Investor Presentation Investor Meetings San Francisco 11 th 15 th - PowerPoint PPT Presentation

innovating for life Investor Presentation Investor Meetings – San Francisco 11 th – 15 th January 2016 Gary Phillips CEO 1

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

Pharmaxis today new business focus creating value Drug developer Management Drug manufacturer Financial strength  Leading position in  Management team  Supplies Bronchitol  A$50m cash amine oxidase and Board with to global markets balance at chemistry and global experience via experienced September 2015 mechanism based commercial partners  Extensive pharma  Significant value inhibitors industry network  Financial risks milestones from  Proven capability in shared existing partner  Proven capability of delivering quality deals within reach executing global BD  Financial upside programs to achieve transactions with from accessing new  Growing institutional phase 2 ready major partners markets – US, presence on share compounds Russia register: >45%  Preclinical, early  Exciting pipeline of and late phase drug candidates for clinical experience valuable targets 3

Board and management experience that counts Broad network and experience in capital markets Board Biotech and Big Pharma commercial experience •Malcolm McComas – Chair •Will Delaat Extensive business development networks •Simon Buckingham •Gary Phillips – CEO Experience of wide variety of partnering transactions Biotech and Big Pharma commercial experience Management Hands on experience across the whole of the Pharma value chain •Gary Phillips – CEO Proven track record in business negotiations and deal •David McGarvey – CFO making •Brett Charlton – Medical Excellent industry and academic networks •Wolfgang Jarolimek – Drug Discovery •Kristen Morgan Australian and international capital markets – Alliance Management Small cap companies 4 Refer to Pharmaxis website for further detail

Pharmaxis product portfolio Product Indication Status LOXL2 inhibitor NASH (fatty liver disease), liver & Lead optimisation kidney fibrosis LOXL2 inhibitor Idiopathic pulmonary fibrosis Lead optimisation; collaboration with Synairgen amine LOX/LOXL2 inhibitor Fibrosis, cancer Exploratory oxidase chemistry SSAO inhibitor NASH Successful phase 1 study reported; platform sold to Boehringer SSAO/MAOB inhibitor Neuro inflammation; Alzheimer's, Lead candidate selected MS, etc. SSAO/MPO inhibitor Respiratory inflammation; Asthma, Exploratory COPD Orbital Dry powder inhalation device Phase 1 – seeking partner ASM8 Asthma Phase 2 - seeking partner Bronchitol US Cystic Fibrosis Partner: Chiesi, funding phase 3 study - currently underway Bronchitol EU Cystic Fibrosis Partner: Chiesi (UK & Germany) - marketed Bronchitol rest of world Cystic Fibrosis Marketed: Australia, CEE Approval pending; Brazil, Russia Aridol Asthma diagnosis Marketed: Australia, EU, Korea 5

Our therapeutic focus global leaders in amine oxidase enzyme chemistry COPD Alzheimer’s Asthma Parkinson’s CF Stroke Pulmonary Fibrosis Cardio-myopathy Heart failure NASH Atherosclerosis Liver fibrosis Liver cancer Scarring Kidney fibrosis Pancreatic cancer Gastric cancer Type 2 diabetes IBD Amine oxidase enzymes are well validated as targets in diseases with a high unmet medical need Confidential 6

Pharmaxis drug discovery strategy building a biotech powerhouse in fibrosis and inflammation Strategy Achievements to date Drug discovery: Drug discovery:  Improve drug discovery hit rate by:  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:  Licence out to Big Pharma with attractive  In house BD expertise achieves valuable 1 st in class drugs post phase 1 or 2 deal with Boehringer Ingelheim - A$39m  Collaborate to de-risk and accelerate PXS upfront, total potential > A$750m programs  Collaborate on in-licensing programs  Collaboration with Synairgen Research plc for early stage fibrosis program to widen spread of indications, enhance time to value inflection and spread risk 7

