Self-renewal and large-scale expansion   Differentiation into all types of cells found in human body

hESCs

iPSCs

For

per

sona

l use

onl

y

Developmental Potential of Pluripotent Stem Cells

5

Pluripotent Stem Cells

Mesoderm Progenitors/stem cells

Ectoderm Progenitors/stem cells

Endoderm Progenitors/stem cells

Brain

Eye

Skin

Lung

Pancreas

Liver

Gut

Hematopoietic Stem Cells

Mesenchymal Stem Cells

Endothelial Progenitors

Cardiac Stem Cells

For

per

sona

l use

onl

y

How Stem Cells Work

  Regenerate diseased �ssues   Deliver a range of therapeu�cally relevant molecules to promote �ssue repair and healing, protect damaged cells, reduce inflamma�on, and restore vasculariza�on

6

For

per

sona

l use

onl

y

Adult Stem Cells

  Hematopoietic Stem Cells –  Standard of care for otherwise non-curable blood diseases

such as blood cancer and genetic blood diseases In the early stages of clinical development

  Mesenchymal Stem Cells –  Cardiovascular diseases –  Musculoskeletal diseases –  Chronic inflammatory disease, transplant rejection

  Endothelial progenitors –  Cardiovascular disease

7

For

per

sona

l use

onl

y

Cynata Technology

8

For

per

sona

l use

onl

y

Pluripotent Stem Cells as a Source of Cells for Cardiovascular Diseases

Pluripotent stem cell

Ectoderm Skin, Neurons

Mesoderm Muscles, connective

tissue, blood etc

Endoderm Gut, respiratory system

Blood Lateral plate mesoderm

Intermediate mesoderm

Urogenital and

reproductive systems

Paraxial mesoderm

Skeletal muscle, bone,

cartilage

Heart Vasculature

Circulatory system develops from lateral plate mesoderm

9

For

per

sona

l use

onl

y

Cynata Patent Estate

  MCAs provide building blocks for the forma�on of circulatory system

  Logical cellular therapeu�c candidate to treat diseases requiring �ssue revascularisa�on

Lateral plate mesoderm

Principal IP is MCA cell: First Vascular Precursor

Mesenchymoangioblast (MCA)

10

For

per

sona

l use

onl

y

MCAs as Stem Cell Therapeutics MCAs – Ideal characteristics for therapeutic development

  Source of critical building blocks for the circulatory system

  Clonal expansion means uniform, pharmaceutical-grade product

  Massive expansion potential: 1→ 1022 cells

  Long patent life and strong “composition of matter” claims

  Relatively low risk of development failure

11

For

per

sona

l use

onl

y

MCAs: Expansion and Differentiation Potentials

Vodyanik et al, 2010; Cell Stem Cell, 7, 717-729

Endothelial C

ells

MSCs

MCA

For

per

sona

l use

onl

y

Cynata’s Science Leadership   Igor Slukvin, MD, PhD, Diplomate of ABP, Associate Professor of

Pathology, University of Wisconsin-Madison

–  Expertise in hematopoietic differentiation and mesoderm development from human pluripotent stem cells

–  First to isolate MCAs (mesenchymoangioblasts) –  First to isolate lymphohematopoietic progenitors from human pluripotent

stem cells –  First to derive transgene-free iPSCs from bone marrow and cord blood

cells –  Collaborated with Dr. Thomson to create iPSCs –  Scientific co-founder of CDI (Wall Street Journal's 2011 Technology

Innovation Award Company) –  Inventor of more than 10 patents covering derivation of cells of

mesodermal lineages from human PSCs (blood, endothelial and mesenchymal progenitors)

13

For

per

sona

l use

onl

y

The use of MCAs in the Cardiovascular Diseases and Vascular

Complica�ons of Diabetes

14

For

per

sona

l use

onl

y

Cardiovascular Diseases   Diseases that involve heart and blood vessels   Biggest cause of death worldwide   Each year kill more Americans than cancer   Major complication of diabetes

–  Peripheral arterial disease (8 million patients in the US) –  Coronary artery disease (13 million patients in the US) –  Stroke (15 million patients in the US) –  Wound healing (10 million patients in the US)

15

For

per

sona

l use

onl

y

16

For

per

sona

l use

onl

y

Critical Limb Ischemia   Dangerously diminished tissue perfusion:

–  Occluded arteries reduces blood flow to extremities.

–  Compromised vasculature, tissue necrosis, ulceration, gangrene.

