ABN 75 082 811 630 Committed to the research, development and Committed to the research, development and commercialisation of human therapeutic products for commercialisation of human therapeutic products for chronic respiratory and autoimmune diseases chronic respiratory and autoimmune diseases February 2004 February 2004
Overview Overview Our Objective Our Objective � to build a valuable business, recognised internationally for its to build a valuable business, recognised internationally for its � approach to therapeutic discovery, development and approach to therapeutic discovery, development and commercialisation in the fields of respiratory and autoimmune commercialisation in the fields of respiratory and autoimmune diseases diseases Our Strategy Our Strategy � attractive product opportunities attractive product opportunities � � engage in the complete product value chain engage in the complete product value chain � � focus on products focus on products � � focus on core therapeutic markets focus on core therapeutic markets � � maintain and build a diversified product pipeline maintain and build a diversified product pipeline �
Company History Company History 1999 GBS Venture Partners invests $2M to develop GBS Venture Partners invests $2M to develop 1999 autoimmune disease research (ANU) autoimmune disease research (ANU) 2001 Licence from Royal Prince Alfred Hospital for Licence from Royal Prince Alfred Hospital for 2001 treatment of respiratory disease treatment of respiratory disease 2002 Second round funding of $9.6 Second round funding of $9.6 – – GBS Venture GBS Venture 2002 Partners, CM Capital, CIBC Australia, ANU and Partners, CM Capital, CIBC Australia, ANU and Mooroolbark Technology Mooroolbark Technology 2002 Consolidation of operations at Frenchs Frenchs Forest Forest 2002 Consolidation of operations at 2003 $3 million grant to develop cystic fibrosis treatment 2003 $3 million grant to develop cystic fibrosis treatment ≈ $5 million secured R&D grants totalling ≈ - secured R&D grants totalling $5 million - 2003 $25 million raised at IPO 2003 $25 million raised at IPO
TGA approved facilities TGA approved facilities Frenchs Forest NSW Forest NSW Frenchs
Products Products Name Category Indication Aridol lung function test asthma/COPD Aridol lung function test asthma/COPD TM TM Bronchitol therapeutic chronic bronchitis Bronchitol therapeutic chronic bronchitis TM TM bronchiectasis bronchiectasis cystic fibrosis cystic fibrosis PXS25 therapeutic multiple sclerosis PXS25 therapeutic multiple sclerosis rheumatoid arthritis rheumatoid arthritis PXS2000 therapeutic rheumatoid arthritis PXS2000 therapeutic rheumatoid arthritis other other
Diversified Product Portfolio Diversified Product Portfolio ---------------Clinical Trials----------------- research preclinical phase I phase II phase III registration Respiratory diseases TM - cystic fibrosis in Bronchitol progress TM - bronchiectasis in Bronchitol progress TM - chronic bronchitis Bronchitol TM – airway function in Aridol progress Autoimmune diseases in PXS25 - multiple sclerosis progress PXS25 - rheumatoid arthritis in PXS2000 – multiple sclerosis progress 3-5 years 15 months 15 months 18 months 18 months 12 months indicative time to complete
Clinical Trials in Progress Clinical Trials in Progress TM (Phase III) Aridol Q3 2004 CTM Nov 03 Jun 04 approvals dosing/recruitment (600 patients) manufacture Report 125 TM for cystic fibrosis (Phase IIa) Bronchitol Q3 2004 CTM Nov 03 Jun 04 approvals dosing/recruitment (60 patients) manufacture Report 1 Bronchitol for bronchiectasis (Phase IIb) CTM Q3 2004 Sep 03 June 04 approvals dosing/recruitment (60 patients) manufacture Report 19
TM - Bronchitol - bronchiectasis bronchiectasis Bronchitol TM Phase IIb IIb, pilot chronic study , pilot chronic study Phase 4 sites 60 subjects 4 sites 60 subjects � � Start: Oct 2003 Start: Oct 2003 � � Finish: June 2004 Finish: June 2004 � � Primary end point: quality of life Primary end point: quality of life � � Secondary end point: lung function, sputum properties Secondary end point: lung function, sputum properties � � Outcome: proof of principle for long term efficacy of Outcome: proof of principle for long term efficacy of � � TM TM Bronchitol Bronchitol Status: Interim analysis (19 subjects) completed Status: Interim analysis (19 subjects) completed � �
