Human therapeutic products Human therapeutic products for for - - PowerPoint PPT Presentation

ABN 75 082 811 630

Human therapeutic products Human therapeutic products for for chronic respiratory and autoimmune diseases chronic respiratory and autoimmune diseases

June 2004 June 2004

Summary Summary

20 40 60 1999 2000 2001 2002 2003 2004

Listed on ASX: Listed on ASX: Nov 03 Nov 03 Cash at end FY04: Cash at end FY04: $25m $25m Grants receivable: $7.6m Grants receivable: $7.6m Shares outstanding: Shares outstanding: 108m 108m

$ millions

Capitalisation Capitalisation

Product focused Product focused Attractive markets Attractive markets Effective management team Effective management team TGA approved facility TGA approved facility One Phase III product One Phase III product Two Phase II products Two Phase II products Two preclinical projects Two preclinical projects

Product Pipeline Product Pipeline

• -------Clinical Trials----------

3-5 years 15 months 15 months 18 months 18 months indicative time to complete 12 months research preclinical phase I phase II phase III registration market

Respiratory diseases

AridolTM – airway function Bronchitol

TM - cystic fibrosis

Bronchitol

TM - bronchiectasis

Bronchitol

TM - chronic bronchitis

Autoimmune diseases

PXS25 - multiple sclerosis PXS2030 – multiple sclerosis PXS2076 – rheumatoid arthritis Mid -2005 Early -2008 Late -2007 Late -2008

Respiratory Disease Respiratory Disease

Bronchitol BronchitolTM

TM in Phase II

in Phase II

• New therapeutic

New therapeutic Cystic fibrosis and COPD Cystic fibrosis and COPD Complete Phase II Q3 2004 Complete Phase II Q3 2004 Commence Phase III Q1 2005 Commence Phase III Q1 2005 Product Launch 2007 Product Launch 2007

• Global Market Opportunity

Global Market Opportunity

CF: $1.1 billion+ CF: $1.1 billion+ COPD: $8 billion+ COPD: $8 billion+

• Revenue Opportunity

Revenue Opportunity

CF: $340 million+ CF: $340 million+ COPD: $1.3 billion+ COPD: $1.3 billion+

Aridol AridolTM

TM in Phase III

in Phase III

• First management tool for asthma

First management tool for asthma and COPD and COPD

• Complete Phase III Q3 2004

Complete Phase III Q3 2004

• Product launch Q3 2005 Aus/Europe

Product launch Q3 2005 Aus/Europe

• Global Market Opportunity:

Global Market Opportunity:

$1.6 billion+ $1.6 billion+

• Revenue Opportunity:

Revenue Opportunity:

$210 million+ $210 million+

Aridol AridolTM

TM

Bronchitol BronchitolTM

TM

Asthma Asthma

Current Management Current Management

Diagnosis by observation Diagnosis by observation Lung testing by specialists Lung testing by specialists Expensive Expensive Inconvenient Inconvenient Not specific for asthma Not specific for asthma Inaccurate Inaccurate Patients self manage Patients self manage

Aridol AridolTM

TM Management

Management

Diagnosis by evidence Diagnosis by evidence Objective measure of severity Objective measure of severity Accurate Accurate Convenient Convenient Reveals lung inflammation Reveals lung inflammation 10 10-

• 20 minute test

20 minute test Physicians monitor disease Physicians monitor disease 52 million people affected worldwide 52 million people affected worldwide 180,000 deaths worldwide (2003) 180,000 deaths worldwide (2003) Cost to US Healthcare Cost to US Healthcare – – US$15 billion per annum US$15 billion per annum Poor Outcomes Poor Outcomes Improved Outcomes Improved Outcomes

Chronic Obstructive Pulmonary Disease Chronic Obstructive Pulmonary Disease

Current Management Current Management

Bronchodilators Bronchodilators Patients trialed on steroids Patients trialed on steroids 1 in 5 respond to steroids 1 in 5 respond to steroids 4 in 5 palliative care 4 in 5 palliative care

Aridol AridolTM

TM Management

Management

Aridol AridolTM

TM selects steroid responders

selects steroid responders Reduces steroid exposure Reduces steroid exposure Reduces healthcare costs Reduces healthcare costs Disease monitoring Disease monitoring Dose Dose optimisation

• ptimisation

30 million people affected worldwide 30 million people affected worldwide 4 4th

th leading cause of death

leading cause of death Cost to US healthcare Cost to US healthcare -

• US$40 billion pa

US$40 billion pa Poor Outcomes Poor Outcomes Improved outcomes Improved outcomes

Aridol AridolTM

TM opportunity by market segment

• pportunity by market segment

440 3,052 5,966 17,299 1,308 3,660

Lung testing asthma-diag asthma mgmt-spec asthma mgmt-GP diag-copd mgmt-copd

Target patient population (000’s)

20 40 60 80

Lung test- spec asthma mgt- spec asthma diag- GP asthma mgmt-GP diag-copd mgmt-copd

