Investor Presentation Australia Biotech Invest Melbourne: 6 - PowerPoint PPT Presentation

innovating for life Investor Presentation Australia Biotech Invest Melbourne: 6 October 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 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 already creating value Drug developer Management Drug manufacturer Financial strength  Leading position in  Management team  Supplies Bronchitol  $54m cash balance amine oxidase and Board with to global markets at June 2015 chemistry and global experience via experienced  Significant value mechanism based commercial partners  Extensive pharma milestones from inhibitors  Financial risks industry network existing partner  Proven capability in shared deals within reach  Proven capability of delivering quality  Financial upside  Growing institutional executing global BD programs to achieve transactions with from accessing new presence on share phase 2 ready major partners markets – US, register - >45% compounds Russia  Preclinical, early  Exciting pipeline of  Possibility to further and late phase drug candidates for clinical experience rationalise valuable targets manufacturing 3 infrastructure

Our therapeutic focus the inhibition of amine oxidase enzymes has broad potential application 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 4

Pharmaxis drug discovery strategy Building a biotech powerhouse in fibrosis and inflammation Strategy Achievements to date Drug discovery: Drug discovery:  Build a biotech powerhouse in fibrosis and  First in class NASH drug taken to phase 1  Two further candidates in lead optimisation inflammation  Prioritise validated targets phase  Multiple drugs from in-house amine  One lead candidate moving to preclinical oxidase chemistry platform  Develop to phase 1 or 2 Partnering: Partnering:  In house BD expertise achieves valuable  Create value via deal with Boehringer Ingelheim - A$39m  Licence out to Big Pharma with attractive upfront, total potential > A$750m 1 st in class drugs post phase 1 or 2  Collaborate to de-risk and accelerate PXS  Collaboration with Synairgen Research plc programs for early stage fibrosis program to widen  Collaborate on in-licensing programs spread of indications, enhance time to value inflection and spread risk 5

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  Development status: disease  Pharmaxis discovery – patent filed  Industry leading development times 2012  Boehringer responsible for all  Effective in pre clinical models of NASH development, and commercialisation and airway inflammation activities  Phase 1 study reported  Competitive deal: • orally bioavailable  Total potential payments to approval for • long lasting inhibition after single 2 indications: €418.5m (~A$600m), dose • acquisition (May 2015): €27.5m • progressive dose response (~A$39m)  Competitors: • commencement of phase 2 and 3: up to total €55m (~A$80m)  Genefit – GF505 in Phase 2b NASH • filing, regulatory & pricing approvals:  Intercept - OCA (FXR agonist) in up to total €140m(~A$200m) Phase 2b NASH • second indication: additional total  Gilead – FXR agonist in pre clinical milestone payments (€195m)  Earn-out payments on annual net sales • tiered percentages starting in high single digits • plus potential sales milestones  External validation of PXS drug discovery and ability to negotiate valuable global deals 6

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  Development status: Fibroblast cells in 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:  Novel target and mechanism of action Excessive ‘cross-linking’ of Collagen fibres 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 7

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  5,000 patients have IPF in biology and respiratory clinical 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  Two drugs approved recently appreciation and partnering  Nintedanib (Boehringer points of phase 1 or 2a Ingelheim)  Synairgen to fund pre clinical  Pirfenidone (Roche) tox and phase 1  Need for new therapies  Shares risk and reward based  Current products expected to on investment in program produce global revenues > $1.1  Allows PXS to pursue further billion by 2017 indications in parallel 8

Pharmaxis opportunities Building a biotech powerhouse in fibrosis and inflammation SSAO program for LOXL2 program for LOXL2 for NASH Bronchitol FOR pulmonary fibrosis and other diseases CF in US NASH (fatty liver)  NASH: US$3.5B  Pulmonary fibrosis:  Big pharma interest  US is largest CF market by 2025 market >$1B in NASH, LOXL2 market and PXS approach  PXS SSAO inhibitor  Collaborate to  Partnered - Chiesi  Complimentary to of NASH phase 1 or 2 then  Chiesi funding successfully taken seek partner SSAO program CF303 to a cap of to phase 1 acquired by BI  Revenue share for US$22m  Acquired by BI for  Next milestone – phase 1 partnering  $25m milestone A$39m upfront, total deal: 50/50 commencement of payments on launch >A$750m formal preclinical  Next milestone – and sales program ~  BI to develop for commencement of thresholds beginning CY 2016 NASH and other formal preclinical  High mid teens inflammatory program ~ royalty% on in- indications (eg. beginning CY 2016 market sales kidney fibrosis,  Mid teens % uplift COPD) on COGs  Next milestone: start of phase 2 ~end CY 2016 9

Major upcoming milestones Cash funds ($54m at 30 June) sufficient to reach near term valuable milestones Calendar years 2016 2017 2015  PXS4728A Phase 2 commences  CF303 fully  CF303 – last patient  FDA decision on recruited completes trial Bronchitol approval in US  CF204 (paediatric)  CF303 – reports  Bronchitol US reports launch  Lead candidate for  Complete pre  Commence phase 1 ad IPF identified clinical program  Partner asset  Lead LOXL2  Complete pre  Commence phase 1 Drug discovery candidate identified clinical program  Partner Asset for NASH / Liver fibrosis  SSAO/MAOB  Complete pre  Commence phase 1 disease indication clinical program  Partner Asset nominated 10