  Poor prognosis. Within 1 year of CLI diagnosis:

–  40-50% patients undergo major amputation –  30-40% patients will die

  3 million CLI cases in the USA - 60% are diabetics

–  Annual cost of treatment $10-20 billion –  Market opportunity $3 billion –  Insurance: $30-40K – amputations

$25-75K – prosthetics $75K annually - rehabilitation

17

For

per

sona

l use

onl

y

Prevention and Treatment Options

  Control of blood pressure, lipids (statins), weight and blood sugars (in diabetes patients)

  Restoration of blood flow (balloon angioplasty with stent placement, coronary artery bypass surgery)

–  Challenges   Lost tissues and affected vasculature following vascular

accident can’t be replaced

18

For

per

sona

l use

onl

y

Therapeutic Neovascularization and Tissue Regeneration

  Stimulation of new vessel formation using pro-angiogenic drugs/growth factors and gene therapy   Transplantation tissue specific progenitors

(cardiac and neural stem cells)   Introducing cells that provide support in

endothelial regeneration and participate in vessel formation

19

For

per

sona

l use

onl

y

Growth Factors and Gene Therapies for Critical Limb Inschemia

  Growth factors: G-CSF, SDF, HGF, EPO   Are yet to successfully progress beyond phase 2   Gene therapy using HGF plasmids is currently under study by Anges (Japan) and Viromed (South Korea)

  Refirmenogene (Sanofi-Aventis) –  FGF1-based gene therapy failed

20

For

per

sona

l use

onl

y

Cellular Therapies for Cardiovascular Diseases: Cell Sources

  Bone Marrow –  Mesenchymal Stem Cells (MSCs; multipotent) –  Multipotent adult progenitor cells –  Hematopoietic Stem Cells (HSCs, multipotent) –  Endothelial Progenitors Cells (EPCs)

  Adipose Tissue –  MSCs –  EPCs

  Umbilical Cord Blood –  EPCs –  MSCs –  HSCs

  Tissue specific progenitors

21

For

per

sona

l use

onl

y

Clinical Trials

  Demonstration of clinical efficacy of cell therapies in experimental studies led to rapid translation of this concept into the clinic

  Clinical phase I and II studies initiated for MI and PAD/CLI

  Indicate a favorable safety profile and improvement of function

22

For

per

sona

l use

onl

y

Major Limitations of Current Therapeutic Stem Cell Products

  Most of them are based on Mesenchymal Stem Cells (MSCs) and or hematopoie�c stem/progenitor cells which have no endothelial poten�al

  Stem cell popula�ons are poorly defined and o�en heterogenous

  Have to be obtained from the donors   Rela�vely limited expansion poten�al

  High variability between individuals and batches   Need extensive quality control for every batch of stem cell product

23

For

per

sona

l use

onl

y

MCA is Multipotential Progenitor with Vasculogenic Potential

Bone

Cartilage

Vasculature

Adipose PSC

Vodyanik et al, 2010: A Mesoderm-Derived Precursor for Mesenchymal Stem and Endothelial Cells. Cell Stem Cell, 7, 717-729

MCA Colony

APLNR+ Mesoderm

MSC

24

For

per

sona

l use

onl

y

Advantages of MCAs

  We will use MCAs –  Building blocks for new vessel formation –  Superior expansion potential –  Clonally derived –  Can’t be obtained from adult tissues –  Efficacy demonstrated in preclinical model

  Our technology can provide unlimited supply of well-defined drug-like quality cellular products for therapies

  Easy to develop continuous manufacturing and quality control procedures to meet FDA criteria

25

For

per

sona

l use

onl

y

Efficacy of MCA in Preclinical Model of CLI

Control (non-treated) animals with CLI

CLI animals treated with MCAs Non-ischemic leg Ischemic leg

26

For

per

sona

l use

onl

y

Efficacy of MCA in Preclinical Model of CLI

27

For

per

sona

l use

onl

y

Summary

  Cynata will develop MCAs into an allogeneic stem cell therapy to treat condi�ons requiring revascularisa�on   MCAs are outstanding therapeu�c stem cell candidates – Provide unlimited supply of well-defined drug-like

quality cellular products for therapies – Easy to develop continuous manufacturing and

quality control procedures to meet FDA criteria

28

For

per

sona

l use

onl

y

for personal use only - asx · ecoquest (asx: ecq) profile ecoquest limited is an environmental and...

Documents