Therapeutic products Therapeutic products for for respiratory and respiratory and autoimmune diseases autoimmune diseases Annual General Meeting Annual General Meeting November 2005 November 2005
Highlights Highlights Bronchitol: Entering Phase III Bronchitol: Entering Phase III Successful Phase II trial in cystic fibrosis Successful Phase II trial in cystic fibrosis � � Successful Phase II trial in bronchiectasis bronchiectasis Successful Phase II trial in � � Bronchitol Bronchitol Orphan drug designation – – Europe and USA Europe and USA Orphan drug designation � � Aridol: Management of airway inflammation Aridol: Management of airway inflammation European Phase III completed (asthma) European Phase III completed (asthma) � � US Phase III to start late 2005 (asthma) US Phase III to start late 2005 (asthma) Aridol Aridol � � Market authorization filed in EU, Australia (target 2006 launch) Market authorization filed in EU, Australia (target 2006 launch) � � COPD clinical study commenced COPD clinical study commenced � � Retained marketing rights for all programs Retained marketing rights for all programs Autoimmune Autoimmune disease disease Experienced management Experienced management Extensive patent portfolio Extensive patent portfolio Near term value enhancing milestones Near term value enhancing milestones Manufacturing Manufacturing 13 13
Pipeline Pipeline Pulmonary and Autoimmune Focus Pulmonary and Autoimmune Focus --------Clinical Trials---------- research preclinical phase I phase II phase III registration # pts (mm) Respiratory diseases Respiratory diseases Aridol – – asthma asthma Aridol EU/Aus 52 30 Aridol - - COPD COPD Aridol Bronchitol - - bronchiectasis bronchiectasis Bronchitol 0.6 Bronchitol – – cystic fibrosis cystic fibrosis Bronchitol 0.1 Bronchitol - - chronic bronchitis chronic bronchitis Bronchitol 30 Autoimmune diseases Autoimmune diseases PXS25/64 - - multiple sclerosis multiple sclerosis PXS25/64 1.0 PXS2076 – – rheumatoid arthritis rheumatoid arthritis PXS2076 6.0 14 14
Management Management Alan Robertson PhD Alan Robertson PhD CEO CEO Wellcome (GSK); Faulding; (GSK); Faulding; Amrad Amrad; Inventor of ; Inventor of Zomig Zomig Wellcome David McGarvey CA CFO David McGarvey CA CFO CFO, Memtec (NYSE); CFO, US Filter Filtration Group CFO, Memtec (NYSE); CFO, US Filter Filtration Group Brett Charlton, PhD CMO Brett Charlton, PhD CMO Stanford; ANU Stanford; ANU Gary Phillips, MBA Commercial Gary Phillips, MBA Commercial CEO, CEO, Novartis Novartis Australia Australia John Crapper, MBA COO John Crapper, MBA COO Managing Director, Memcor Memcor; ; Syntex Syntex (Roche) (Roche) Managing Director, William Cowden, PhD CSO William Cowden, PhD CSO ANU; Co ANU; Co- -inventor of TNF inventor of TNF mAb mAb’ ’s s Ian McDonald, PhD CTO Ian McDonald, PhD CTO VP Discovery, SIBIA (Merck); VP Discovery, SGX VP Discovery, SIBIA (Merck); VP Discovery, SGX 15 15
Bronchitol Bronchitol cystic fibrosis cystic fibrosis Background Background Genetic disorder affecting 30,000 in U.S. Genetic disorder affecting 30,000 in U.S. � � Poorly hydrated, tenacious, thick mucus Poorly hydrated, tenacious, thick mucus � � Current life expectancy is 31 years Current life expectancy is 31 years � � Current treatments: rhDNase Current treatments: rhDNase and tobramycin and tobramycin Delivered by nebulizer (preparation, sterilization) Delivered by nebulizer (preparation, sterilization) � � rhDNase rhDNase ( (pulmozyme pulmozyme): $265mm @ ~30% penetration ): $265mm @ ~30% penetration � � 16 16
Bronchitol Bronchitol Phase II CF trial Phase II CF trial Crossover, 8 site study in 39 CF patients Crossover, 8 site study in 39 CF patients Randomised two week treatment periods Randomised two week treatment periods Double- -blind, placebo controlled blind, placebo controlled Double Primary Endpoint: Primary Endpoint: Change in FEV 1 Change in FEV � � 1 Secondary Endpoints: Secondary Endpoints: Effect on other lung function measures Effect on other lung function measures � � Effect on symptoms/signs Effect on symptoms/signs � � Effect on QoL QoL Effect on � � Safety (including microbiology) Safety (including microbiology) � � 17 17
