Investor Presentation Gary Phillips CEO 6 September 2016 1 Forward - PowerPoint PPT Presentation

Investor Presentation Gary Phillips CEO 6 September 2016 1

Forward 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

Business overview Built to deliver value Drug development Management Partnerships Financial strength  Focus on fibrosis and  Management and Board  First drug out licensed to  A$39m cash balance at inflammation with global experience & Boehringer Ingelheim in June 2016; average globally competitive Pharma network monthly cash usage $1.3m  Strong Pharma interest deal ‐ total potential  Proven capability of  Boehringer phase 2 in validated small deal >A$750m molecule technology executing global BD with initiation milestone  Significant value platform major partners expected Q1 2017 milestones from existing ~A$25m  Three additional drugs  In house capability to partner deals near term  Market cap $94M* acting on high value run multi ‐ centre  Pipeline providing targets approaching the international trials  institutional investor’s clinic over next 24 multiple future >45% opportunities months  Increasing Bronchitol sales  Synairgen collaboration globally in new and developing additional existing markets indication 3 * Note: Market Cap as of 31/08/16

Senior management Significant experience in drug development, commercialisation and partnering Gary Phillips – CEO Wolfgang Jarolimek – Drug Discovery   more than 30 years of operational management more than 15 years’ experience in pharmaceutical drug experience in the pharmaceutical and healthcare industry discovery and published more than 20 peer reviewed in Europe, Asia and Australia articles.   joined Pharmaxis in 2003 and was appointed Chief previously Director of Assay Development and Compound Executive Officer in March 2013 at which time he was Profiling at the GlaxoSmithKline Centre of Excellence in Chief Operating Officer Drug Discovery in Verona, Italy   previously held country and regional management roles spent 8 years as post ‐ doc at the Max ‐ Plank Institute in at Novartis – Hungary, Asia Pacific and Australia Munich, Germany; Baylor College of Medicine, Houston, Texas; Rammelkamp Centre, Cleveland Ohio; and University of Heidelberg, Germany David McGarvey – CFO Kristen Morgan – Alliance  Management more than thirty years’ experience building and funding Australian based companies from inception to globally  responsibility for alliance management and medical and successful enterprises regulatory affairs  joined Pharmaxis as Chief Financial Officer and Company  more than 19 years’ experience in the pharmaceutical Secretary in December 2002 industry having previously held a senior role in medical  previously Chief Financial Officer of the Filtration and affairs at Sanofi ‐ Aventis, and a commercial sales role at Separations Division of US Filter (1998 ‐ 2002), and Memtec GlaxoSmithKline. Limited (1985 ‐ 1998)  commenced career at PriceWaterhouseCoopers Brett Charlton ‐ Medical Board of Directors    more than 15 years experience in clinical trial design and Will Delaat – Non executive director Malcolm McComas – Chair management former investment banker at former CEO of Merck Australia – –  author of more than 60 scientific papers Grant Samuel, County Natwest former chair of Medicines Australia –  founding Medical Director of the National Health Sciences and Morgan Grenfell Centre   Gary Phillips – Managing director Simon Buckingham – Non executive director  previously held various positions with the Australian former President Global Corporate and – National University, Stanford University, the Baxter Business Development at Actellon Centre for Medical Research, Royal Melbourne Hospital, and the Walter and Eliza Hall Institute 4

Pharmaxis product portfolio Lead Pre Phase Indication Discovery Phase I Phase II Marketed Optimisation Clinical III Bronchitol US Cystic fibrosis RoW Cystic fibrosis Distributors Aridol Asthma diagnosis Distributors SSAO NASH+ Discovery Neuro SSAO/MAO ‐ B inflammation Respiratory SSAO/MPO inflammation NASH, liver LOXL ‐ 2 fibrosis Pulmonary LOXL ‐ 2 (IPF) fibrosis Cancer, wound Leading universities/academics assessing in LOX/LOXL ‐ 2 healing kidney fibrosis, cancer and wound healing Dry powder Orbital Seeking Partners inhalation device ASM ‐ 8 Seeking Partners Asthma 5

