Investor Presentation Gary Phillips CEO 15 May 2017 1 Forward - PowerPoint PPT Presentation

Investor Presentation Gary Phillips CEO 15 May 2017 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 create value Drug development Management Partnerships Financial strength  Focus on fibrosis and  Management and Board  First drug out licensed to  A$26.5m cash balance at inflammation with global experience & Boehringer Ingelheim in Mar 2017; average annual globally competitive Pharma network cash usage $1.4m/month  Strong Pharma interest deal - total potential  Proven capability of  Boehringer NASH phase 2 in validated small deal >A$750m molecule technology executing global BD with initiation milestone  Synairgen collaboration platform major partners expected Q2 2017 €18m for LOXL2  Several new drugs acting  In house capability to  Boehringer 2nd indication  Significant value on high value targets in run multi-centre phase 2 milestone of milestones from existing current pipeline international trials €10m expected H2 2017 partner deals near term  Market cap $83m*  Pipeline providing  Institutional investor’s multiple future ~50% opportunities  Increasing Bronchitol sales globally in new and existing markets 3 * Note: Market Cap as of 11/05/17

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 18 years’ experience in pharmaceutical drug experience in the pharmaceutical and healthcare industry discovery and published more than 30 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 30 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 Board of Directors Brett Charlton - Medical  more than 25 years experience in clinical trial design and   Will Delaat – Non executive director Malcolm McComas – Chair management – – former CEO of Merck Australia former investment banker at  author of more than 80 scientific papers – former chair of Medicines Australia Grant Samuel, County Natwest  founding Medical Director of the National Health Sciences Centre and Morgan Grenfell  previously held various positions with the Australian  National University, Stanford University, the Baxter Gary Phillips – Managing director  Simon Buckingham – Non executive director Centre for Medical Research, Royal Melbourne Hospital, – former President Global Corporate and and the Walter and Eliza Hall Institute Business Development at Actellon 4

Pharmaxis product portfolio Lead Pre Phase Indication Discovery Phase I Phase II Marketed Optimisation Clinical III Bronchitol US Cystic fibrosis RoW Distributors Cystic fibrosis Aridol Asthma diagnosis Distributors SSAO NASH 2 nd indication SSAO Discovery NASH, fibrosis - LOXL-2 liver, pulmonary, kidney Respiratory & SSAO/MPO cardiovascular LOX Scarring+ LOXL-2 (other) Leading universities/academics assessing in cancer Cancer Dry powder Orbital inhalation device ASM-8 Seeking Partners Asthma 5

Pharmaxis drug discovery strategy Achieving value in the high risk world of early stage drug development • Focus on inflammation and fibrosis • Validated targets that are of value to pharma Prioritise • Initiate early pharma discussions to guide development program validated targets • Expertise in inflammation & fibrosis • Proven capability with small molecules from amine oxidase chemistry platform • Capability in drug discovery, preclinical and clinical development Leverage • Expand capability in inflammation & fibrosis expertise • Develop to phase 1 or 2 • Engage with pharma to understand value of deals at phase 1 vs phase 2 Assess risk vs • Collaborate to de-risk, expand and/or accelerate programs return 6

Achievements to date Building a biotech powerhouse in fibrosis and inflammation Drug discovery • First in class SSAO inhibitor drug taken to phase 1. • Initial indication NASH. • Partner developing second indication. • Two lead candidates in preclinical tox studies • Two further lead candidates to enter preclinical in 2017 Partnering • In house BD expertise achieves valuable deal with Boehringer Ingelheim - A$39m upfront, total potential > A$750m • Collaboration with Synairgen Research plc for early stage fibrosis program to widen spread of indications, enhance time to value inflection and spread risk 7

Drug discovery chemistry platform Amine oxidase enzymes; well validated targets in diseases with a high unmet medical need Alzheimer’s Ophthalmology Parkinson’s Stroke COPD Asthma CF Cardiomyopathy Pulmonary Fibrosis Heart failure Atherosclerosis NASH Liver fibrosis Liver cancer Scarring Kidney fibrosis Pancreatic cancer Gastric cancer Type 2 diabetes IBD Current focus on Liver fibrosis and NASH 8

30-38% progress to fibrosis 5-10% progress to NASH 3-5% progress to hepatocellular 30-40% of US population have (Non-alcoholic steatohepatitis) carcinoma steatosis (fatty liver) 9

10

Drugs in the clinic targeting NASH Several large Pharma companies seeking to build competitive portfolios Metabolic Anti- Anti-fibrotic modifiers inflammatory Intercept Ph 3 Genfit Ph 3 Galmed Ph 2/3 Allergan Ph 2 Ph 2 Gilead Ph 2 x 2 Ph 2 BMS Ph 2 Ph 1 Galectin Ph 2 Novartis Ph 2 AstraZeneca Ph 2 Shire Ph 2 Boehringer Ingelheim Ph 1 Other Ph 2 x 3 Ph 2 x4 11

SSAO for NASH SSAO inhibitor PXS4728A sold to Boehringer Ingelheim in May 2015 PXS-4728A End of Phase 1 deal with Boehringer  Mechanism based inhibitor of SSAO  Potential milestones to approval: €418.5m (~A$600m ) – Small molecule oral drug – – Important pathway in several Upfront (May 2015): €27.5m (~A$39m) inflammatory diseases of the liver, kidney, 1 st Indication (NASH) – heart, eye and CNS. • Commencement of phase 2 and 3: total €55m  Development status (~A$80m) • Filing, regulatory & pricing approvals: total – Pharmaxis discovery – patent filed 2012 €140m(~A$200m ) – 2 nd indication (commercial in confidence) – Effective in pre clinical models of NASH and airway inflammation • Additional milestone payments to approval total – €195m (~A$280m) Phase 1 study reported  Earn-out payments on annual net • orally bioavailable sales • long lasting enzyme inhibition after single dose – Tiered percentages increasing from high • progressive dose response single digits – – Phase 2 NASH trial scheduled H1 2017 Plus sales milestones External validation of PXS drug discovery and ability to negotiate valuable global deals 12