Bronchitol Bronchitol CF Phase II Results: Change in Lung Function CF Phase II Results: Change in Lung Function Bronchitol* Control* p value Bronchitol* Control* p value Change in FEV 1 Change in FEV 7 ± 7 ± 2% 2% 0 ± 0 ± 2% 2% 0.008 0.008 1 Change in FEF 25 15.5 ± ± 5% 5% 0.6 ± ± 5% 5% < 0.01 Change in FEF 15.5 0.6 < 0.01 25- -75 75 *includes patients being treated with pulmozyme (FEF 25 (FEF 75 or MMEF is considered a measure of small airway function) or MMEF is considered a measure of small airway function) 25- -75 18 18
Bronchitol Bronchitol CF Phase II Results: FEV 1 Change CF Phase II Results: FEV 1 Change 200 * FEV 1 change (mls) p = 0.008 150 100 50 0 Bronchitol Control 19 19
Bronchitol Bronchitol CF Phase II Results: FEF 25 Change CF Phase II Results: FEF 75 Change 25- -75 p<0.01 * 200 150 FEF 25-75 (mls/sec) 100 50 0 -50 -100 Bronchitol Control 20 20
Bronchitol Bronchitol cystic fibrosis registration strategy cystic fibrosis registration strategy Phase III trial (EU & Aus): Phase III trial (EU & Aus): Dosing to be finalized based on ongoing dose- -ranging study ranging study Dosing to be finalized based on ongoing dose � � Commence 1H2006 Commence 1H2006 � � Primary endpoint: Change in FEV 1 Primary endpoint: Change in FEV � � 1 Placebo- -controlled, 6 month dosing, finalising design with EMEA controlled, 6 month dosing, finalising design with EMEA Placebo � � Phase III trial (US) to commence 2006 Phase III trial (US) to commence 2006 Similar size, design to EU/Aus trial Similar size, design to EU/Aus trial � � Orphan drug designation – Orphan drug designation – EU and USA EU and USA 21 21
Bronchitol Bronchitol bronchiectasis bronchiectasis Background Background Abnormal, irreversible dilation of the lower airways Abnormal, irreversible dilation of the lower airways � � Daily mucus production, constant coughing, breathlessness: Daily mucus production, constant coughing, breathlessness: � � major quality of life impact major quality of life impact Normal lung clearance impaired Normal lung clearance impaired � � 100,000 affected in the U.S. 100,000 affected in the U.S. � � Current treatments: bronchodilators, antibiotics Current treatments: bronchodilators, antibiotics No drugs effective to clear mucus No drugs effective to clear mucus � � 22 22
Bronchitol Bronchitol bronchiectasis bronchiectasis Phase II Trial results Phase II Trial results 60 patient, double- -blind, crossover, placebo blind, crossover, placebo- -controlled controlled 60 patient, double � � Promising results in QoL Promising results in QoL, symptom scores (p<0.05 versus placebo) , symptom scores (p<0.05 versus placebo) � � For all patients – – 4.5 unit improvement in St. George 4.5 unit improvement in St. George’ ’s impact score s impact score For all patients � � For the 75% of patients with unclear chests – For the 75% of patients with unclear chests – 6.9 unit improvement in St 6.9 unit improvement in St � � George’ ’s impact score s impact score George Well tolerated, no adverse events Well tolerated, no adverse events � � Phase III Trials Phase III Trials Plan to commence 4Q05/1Q06 in Australia, EU Plan to commence 4Q05/1Q06 in Australia, EU � � Finalising protocol following FDA meeting Finalising protocol following FDA meeting � � Initiate US pivotal trial mid- -2006 2006 Initiate US pivotal trial mid � � Supplied on compassionate- -use basis in Australia use basis in Australia Supplied on compassionate 23 23
Bronchitol Bronchitol chronic bronchitis chronic bronchitis Background Background Chronic cough, breathlessness, tenacious sputum Chronic cough, breathlessness, tenacious sputum � � >30 million people affected in 7 major pharma pharma markets markets >30 million people affected in 7 major � � No therapy halts disease progression No therapy halts disease progression � � Current treatments aimed at symptom relief / bronchodilation bronchodilation Current treatments aimed at symptom relief / � � Acute pilot studies completed Acute pilot studies completed Phase II clinical protocol in development Phase II clinical protocol in development Quality of Life Quality of Life � � Reduction in exacerbation period Reduction in exacerbation period � � Study to commence 2006 Study to commence 2006 24 24
TM Aridol TM Aridol A rapid and simple test for airways inflammation that facilitates s A rapid and simple test for airways inflammation that facilitate diagnosis and management of asthma and COPD patients. diagnosis and management of asthma and COPD patients. 25 25
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