A Focus on Spinifex Pharmaceuticals’

Research & Development of

Angiotensin II Type 2 (AT2) Receptor

Antagonists to Treat Chronic Pain

Tom McCarthy, Ph.D.

CEO and Managing Director

South Yarra Corporate Centre Level 1, 122 Toorak Road, South Yarra

Melbourne, Victoria AUSTRALIA

tom.mccarthy@spinifexpharma.com.au +61 411 102 143

AIRG National Winter Meeting 2013

“How is Technology and Innovation Performed in Australasia’s Newer Industries?”

Overview of Presentation

2

“How is Technology and Innovation Performed in Australasia’s Newer Industries?”

Spinifex Pharmaceuticals: A case study

– Who is Spinifex and what do we do?

– Why is Spinifex developing new treatments for chronic pain?

– What R&D process do we follow?

– History and timelines for Spinifex Pharmaceuticals

University spin-out company

Capital raising through syndicates of Australian based venture capital companies

Key federal government initiatives and support during the evolution of Spinifex

– How Spinifex conducts research & development to discover and commercialise a new approach to the

treatment of chronic pain

– How do we intend to make a return for all Spinifex shareholders?

Overview of Presentation

3

4

Private Australian

Spinifex Pharma

Venture Capital backed

Biotech

company

Developing new

treatments for chronic

pain

Who are Spinifex?

&

What do we do?

Patients need new treatment options

Not all patients respond to current therapy or are unable to tolerate the side effects

– Postherpetic neuralgia (PHN) as an example

– A chronic painful conditionthat lingers even after the shingles (herpes zoster rash) has healed

Why are we Developing New Treatments for Chronic Pain?

5

“I've had PHN (post-

herpetic neuralgia; a

chronic painful condition

that develops in some

people following

shingles) for 9 months. I'm

on 3600 mg of

Gabapentin & Lidoderm

patches daily.

I'm very interested in

trying your product...”

“My 75 year old father has

suffered with PHN

In the eye for the last 3.5

years!

He is in a lot of pain all the

time we are at wits end as all

the medications he has tried

and that he is on doesn't help

much also has doses of

OxyContin and endone

(opioids) everyday.

When will EMA401 for PHN be

available in pharmacies?...”

6

Spinifex Gameplan: “Rules” of the Biotech Sector

Source: innovation.org, a project of the Pharmaceutical Research and Manufacturers of America

7

Spinifex Gameplan: Biotech Sector cf. Other Industries

Source: innovation.org, a project of the Pharmaceutical Research and Manufacturers of America

Prospecting

“Test Drill”

Commercial

Feasibility

Commercial Scale

Production

Cf. Mining Sector:

8

0 Risk From Buyer or Acquirers Perspective

Commercial Return To Spinifex

Investors

Spinifex Gameplan: Risk Versus Reward

9

0 Risk From Buyer or Acquirers Perspective

Commercial Return To Spinifex

Investors

Spinifex Gameplan: Exit at the Right Time

10

Venture

Capitalists

“Big

Pharma”

Spinifex

“ The Next Spinifex”

“The Medical Research Commercialisation Fund (MRCF)

provides dedicated, investment funding to support the

commercialisation of early-stage medical research discoveries

that originate from its member institutes. The collaborative

nature of the MRCF seeks to foster best practice in the

commercialisation of medical innovations.”

Spinifex Gameplan: Incubate and Exit … & Reinvest

11

Maree Smith and Bruce

Wyse patent: AT2

receptor antagonists

are potential new

treatments for

neuropathic conditions,

including pain

Year: 2006 2003 2004 2005 2007 2008

How did Spinifex get Started?

12

Maree Smith and Bruce

Wyse patent: AT2

receptor antagonists

are potential new

treatments for

neuropathic conditions,

including pain

Year: 2006 2003 2004 2005 2007

UniQuest work to secure

first VC syndicate

Spinifex

Pharmaceuticals

Pty Ltd formed

2008

How did Spinifex get Started?

13

Maree Smith and Bruce

Wyse patent: AT2

receptor antagonists

are potential new

treatments for

neuropathic conditions,

including pain

Year: 2006 2003 2004 2005 2007

Tranche 1

Series A

$984K

UniQuest work to secure

first VC syndicate

Spinifex

Pharmaceuticals

Pty Ltd formed

2008

Pre-Seed Fund (PSF)

via

&

How did Spinifex get Started?

