Annual General Meeting

21 November 2019

Presentation by Chief Executive Officer

Gary Phillips

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. All statements, other than statements of historical facts, are 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. For example, despite our efforts there is no certainty that we will be successful in partnering our LOXL2 program or any of the other products in our pipeline on commercially acceptable terms, in a timely fashion or at all. 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.

Summary

A global leader in drug development for fibrosis & inflammation

1. Exciting product pipeline with multiple near term opportunities
- Lead drug (BI1467335) sold to Boehringer Ingelheim (BI) in 2015 in development for two disease indications
  - Total deal >$600m+ in development milestones plus royalties; $83m received to date from BI
- Anti fibrotic LOXL-2 program for the treatment of diseases including NASH and IPF completed phase 1 safety trials
  - Commercial partnering in progress
- Bronchitol and Aridol (Mannitol business) business unit nearing breakeven revenues
  - US FDA approval expected H1 2020
- Two further anti fibrotic programs in late stage pre clinical / phase 1
  - Patient proof of clinical efficacy trials due to start in 2020; myelofibrosis & skin scar revision
2. Management team with significant international experience in drug development, commercialisation and partnering
- Big Pharma validation of science and commercial acumen from existing deals with BI and Chiesi
3. Strong balance sheet A$23m cash (9/19), $6m R&D Tax Incentive received October 2019
4. Specialist US, UK and Australian institutional biotech investors on the share register
5. Numerous catalysts over next 12 months including two cash generating events (LOXL2 partnering & Bronchitol US)

Experienced senior management team

Significant experience in drug development, commercialisation and partnering

Gary Phillips – CEO

more than 30 years of operational management experience in the pharmaceutical and healthcare industry in Europe, Asia and Australia
joined Pharmaxis in 2003 and was appointed Chief Executive Officer in March 2013 at which time he was Chief Operating Officer
previously held country and regional management roles at Novartis – Hungary, Asia Pacific and Australia

David McGarvey – CFO

more than 30 years' experience building Australian based companies from inception to globally successful enterprises
joined Pharmaxis as Chief Financial Officer and Company Secretary in December 2002
previously Chief Financial Officer of the Filtration and Separations Division of US Filter (1998-2002), and Memtec Limited (1985-1998)
commenced career at PricewaterhouseCoopers

Brett Charlton - Medical

more than 25 years experience in clinical trial design and management
author of more than 80 scientific papers
founding Medical Director of the National Health Sciences Centre
previously held various positions with the Australian National University, Stanford University, the Baxter Centre for Medical Research, Royal Melbourne Hospital, and the Walter and Eliza Hall Institute

Wolfgang Jarolimek – Drug Discovery

more than 20 years' experience in pharmaceutical drug discovery and published more than 30 peer reviewed articles
previously Director of Assay Development and Compound Profiling at the GlaxoSmithKline Centre of Excellence in Drug Discovery in Verona, Italy
spent 8 years as post-doc at the Max-Plank Institute in Munich, Germany; Baylor College of Medicine, Houston, Texas; Rammelkamp Centre, Cleveland Ohio; and University of Heidelberg, Germany

Kristen Morgan – Alliance Management

more than 20 years' experience in the pharmaceutical industry having previously held a senior role in medical affairs at Sanofi-Aventis, and a commercial sales role at GlaxoSmithKline
responsibility for alliance management and medical and regulatory affairs

Non Executive Directors

Malcolm McComas Chair
- former investment banker
- former MD Citi Group

Kathleen Metters

former head of worldwide basic research at Merck
former CEO of biopharmaceutical company Lycera Corp
Will Delaat
- former CEO of Merck Australia
- former chair of Medicines Australia
Edward Rayner
- over 20 years' experience in global capital markets