16% 16% 9% 29% 11% 75%

$m Total opportunity = $210m+ Potential revenue $ Penetration %

blue-asthma green - COPD

Bronchitol BronchitolTM

TM –

– breaks the mucus cycle breaks the mucus cycle

COPD / Cystic Fibrosis COPD / Cystic Fibrosis

Three way action to accelerate Three way action to accelerate sputum clearance sputum clearance Improves lung function Improves lung function Improves quality of life Improves quality of life Patient friendly Patient friendly Complementary with other Complementary with other treatments treatments Target Profile Target Profile

• Reduce exacerbations

Reduce exacerbations

• Reduce

Reduce hospitalisations hospitalisations

• Extend life expectancy

Extend life expectancy

impaired mucus clearance infection increased mucus production inflammation

X

Chronic bronchitis Chronic bronchitis

without without Bronchitol Bronchitol

TM TM

Chronic bronchitis Chronic bronchitis

with with Bronchitol Bronchitol

TM TM (400mg)

(400mg)

Bronchitol BronchitolTM

TM Opportunity

Opportunity

0.075 0.58 27,000

10000 20000 30000

CF BCS CB

$340 $370 $890

$0 $500 $1,000

CF BCS CB

Healthy uptake from cystic fibrosis patients (30%) Healthy uptake from cystic fibrosis patients (30%)

• Current treatments used by 33% patients

Current treatments used by 33% patients

Moderate uptake from Moderate uptake from bronchiectasis bronchiectasis patients (9%) patients (9%)

• Severe patients

Severe patients

Low uptake from chronic bronchitis patients (2%) Low uptake from chronic bronchitis patients (2%) Total revenue potential = $1.6 billion+ Total revenue potential = $1.6 billion+

Patient population (000’s) Patient population (000’s) Revenue potential (millions) Revenue potential (millions)

• -----COPD------
• -----COPD------

Next Steps Next Steps – – respiratory disease respiratory disease

July Aug Sep Oct Nov Dec Jan Feb Mar

Complete COPD PII study Submit AridolTM registration – Aus/EU AridolTM launch – Aus Complete CF PII study Commence CF PIII study Commence COPD PIII study FDA meeting AridolTM Commence CF dosing study Complete AridolTM PIII study

2004 2005

Results from AridolTM study available GP asthma management study commences-UK COPD PII study report CF comparator study commence-UK Presentation

• f COPD

study at ERS mid-05

Autoimmune Disease Autoimmune Disease

PXS25 PXS25

• Selective inhibitor of T cell

Selective inhibitor of T cell migration migration

• Novel mechanism of action

Novel mechanism of action

• Effective in MS and RA models

Effective in MS and RA models

• Complementary with existing

Complementary with existing treatments treatments

Competitive Edge Competitive Edge

• Delivery by oral route

Delivery by oral route

• Approach clinically validated

Approach clinically validated

Status Status

• Preclinical

Preclinical

• Human studies Q2 2005

Human studies Q2 2005

PXS2030 PXS2030

• Inhibitor of T cell migration

Inhibitor of T cell migration

• Inhibitor of B cell proliferation

Inhibitor of B cell proliferation

• Cannabinoid

Cannabinoid receptor agonist receptor agonist

• Effective in MS and RA models

Effective in MS and RA models

Competitive Edge Competitive Edge

• Delivery by the oral route

Delivery by the oral route

Status Status

• Research

Research

PXS2076 PXS2076

• Inhibitor of TNF

Inhibitor of TNF

• Novel mechanism of action

Novel mechanism of action

• Effective in RA models

Effective in RA models

Competitive Edge Competitive Edge

• Delivery by the oral route

Delivery by the oral route

Status Status

• Research

Research

Very large market opportunities

Finances Finances

6.1 1.5 25 Cash at June 2004 CF Start Grant P3

Financial Position Financial Position

Total Total -

• $32.6m

$32.6m

Summary Summary

1 1st

st product launch 2005

product launch 2005 Integrated business Integrated business Developing products for unmet clinical need Developing products for unmet clinical need Experienced management team Experienced management team TGA approved manufacturing facility TGA approved manufacturing facility Products from internationally respected groups Products from internationally respected groups Clinical validation for inhalation products Clinical validation for inhalation products

Investor Presentation Disclaimer Investor Presentation Disclaimer

This presentation is not an offer or invitation to subscribe for or purchase securities. To the extent that the presentation contains any forecasts, potential, projections or

• ther forward looking statements, those statements involve a number of known and

unknown risks, uncertainties and other facts that may cause actual results, performance or achievements to be materially different from those expressed or implied by those statements. There can be no guarantee that actual results, performance or achievements will be as stated. The reliance that the recipient of this presentation places on any statement, forecast, potential protection or assumption is a matter for its own commercial judgement. Pharmaxis Ltd does not undertake, nor is it obliged, to update or revise any statements, projections or forecasts or the assumptions underlying them whether as a result of new information, future events or otherwise.