Bronchitol for cystic fibrosis Overview Pooled adult data from CF301 and CF302 Cystic fibrosis Bronchitol CF301/2 trial (adult) CF204 trial results  Patients  Active ingredient  Total 317 adults  Paediatric age 6 ‐ 17 mannitol delivered as an  FEV1 – Placebo ‐ controlled – US: 30,000; inhalable dry powder – 8 weeks crossover design – CF301; p=0.001 – Europe: 37,000;  Restores airway surface – standard therapy – Rest of world: 21,000 – CF302; p=0.038 continued liquid  Disease characterised by – Pooled; p=0.001  Primary endpoint:  Mucus clearance poorly hydrated, rel % change = 4.7% – Absolute change in FEV1: enhanced tenacious, thick mucus  Exacerbations 3.42%; p=0.004  Improves lung function  Rapid decline in lung  Key secondaries – Pooled data  Reduces incidence of function – 26% reduction – Absolute change in lung infections  Frequent infections FEF25 ‐ 75: 5.75% – 60% reduction in (p=0.005) Bronchitol  Acceptable safety profile responders – Exacerbations and lung infection reduced by ~ 25% 6

Bronchitol for cystic fibrosis Partnering for success US market US partner: Chiesi US trial: CF303 Rest of world  Largest CF market by  Fund CF303 up to  Tie ‐ breaker phase 3 trial  Sold by Chiesi in UK & value US$22m commenced Q1 2015, Germany managed by PXS  28,103 CF patients  ~A$13m milestone  Sold by PXS in Australia  440 adult patients payment on launch, plus & Denmark  49.7% adults sales milestones  Pending – 20+ countries  Bronchitol price target  High mid teens royalty % approval/distributor US$20k per patient / – 130 sites on in ‐ market sales appointments in ten year  Design countries including  Mid teens % uplift on  7 year post launch – Full consultation with Russia, Israel, Turkey, COGs market exclusivity FDA Brazil, Eastern Europe  Chiesi responsible for – Similar design to  Additional distributors regulatory filing & CF301/2 being appointed commercialisation  Fully recruited July 2016  Results H1 2017 7

Drug discovery Applying amine oxidase chemistry to inflammation and fibrosis Alzheimer’s Ophthalmology Parkinson’s Stroke COPD Asthma CF Cardio ‐ myopathy Pulmonary Fibrosis Heart failure Atherosclerosis NASH Liver fibrosis Liver cancer Scarring Kidney fibrosis Pancreatic cancer Gastric cancer Type 2 diabetes IBD Amine oxidase enzymes are well validated as targets in Amine oxidase enzymes are well validated as targets diseases with a high unmet medical need in diseases with a high unmet medical need 8

Pharmaxis drug discovery strategy Building a biotech powerhouse in fibrosis and inflammation Strategy Achievements to date Drug discovery: Drug discovery:  First in class NASH drug taken to phase 1  Prioritise validated targets  Two further candidates in lead optimisation Multiple small molecule drugs from in ‐ – phase house amine oxidase chemistry platform  One lead candidate moving to preclinical  Develop to phase 1 or 2 Partnering: Partnering:  Create value via:  In house BD expertise achieves valuable deal with Boehringer Ingelheim ‐ A$39m upfront, Licence out to Big Pharma with attractive – total potential > A$750m 1st in class drugs post phase 1 or 2 Collaborate to de ‐ risk and accelerate PXS –  Collaboration with Synairgen Research plc for programs early stage fibrosis program to widen spread Collaborate on in ‐ licensing programs – of indications, enhance time to value inflection and spread risk 9

Drug discovery Our therapeutic focus is inflammation and fibrosis Pharmaxis drug discovery • NASH & liver fibrosis (LOXL2) • Respiratory – COPD, asthma, cystic fibrosis (SSAO/MPO) • Neuro inflammation – Alzheimer’s, Parkinson’s, stroke (SSAO/MAO ‐ b) Collaborations allow us to leverage our platform without losing focus Collaboration with Synairgen •Pulmonary fibrosis (LOXL2) Exploratory academic collaborations (LOX/LOXL2) •Scarring •Kidney fibrosis •Some cancers 10