14

Maree Smith and Bruce

Wyse patent: AT2

receptor antagonists

are potential new

treatments for

neuropathic conditions,

including pain

Year: 2006 2003 2004 2005 2007

Tranche 1

Series A

$984K

UniQuest work to secure

first VC syndicate

Spinifex

Pharmaceuticals

Pty Ltd formed

Commercial Ready grant

$1.67M

Tranche 2 Series A $2.266M

2008

Pre-Seed Fund (PSF)

via

&

How did Spinifex get Started?

15

Maree Smith and Bruce

Wyse patent: AT2

receptor antagonists

are potential new

treatments for

neuropathic conditions,

including pain

Year: 2006 2003 2004 2005 2007

Tranche 1

Series A

$984K

UniQuest work to secure

first VC syndicate

Spinifex

Pharmaceuticals

Pty Ltd formed

Commercial ready grant

$1.67M

Tranche 2 Series A $2.266M

2008

Pre-Seed Fund (PSF)

via

&

Additional IP

from Maree

Smith/UniQuest

acquired

How did Spinifex get Started?

16

Maree Smith and Bruce

Wyse patent: AT2

receptor antagonists

are potential new

treatments for

neuropathic conditions,

including pain

Year: 2006 2003 2004 2005 2007

Tranche 1

Series A

$984K

UniQuest work to secure

first VC syndicate

Spinifex

Pharmaceuticals

Pty Ltd formed

Commercial ready grant

$1.67M

Tranche 2 Series A $2.266M

2008

Convertible note

$750K

Pre-Seed Fund (PSF)

via

&

Additional IP

from Maree

Smith/UniQuest

acquired

EMA401 enters

pre-clinical

development

How did Spinifex get Started?

17

Clinical

trials start

Year: 2012 2008 2010 2014

Pre-clinical

development of

EMA401

Spinifex R&D History and Timelines Summary

18

5 x Phase 1 studies

Clinical

trials start

Year: 2012 2008 2010 2014

Pre-clinical

development of

EMA401

Tranche 1 Series B $6M

Spinifex R&D History and Timelines Summary

19

Phase 2 proof

of concept

efficacy

clinical trial in

PHN patients

5 x Phase 1 studies

Clinical

trials start

Year: 2012 2008 2010 2014

Topline data

presented Pre-clinical

development of

EMA401

Clinical study

report signed off

& publication

submitted Tranche 1 Series B $6M Con

note $400K

Tranche 2 Series B

$10.5M

Tranche 3 Series B $2.05M + con note

Tranche 3 Series B $1.35M

Spinifex R&D History and Timelines Summary

20

Phase 2 proof

of concept

efficacy

clinical trial in

PHN patients

5 x Phase 1 studies

Clinical

trials start

Year: 2012 2008 2010 2014

Topline data

presented Pre-clinical

development of

EMA401

Clinical study

report signed off

& publication

submitted Tranche 1 Series B $6M Con

note $400K

Tranche 2 Series B

$10.5M

Tranche 3 Series B $2.05M + con note

Tranche 3 Series B $1.35M

IIF Follow on Fund

Spinifex R&D History and Timelines Summary

Go where the expertise is: Drug Manufacture & Bioanalytical

21

Microconstants, San Diego, USA Bioanalytical work

Pharmatek, San Diego, USA Drug capsule manufacture

Spinifex Headquarters, Melbourne, Australia

GlycoSyn, New Zealand

Drug substance manufacture

Corden Pharma, Switzerland Drug substance manufacture

How Does Spinifex Conduct R&D/Innovation

Go where the expertise is: Clinical

22

Spinifex Headquarters, Melbourne, Australia

Pharm-Olam, Poland Clinical Project Management

B&H Consulting, New Jersey, USA Regulatory Affairs

Bulgaria Clinical Trial Sites

Serbia Clinical Trial Sites

Georgia Clinical Trial Sites

Czech Republic Clinical Trial Sites

Ukraine Clinical Trial Sites

Sth Africa Clinical Trial Sites

London Clinical Trial Site

How Does Spinifex Conduct R&D/Innovation

Australia Clinical Trial Sites

23

0 Risk From Buyer or Acquirers Perspective

Commercial Return To Spinifex

Investors

Spinifex: “Progress Against Gameplan”