Trial Design Trial Design Design Design Randomised Randomised � � Placebo controlled Placebo controlled � � Blinded Blinded � � Crossover Crossover � � Objective Objective TM on disability (symptoms) and handicap TM Effect of twice daily Bronchitol Bronchitol on disability (symptoms) and handicap Effect of twice daily � � (sleep quality, fatigue, and QOL) (sleep quality, fatigue, and QOL)
Positive Interim Results Positive Interim Results Quality of Life (St George Questionnaire) Quality of Life (St George Questionnaire) Trend versus placebo Trend versus placebo � � significant versus control significant versus control � � Symptoms Symptoms Trend versus placebo Trend versus placebo � � Trend versus control Trend versus control � � Activity (disturbances to patients physical activity) Activity (disturbances to patients physical activity) Trend versus placebo Trend versus placebo � � Trend versus control Trend versus control � � Impact (disturbances of psycho- -social function) social function) Impact (disturbances of psycho Significant versus placebo Significant versus placebo � � Highly significant versus control Highly significant versus control � �
Next Steps……… ……… Next Steps Complete remainder of study Complete remainder of study Complete preclinical safety program Complete preclinical safety program Prepare for Phase III study Prepare for Phase III study
Trial Participants Trial Participants “It has been some weeks since my part of the trial It has been some weeks since my part of the trial “ finished and to be honest I have not felt as well as when finished and to be honest I have not felt as well as when I was on the Bronchitol Bronchitol. .” ” I was on the Trial participant 1 Trial participant 1 “Thank you for any help that you can give and I wish you “ Thank you for any help that you can give and I wish you well with the Bronchitol Bronchitol project as it has made such a project as it has made such a well with the difference to my health.” ” difference to my health. Trial participant 2 Trial participant 2 “This patient has been on the Phase II clinical trial of This patient has been on the Phase II clinical trial of “ Bronchitol for for bronchiectasis bronchiectasis. This has . This has revolutionised revolutionised her her Bronchitol life, she would benefit and would be greatly relieved of life, she would benefit and would be greatly relieved of her daily symptoms if she could continue this treatment.” ” her daily symptoms if she could continue this treatment. Physician Physician
Respiratory diseases Respiratory diseases (Chronic obstructive lung diseases) (Chronic obstructive lung diseases) Lung defence (normal) Lung defence (normal) Mucus Mucus Airway Surface Airway Surface Cilia Cilia Liquid Liquid Ciliated Cell Goblet Cell Submucosal glands Submucosal glands
Respiratory diseases Respiratory diseases (Chronic obstructive lung diseases) (Chronic obstructive lung diseases) Lung defence (compromised) Lung defence (compromised) Mucus Mucus Airway Surface Airway Surface Cilia Cilia Liquid (thin/viscous) Liquid (thin/viscous) Ciliated Cell Goblet Cell Submucosal glands Submucosal glands
Respiratory diseases Respiratory diseases (Chronic obstructive lung diseases) (Chronic obstructive lung diseases) Lung defence (after Bronchitol Bronchitol) ) Lung defence (after Mucus Mucus Airway Surface Airway Surface Cilia Cilia Liquid Liquid Ciliated Cell Goblet Cell H 2 O H 2 O H 2 O H 2 O H 2 O Submucosal glands Submucosal glands
Chronic bronchitis Chronic bronchitis without Bronchitol Bronchitol without TM TM
Chronic bronchitis Chronic bronchitis TM (400mg) with Bronchitol Bronchitol (400mg) with TM
Bronchitol Bronchitol TM TM for Chronic Obstructive Lung Diseases for Chronic Obstructive Lung Diseases
TM Aridol TM Aridol Dose required to cause 15% fall in lung function 600 500 400 300 Aridol 200 100 0 Severe Moderate Mild Normal Asthma severity Current With Aridol monitoring Steroid use
Aridol TM Aridol TM for asthma management for asthma management
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