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Investor Presentation 7 th August 2015 Gary Phillips CEO 1 Forw - PowerPoint PPT Presentation

innovating for life Investor Presentation 7 th August 2015 Gary Phillips CEO 1 Forw ard looking statement This document contains forward-looking statements, including statements concerning Pharmaxis future financial position, plans, and the

  1. innovating for life Investor Presentation 7 th August 2015 Gary Phillips CEO 1

  2. Forw ard looking statement This document contains forward-looking statements, including statements concerning Pharmaxis’ future financial position, plans, and the potential of its products and product candidates, which are based on information and assumptions available to Pharmaxis as of the date of this document. Actual results, performance or achievements could be significantly different from those expressed in, or implied by, these forward-looking statements. These forward-looking statements are not guarantees or predictions of future results, levels of performance, and involve known and unknown risks, uncertainties and other factors, many of which are beyond our control, and which may cause actual results to differ materially from those expressed in the statements contained in this document. Except as required by law we undertake no obligation to update these forward-looking statements as a result of new information, future events or otherwise. 2

  3. Innovate Develop Partner Pharmaxis overview our path to value Strategy Opportunities Achievements  Build a regional biotech  Milestone payments from  First in class NASH drug powerhouse in fibrosis and Boehringer as PXS4728A taken to phase 1 inflammation progresses in NASH  In house BD expertise  Multiple drugs from  Synairgen LOXL2 lands deal - A$39m amine oxidase platform collaboration in pulmonary upfront, total up to fibrosis to phase 1 or 2 and A$750m  Develop to phase 1 or 2 subsequent partnering  Restructured Bronchitol  Create value via partnering  3 additional drug programs business to reduce  Licence out to Big in drug discovery pipeline investment (>50%) and Pharma with attractive shorten time to profitability 1 st in class drugs post  A stake in US commercialisation of  Attracted collaborators phase 1 or 2 Bronchitol (funded by into early stage fibrosis  Collaborate to de-risk partner) and sales by program to widen spread and accelerate PXS distributors in rest of world of indications, enhance programs time to value inflection and  Resources for collaborating  Collaborate on in- spread risk on selected in-licensing licensing programs 3

  4. Pharmaxis today building a regional biotech powerhouse in fibrosis and inflammation Manufacturer Drug developer BD expertise Financial strength  Supplies Bronchitol  Leading position in  Experienced  $54m cash balance to global markets amine oxidase management team at June 2015 and via experienced chemistry and and board reduced cash burn commercial partners mechanism based  Extensive Pharma  Significant value inhibitors  Financial risks industry network milestones from minimised/shared  Proven capability in existing partner  Proven capability of delivering quality deals within reach  Financial upside executing global programs to achieve from accessing new transactions with  Cash strengthens phase 2 ready markets major partners negotiating position compounds in future licensing  Possibility to further  BI deal energises  Exciting pipeline of activities rationalise ongoing pharma drug candidates for manufacturing interest in platform valuable targets infrastructure programs 4

  5. Pharmaxis product portfolio Product Indication Status Partner Aridol Asthma diagnosis Marketed: Australia, EU, Korea Various Bronchitol US Cystic Fibrosis Phase 3 study underway Chiesi Bronchitol EU Cystic Fibrosis Marketed Chiesi Bronchitol rest of Cystic Fibrosis Marketed: Australia, CEE Various world Approval pending; Brazil, Russia ASM8 Asthma Phase 2 - Orbital Dry powder inhalation device Phase 1 - SSAO inhibitor NASH Phase 1 Boehringer SSAO/MAOB Neuro inflammation; Lead candidate selected - inhibitor Alzheimer's, MS, etc. SSAO/MPO inhibitor Respiratory inflammation; Lead optimisation - Asthma, COPD LOXL2 inhibitor NASH, Liver & kidney fibrosis Lead optimisation - LOXL2 inhibitor (IPF) Idiopathic pulmonary fibrosis Lead optimisation Synairgen LOX/LOXL2 inhibitor Fibrosis, cancer Exploratory LOX inhibitor Cancer, scarring Exploratory 5