A broad pipeline with multiple opportunities

	Indication	Discovery	LeadOptimisation	PhaseIII	Approval
Mannitol business
Bronchitol® US	Cysticfibrosis	FDA expected to complete review of NDA in Q1 2020. Subject to FDA approval, US partner Chiesi will launchcommercially in the US in 2020.
Bronchitol RoW	Cysticfibrosis	Bronchitol is currently sold in the UK, Germany, Italy, Greece & Nordic by Chiesi; in certain other European countriesand Russia by specialist distributors; and by PXS in Australia and smaller countries.		Direct &Dist
Aridol®	Asthmadiagnosis	Aridol is approved and sold in US, Australia, South Korea and a number of European countries. Canadian approvalreceived June 2019.		Direct &Dist
Drug development	Clinical
AOC3	NASH	Sold to Boehringer Ingelheim in May 2015. Phase 2a trial completed June 2019–to report Q4 CY 2019. PXS has received payments of A$68m to date.
AOC3	Diabeticretinopathy	received A$15m to date.	Boehringer commenced dosing a Phase 2a trial in January 2018. PXS
LOXL-2	NASH,fibrosis -liver,lung,kidney, heart	process commenced.	Phase 1 trials in 2 compounds complete. Commercial partnering
Systemic LOX	Anti-fibrotic:cancer		Completed phase 1a SAD. To complete phase 1b MAD Q1 2020		Progress in last 12 months
	Preclinical
Topical LOX	Anti-fibrotic:scarring	CY 2020	Effective in scarring models. Commence phase 1

AOC3: amine oxidase copper containing type 3 inhibitor; LOXL-2 (lysyl oxidase like 2) inhibitor (also inhibits LOX-3); LOX: pan (all) lysyl oxidase inhibitor; Systemic: oral drug; Topical: drug to be applied directly to the skin.

Highlights of the past year

2019 Progress Positions Pharmaxis for Pivotal 2020

New Drug Development

BI acquired drug
- NASH phase 2a study completed
- DR phase 2a > 50% recruited
LOXL2 program completes phase 1 and 3 month tox
System LOX program completes phase 1a, starts phase 1b; positive preclinical studies in myelofibrosis
Topical LOX prepares for phase 1

Mannitol Business

Bronchitol in-market sales increase in EU (17%) & Aust (12%)
FDA approves Frenchs Forest factory
Aridol relaunched in US, approved in Canada. Sales up 55%
Bronchitol reimbursed in Russia and commercial sales commence
FDA clarifies Bronchitol approval requirements

Corporate

$24m share placement
UK specialist biotech investor Arix joins share register
Ed Rayner joins board
R&D day presentations by Boehringer Ingelheim and the Garvan
Pharmaxis recognised as a top innovator company in Asia Pacific
Pharmaxis receives $6.2m R&D tax incentive

Boehringer Ingelheim deal

No further investment required from Pharmaxis
Commercial go/no go for phase 2b in NASH expected Q4 2019
Start of Phase 3 milestones ~A$100m

BI 1467335 in two "proof of clinical concept" Phase IIa trials

Phase IIa in NASH patients

ClinicalTrials.gov Identifier: NCT03166735

Safety, Tolerability, PD, and PK in four doses
Secondary efficacy endpoints
N=114 from Europe and North America
12 week treatment period compared to placebo in patients with clinical evidence of NASH.
Study completed June 2019
Study to report Q4 2019

BI phase 1 safety studies: 6 completed, 2 ongoing

Phase IIa in Diabetic Retinopathy patients

ClinicalTrials.gov Identifier: NCT03238963

Safety, Tolerability, PD, and PK with and without treatment
Secondary efficacy endpoints
N=100 from Europe and US
12 week treatment compared to placebo with a 12 week follow up period in patients with nonproliferative diabetic retinopathy
50% recruited
Estimated study completion May 2020

1

LOXL2 inhibition program in partnering process

for NASH, IPF & other high value fibrotic diseases

Potential indications / market size:

NASH / Liver Fibrosis; $35b1
Pulmonary fibrosis (IPF); $3.5b2
Kidney fibrosis
Cardiac fibrosis

