Therapeutic products Therapeutic products for respiratory diseases - - PowerPoint PPT Presentation

Therapeutic products Therapeutic products for respiratory diseases for respiratory diseases for respiratory diseases for respiratory diseases

July 2009

Forward Looking Statements

This presentation may contain forward-looking statements that are based on management’s current expectations and beliefs and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking

• statements. The forward-looking statements contained in this presentation include statements

about future financial and operating results, results of our clinical trials, status of our regulatory submissions, possible or assumed future growth opportunities and risks and uncertainties that , p g pp could affect Pharmaxis’ product and products under development. These statements are not guarantees of future performance, involve certain risks, uncertainties and assumptions that are difficult to predict, and are based upon assumptions as to future events that may not prove accurate Therefore actual outcomes and results may differ materially from what is expressed

• accurate. Therefore, actual outcomes and results may differ materially from what is expressed
• herein. In any forward-looking statement in which Pharmaxis expresses an expectation or belief

as to future results, such expectation or belief is expressed in good faith and believed to have a reasonable basis, but there can be no assurance that the statement or expectation or belief will result or be achieved or accomplished. Factors that could cause or contribute to such differences include, but are not limited to, factors discussed in the “Risk Factors and Other Uncertainties” section of our Form 20-F filed with the US Securities and Exchange Commission US Secu es a d c a ge Co ss o We are not under any duty to update forward-looking statements unless required by law. This investor presentation is not an offer of the sale of securities. 2

Development Pipeline Development Pipeline

• ----------Clinical Trial Phases-----------

Research preclinical phase I phase II phase III registration market Research preclinical phase I phase II phase III registration market

Aridol Aridol – asthma (Aus/EU/Korea) asthma (Aus/EU/Korea) Aridol Aridol – – asthma (US) asthma (US) Bronchitol Bronchitol – – bronchiectasis bronchiectasis (Aus) (Aus) Bronchitol Bronchitol – bronchiectasis bronchiectasis (US/EU) (US/EU) ( ) ( ) Bronchitol Bronchitol – – cystic fibrosis (EU/Aust) cystic fibrosis (EU/Aust) Bronchitol Bronchitol – – cystic fibrosis (US) cystic fibrosis (US) Bronchitol Bronchitol – – acute indications acute indications PXS25 PXS25 – – lung fibrosis lung fibrosis PXS4159 PXS4159 – – asthma asthma 3

Operational Highlights of Quarter 2, 2009 Operational Highlights of Quarter 2, 2009

• Phase 3 trial with Bronchitol in CF returns positive

lt result

• Oral presentation at the European annual cystic

fibrosis scientific meeting fibrosis scientific meeting.

• Meetings with European regulators outlined

regulatory review path regulatory review path

• Aridol New Drug Application accepted for review by

FDA

• PXS25 presented at the 2009 American Thoracic

Society meeting in San Diego y g g

4

Bronchitol for Cystic Fibrosis Bronchitol for Cystic Fibrosis

5

Not an approved pack – for illustration purposes only

Primary and key secondary endpoint Primary and key secondary endpoint – – CF 301 CF 301

120 140 )

FEV1 changes at week 26

60 80 100 n FEV1 (mL

6.5%

P=0.001

5.2%

20 40 60 n change in

P=0.002

• 40
• 20

Overall rhDNase Mean Bronchitol Control Safety:

• Bronchitol well tolerated
• Bronchitol well tolerated
• Favorable safety profile
• Adverse events – consistent between treatment groups

6

Mean change (ml) in FEV1 over time Mean change (ml) in FEV1 over time

140 )

Bronchitol

100 120 EV1 (mL)

Bronchitol

• vs. control

p<0.001 (overall)

60 80 nge in FE

∆ 77 mL p< 0.001 ∆ 97 mL p<0.001 ∆ 78 mL p<0.001

20 40 ean chan Mannitol Bronchitol 20 Week 0 Week 6 Week 14 Week 26 Me Mannitol Control Bronchitol Week 0 Week 6 Week 14 Week 26

Weeks of Treatment

7

CF301 CF301 – – Key Secondary Endpoint Key Secondary Endpoint

e

Absolute (ml) change from baseline in FEV1 over time for Absolute (ml) change from baseline in FEV1 over time for rhDNase rhDNase+ subjects + subjects 80 100 120

baseline

DNase + p = 0.008 40 60 80

L) from

p (overall)

∆ 63.8 p = 0.04 ∆ 71.8 p = 0.02 ∆ 116 p = 0.002

20 Mannitol Control

ange (m

Bronchitol

• 40
• 20

Week 0 Week 6 Week 14 Week 26

FEV1 Cha

Week 0 Week 6 Week 14 Week 26 Weeks of Treatment

F

8

Positioning Bronchitol in CF Treatment Positioning Bronchitol in CF Treatment

Grade of recommendation Mild Moderate/Severe

A Benefit is substantial

• None
• rhDNase
• Tobi (if p.a. present)