  6. Drug discovery strategy Exploiting the amine oxidase chemistry platform •Identify Target / Disease condition Drug discovery objective •Research mechanism of action, points of intervention •Develop chemistry strategy at least one new drug candidate to Stage 1 •Develop predictive assays / animal models Exploratory complete formal pre clinical testing and be phase 1 ready each year •pK / pD evaluation •Informal toxicity studies •Lead candidate selection Stage 2 •6 - 12 months Lead optimisation Projects # of $m / projects project •Submit to full pharmacology panel Exploratory 2 - 4 0.2 •Manufacturing scale up •Toxicity and safety studies Stage 3 Lead •File IND Formal pre 2 - 3 1.5 clinical optimisation Formal 1 1.5 •1a - Single Ascending dose pre-clinical •1b - Multiple ascending dose Stage 4 •$2m approx Phase 1 Indicative costs per project clinical study (including FTE & laboratory costs) 6

  7. Our therapeutic focus the inhibition of amine oxidase based enzymes has broad potential applications Alzheimer’s COPD Parkinson’s Asthma Stroke CF Pulmonary Fibrosis Cardio-myopathy Heart failure NASH Atherosclerosis Liver fibrosis Liver cancer Scarring Kidney fibrosis Pancreatic cancer Gastric cancer Type 2 diabetes IBD there is a strong positive correlation between increases in amine oxidase activity and these diseases. Confidential 7

  8. Biology of amine oxidase based enzymes amine oxidase based enzymes facilitate inflammatory and fibrotic processes Genes Infection environment Metabolic disorders Chronic Fibrosis inflammation Leukocyte excess Cancer Oxidative stress SSAO/VAP-1 SSAO/VAP-1 LOX LOX LOXL2 LOXL2 MAO SSAO/VAP-1 Retina SSAO inhibition of these enzymes give multiple potential pathways to treat several important diseases 8

  9. SSAO inhibition and NASH a novel therapeutic target  Primary indication: NASH  ~US$3.5b market by 2025  Estimated 6 million patients in US Increasing levels of SSAO  Development status: Metabolic Syndrome  Pharmaxis discovery – patent filed 2014 • Modulates leucocyte  Effective in pre clinical models of Non migration Alcoholic NASH and airway inflammation Fatty Liver  Completed single ascending dose Disease • Local generation of stage of phase 1 Reactive Oxygen  orally bioavailable Species Non Alcoholic  long lasting inhibition after single Steatohepatitis dose • Promotes  progressive dose response inflammation Liver  PXS total investment to Cirrhosis • Promotes fibrosis phase 1:  ~A$9m  Competitors: Liver Carcinoma  Genefit – GF505 in Phase 2b NASH  Intercept - OCA (FXR agonist) in Phase 2b NASH 9  Gilead – FXR agonist in pre clinical

  10. Boehringer Ingelheim acquisition of PXS4728A Average drug development times Acquisition (May 2015). • €27.5m (~A$39m) Commencement of phase 2 and 3 • up to total €55m (~A$80m) Source: Pharmaceutical Research and Manufacturers of America Filing, regulatory &  Excellent partner pricing approvals  Boehringer leaders in metabolic disease  Industry leading development times • up to total €140m(~A$200m)  Boehringer responsible for all development, and commercialisation activities Second indication  Competitive deal • additional total milestone  Demonstrates PXS ability to negotiate valuable global payments (€195m) deals  Total potential payments to approval for 2 indications: €418.5m (~A$600m), Earn-out payments on  Plus potential sales milestones, and potential earn- annual net sales out at high single digit % of sales • tiered percentages starting in high single digits  External validation of PXS drug discovery 10

  11. Excessive production and linking of LOXL2 inhibition collagen fibres results in fibrosis an attractive target and development program  Potential indications:  NASH / Liver Fibrosis  Pulmonary fibrosis Fibroblast cells in human tissue  Cancer  Wound healing LOXL2 (from fibroblasts)  Development status:  Pharmaxis discovery – patent filed 2014  Lead compounds with differentiated PK / PD profile identified Excessive ‘cross-linking’ of Collagen fibres  Effective in pre clinical models of fibrosis and cancer collagen fibres, stiffens tissue, causing fibrosis  Competitive profile:  Novel target and mechanism of action Gilead – LOXL2 antibody  Once daily oral drug • Acquired Arresto program $225m pre  Complete inhibition of LOXL2 versus partial inhibition phase 1 by antibody • Now in broad phase 2b trial program  Low cost of goods • Liver fibrosis; Idiopathic pulmonary fibrosis; Metastatic pancreatic cancer; Myelofibrosis; Solid tumours; Metastatic colorectal cancer 11

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