LOXL2 and fibrosis
- LOX family of enzymes catalyse the final step in the fibrotic disease process
- Clear association of increased levels of serum LOXL2 with disease progression in IPF, NASH and cardiac fibrosis
Competitive profile
- Novel target and mechanism of action
- Once daily oral drug
- Best in class drug with high level inhibition of LOXL2 enzyme for 24 hours from one dose in phase 1 studies
- 13 week tox studies (2 species) for both compounds
- Only known drug in clinical development to also inhibit LOXL3
- Place of LOXL2 at the end of the fibrotic cascade provides opportunity to treat various fibrotic diseases and use in combination with other Pharma pipeline drugs

Systemic LOX inhibitor

Opportunities to fast track into patient proof of clinical efficacy studies

Program	Systemic LOX
Indication	Severe fibrotic indicationsmyelofibrosispancreatic cancer
Status	Phase 1a completedEffective in animal modelsofmyelofibrosis and otheracute fibrotic diseases2018 patent priority date
Next steps	Complete 3 month toxstudiesComplete Phase 1b(Multiple Ascending Dose)Commence phase 1c/2study in myelofibrosis andor pancreatic cancerpatients by H1 2020

Phase 1a - Single Ascending Dose

Dose dependent reduction in LOX activity in plasma

Good safety profile cleared to proceed to Multiple Ascending Dose stage
Significant 24 hour inhibition of LOX enzymes from single once a day dose

Myelofibrosis background

A rare type of bone marrow cancer that disrupts your body's normal production of blood cells

5k US patients diagnosed per year
16k total US patients
5 Years Median survival
US$1b+ market
Current treatments target JAK1/2 enzymes
- Improve constitutional symptoms (e.g. fevers, weight loss) reduce spleen size and bone pain
- Don't impact bone marrow histopathology and anaemia
Limited pipeline of drugs in development
- 2 nd generation JAK inhibitors
- Few anti fibrotic drugs
LOX an important target in myelofibrosis
- The role of the extracellular matrix and LOX in primary myelofibrosis highlighted in Blood Cancer Journal review article
- http://dx.doi.org/10.1038/bcj.2017.6

Topical LOX inhibitor

Opportunities to fast track into patient proof of clinical efficacy studies

Program	Topical LOX
Indication	ScarrevisionKeloid scarringBurns
Status	Limited competitionLead candidate selectedImprovesscar appearance andfunction in animal modelsStrong academic and clinicaladvocacyShort term tox studiescompletedsuccessfully
Next steps	Complete 3 month tox studiesReady to commence proof ofconcept study in patients 2020

Normal skin

Mannitol business – profitable from 2020

Driven by existing market growth plus market entry of Bronchitol into US

Two mannitol based products from Sydney factory; FDA, TGA, EU approved
- Aridol (Asthma Diagnostic)
- Bronchitol (Cystic Fibrosis)
Strong 2019 sales and healthy order book for both drugs
- Bronchitol EU FY 19 in-market sales +17%
- Bronchitol Australia FY 19 in-market sales +12%
- Aridol global sales FY 19 +55%
Increasing rate of profitability on growing sales as factory increases capacity utilisation

The US Market Tipping Point

FDA issued a Complete Response Letter (CRL) in June 2019
- Details all of the remaining matters to be addressed before Bronchitol® can be approved
- Main requirements in CRL are that Chiesi:
  - Revise the product packaging and user instructions
  - Conduct a human factor study (HFS) demonstrating healthcare professionals can properly administer the mannitol tolerance test.
Expected timing
- Design HFS
- FDA review of HFS
- Completion of HFS Q1 2020
- File HFS and other requested information with FDA – Q1 2020
- FDA completes review 60 days from filing
US sales commence in H2 CY 2020 and turn business cash flow positive.
Launch milestone US$10m in H1 CY 2020