SSAO for NASH SSAO inhibitor PXS4728A sold to Boehringer Ingelheim in May 2015 PXS 4728A Boehringer Ingelheim  Excellent partner:  Mechanism based inhibitor of SSAO  Boehringer leaders in metabolic  Small molecule inhibitor of disease semicarbazide-sensitive amine oxidase  Industry leading development times / vascular adhesion protein (VAP-1)  Important inflammatory pathway in  Boehringer responsible for all development, and commercialisation several diseases including NASH and activities COPD  Competitive deal:  Development status:  Pharmaxis discovery – patent filed  Total potential payments to approval for 2 indications: €418.5m (~A$600m), 2012 • acquisition (May 2015): €27.5m  Effective in pre clinical models of NASH (~A$39m) and airway inflammation • commencement of phase 2 and 3:  Phase 1 study reported up to total €55m (~A$80m) • orally bioavailable • filing, regulatory & pricing approvals: • long lasting inhibition after single up to total €140m(~A$200m) dose • second indication: additional total • progressive dose response milestone payments (€195m)  Earn-out payments on annual net sales  Competitors: • tiered percentages starting in high Genefit – GF505 Phase 2b NASH  single digits (reported) • plus potential sales milestones  Intercept - OCA (FXR agonist) Phase  External validation of PXS drug 2b NASH (reported) discovery and ability to negotiate  Gilead – FXR agonist in pre clinical valuable global deals 8

LOXL2 inhibition for NASH & other fibrotic diseases an attractive target and development program Excessive production and linking of  Potential indications: collagen fibres results in fibrosis  NASH / Liver Fibrosis  Pulmonary fibrosis (IPF)  Cancer  Wound healing Fibroblast cells in  Development status: human tissue  Pharmaxis discovery – patent filed 2014  Lead compounds with differentiated PK / PD profile LOXL2 identified (from fibroblasts)  Effective in pre clinical models of fibrosis and cancer  Competitive profile: Excessive ‘cross-linking’ of Collagen fibres  Novel target and mechanism of action collagen fibres, stiffens  Once daily oral drug tissue, causing fibrosis  Complete inhibition of LOXL2 versus partial inhibition by antibody  Low cost of goods Gilead – LOXL2 antibody • Acquired Arresto program $225m pre phase 1 • Now in broad phase 2b trial program • Liver fibrosis; Idiopathic pulmonary fibrosis; Metastatic pancreatic cancer; Myelofibrosis; Solid tumours; Metastatic colorectal cancer 9

LOXL2 for pulmonary fibrosis collaboration with Synairgen Idiopathic Pulmonary Synairgen Fibrosis (IPF) collaboration  IPF primarily affects people over  Access to the age of 50  Synairgen’s strength in fibrosis biology and respiratory clinical  5,000 patients have IPF in development - BioBank human Australia tissue models technology  100,000 people with IPF in the platform US  expertise at University of  Prognosis is worse than that of Southampton many cancers  Faster time to value appreciation  Two drugs approved recently and partnering points of phase 1  Nintedanib (Boehringer or 2a Ingelheim)  Synairgen to fund pre clinical tox  Pirfenidone (Roche) and phase 1  Need for new therapies  Shares risk and reward based on  Current products expected to investment in program produce global revenues > $1.1  Allows PXS to pursue further billion by 2017 indications in parallel 10

Simtuzumab 1 versus PXS small molecule LOXL2 activity normalised α SMA DAB (IC 50 = 28 nM) [PXS 3 rd gen] (nM) PXS molecules are mechanism-based inhibitors with • higher potency • achieving complete inhibition Enzyme: R&D Systems recombinant human LOXL2 11 1. Simtuzmab: Gilead antibody for LOXL2.

CCl 4 model of liver fibrosis: Picro Sirius Red - fibrosis ** * *** * 50% change in Fibrotic Area (%) fibrotic area Week 4-6 ? effect size of AB0023 Therapeutic 3 of 6 weeks dosing Prophylactic 3 of 3 weeks dosing • PXS compound significantly reduced area of fibrosis in whole liver • Antibody effect measured on small area of liver and significant only in severe fibrosis. 12