Screening & Baseline

Pain Assessment

Day

-14 -7 -1 1 15 8 22

Treatment Phase Follow-up

28

1o

Endpoint

24

42

Trial design for PoC in post herpetic neuralgia (PHN) patients

– Randomised (1:1), double-blind, placebo controlled

– Multicentre in 6 countries

– Single dose level of EMA401 [100mg b.i.d. for 28 days versus placebo]

– 183 patients randomised.

– Patients were either not receiving a current treatment for their PHN or not responding to single

agent front line therapy

– In the evening prior to sleep, patients were asked to record their average pain they

experienced during that day

0 = no pain; 10 = worst pain imaginable

– Changes in a variety of other pain and quality of life measures were assessed at the beginning

and end of treatment

EMA401-003: PoC Efficacy Study in PHN Patients

25

Diff. of LS Means (SE): -0.69 (0.25)

p = 0.0066

Diff. of LS Means (SE): -0.53 (0.22)

p = 0.0184

(1) Based on LOCF imputation method for missing data.

(2) P-value for treatment group comparison is based on analysis of covariance (ANCOVA). See MCG reports SCD13011 & SCD13018.

LS = Least Squares, SE = Standard Error

Standard Error derived from all pain diary data points per patient per week

EMA401 Relieves Pain and PHN Patients

26

0 Risk From Buyer or Acquirers Perspective

Commercial Return To Spinifex

Investors

Spinifex Gameplan: Are We in the Fourth Quarter?

Drug Discovery

Alan Naylor Scientific Advisory Board

(UK)

SYNthesis, HD Biosciences & CEREP

(France, USA, China)

Toxicology/ Safety Pharmacology

Fred Reno Toxicologist, (USA)

Colleen Johnson Toxicologist, (USA)

Martha Charney PK and TK analysis, (USA)

Preclinical/ Mechanism of Action

Prof. Maree Smith University of Qld (Australia)

Inventor

Prof. Anand’s Laboratory Imperial College London

(UK)

Profs. Cameron & Cotter University of Aberdeen (UK)

Clinical/ Medical Affairs

Nuket Desem VP Clinical & Regulatory

Affairs (Australia)

Geoff Kitson Chief Medical Officer

(UK)

McCloud Consulting Group Statistics (Australia)

Manufacturing (CMC)

Glycosyn (New Zealand)

Corden Pharma (Switzerland)

Pharmatek, (USA)

MicroConstants Human and non-clinical

bioanalytical. Drug-like properties. Analytical. (USA)

Prof. Chas Bountra Chair: Scientific Advisory

Board (UK)

Prof. Praveen Anand Scientific Advisory Board &

Clinical Investigator (UK)

Scientific & Clinical Leadership

Prof. Andrew Rice Scientific Advisory Board (UK)

Prof. Robert Dworkin (USA)

Pharmorphix (SAFC), (UK)

Regulatory Affairs

Helen Ribbans Bridgette Kunst/Sandy Lee

B&H Consulting, (USA)

27

Investors, Observers & Board

Josh Funder GBS Venture Partners

Ben Gust GBS Venture Partners

Chris Nave Brandon Capital Partners

Peter Devine Uniseed Management

Dean Moss UniQuest

Declan Doogan Spinifex Chairman

Eliot Forster Spinifex Non-Exec. Director

Intellectual Property

Kathryn Morris Davies Collison Cave

Jessica Wolff Wolff IP (USA)

Acknowledgements

A Focus on Spinifex Pharmaceuticals’

Research & Development of

Angiotensin II Type 2 (AT2) Receptor

Antagonists to Treat Chronic Pain

Tom McCarthy, Ph.D.

CEO and Managing Director

South Yarra Corporate Centre Level 1, 122 Toorak Road, South Yarra

Melbourne, Victoria AUSTRALIA

tom.mccarthy@spinifexpharma.com.au +61 411 102 143

AIRG National Winter Meeting 2013