B

• rhDNase
• Hypertonic saline

Benefit is moderate

• Tobi (if p.a. present)
• Azithromycin (if p.a. present)
• Hypertonic saline
• Ibuprofen (FEV1>60%)
• Azithromycin (if p.a. present)
• Ibuprofen (FEV1>60%)
• Inhaled β2 agonists
• Inhaled β2 agonists

Insufficient evidence

• Other inhaled antibiotics
• Oral corticosteroids (18+ yr olds)
• Leukotriene inhibitors / cromolyn sodium

Leukotriene inhibitors / cromolyn sodium

• Anticholinergic bronchodilators
• N-acetylcysteine

Against

• Inhaled corticosteroids (if asthma / ABPA absent)

Oral corticosteroids (6 18 yr olds)

• Oral corticosteroids (6 -18 yr olds)

Source: Treatment Progression – CFF Guidelines

9

Cystic Fibrosis market research Cystic Fibrosis market research

The time commitment to treatment is the biggest challenge to physicians and patients

• Time requirements and adherence to

th i h ll

Time

gg g p y p

therapy are pervasive challenges

• ”the treatments take time. Although

the payback is longevity and QOL, at the moment the treatments can take

Adherence

up a large part of the day.”

• ”patients feel very pressed for time.”
• ”Because of the time

requirement, you have to prioritise

Adherence /discipline

Financial

Obstacles to CF treatment

meds sometimes. Do the biggest bang for the commitment buck.”

• ”The time element is the key to

adherence.”

Drug

• ”Therapy gets in the way of daily

activities – 50 minutes two times a day!”

• Treating resistance to antibiotics is

Compliance Drug resistance

g another challenge for physicians

Source: Willowdale market research

10

Positioning Bronchitol in CF Treatment Positioning Bronchitol in CF Treatment

Mucus Alteration / Liquid Restoration CF Products Mucus Alteration / Liquid Restoration CF Products Pulmozyme Hypertonic Saline Bronchitol Denufosol Moli1901 Saline Company Genentech n/a Pharmaxis Inspire AOP St t M k t N t Ph III Ph III Ph II Status Market Not registered Phase III Phase III Phase II Administration Nebulizer Nebulizer Dry powder inhaler Nebulizer Nebulizer inhaler Dosing 1x daily 2-3x daily 2x daily 3x daily 1x daily Administration 20 minutes 20 minutes 3-5 minutes 20 minutes 20 minutes Time (per dose) 20 minutes 20 minutes 3 5 minutes 20 minutes 20 minutes FEV1 Benefit 5.6% 3.2%(n.s.) 6.5% 1-2% 2% All products complimentary to anti-infective & anti-inflammatory therapies 11

Sizing the CF Market Opportunity Sizing the CF Market Opportunity

Patients (75,000)

EU Top RoW

Worldwide sales of rhDNase US$476m (2008)

EU Top Five 27,000 5,000 USA EU $201m EU Other 13,000 USA 30,000 USA $275m

Worldwide sales of rhDNase (2005 – 2008)

426 476 400 450 500

CF Market US EU (T5)

Existing use of rhDNase 62% 52%

299 358 200 250 300 350 400 US$m

Annual cost US$22k US$13k CF Centres 110 350 R i d Fi ld F 15 25

Source: 2005 – 2008 Roche annual reports Note: Sales are converted from CHF to USD using the exchange rate on the last day of each financial year

00 2005 2006 2007 2008 Year

Required Field Force ~15 ~25 12

Commercialisation Timetable Commercialisation Timetable -

• CF

CF

Europe USA

H2 2009 Fil MAA i EU ( t li d) Close Recruitment Second Phase III Trial H2 2009 File MAA in EU (centralised) Close Recruitment Second Phase III Trial (CF302) H1 2010 Second Phase III Trial Reports H2 2010 Earliest Anticipated Approval File NDA H1 2011 Target sales Earliest Anticipated Approval H2 2011 Target sales

13

Manufacturing Capacity Manufacturing Capacity

• Current GMP facility
• Manufactures Aridol for sale in EU, Asia & Australia
• Manufacture Bronchitol for clinical trials
• New facility
• Relocated May 2009
• Equipment installation & validation complete - Q3 2009
• Complete process validation – Q2 2010
• Capacity
• Initial capacity - 1 spray drier:

40,000 patients p.a.

• Expanded capacity – 2nd spray drier: 80,000 patients p.a.

14

Aridol Aridol™ ™

• Identifies airway reactivity (active airway inflammation) which

helps physicians in the diagnosis and management of asthma

• An easy-to-use test kit provides rapid results and doesn’t

require specialized equipment q p q p

15

Major near term catalysts ahead Major near term catalysts ahead

Milestone 3Q-09 4Q-09 1Q-10 2Q-10 Bronchitol – cystic fibrosis Bronchitol – cystic fibrosis

P III trial (CF301) Additional data available File MAA in EU (centralised) P III trial (CF302) fully enrolled P III trial (CF302) data available

Bronchitol – bronchiectasis

MAA decision (Aus) Start 2nd P III trial enrollment Complete 2nd PIII enrollment

Aridol Aridol

U.S. NDA complete response

Facilities

New factory validation complete New factory validation complete

PXS25

Commence Phase 1 program

16

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