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SYNTARA LIMITED Investor Presentation 2021

Apr 6, 2021

65830_rns_2021-04-06_bf8859a7-3819-4026-97ef-8f65a3b105ba.pdf

Investor Presentation

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Investor Presentation | April 2021
Gary Phillips CEO

developing breakthrough treatments for fibrosis and inflammation

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 developing or partnering any of the 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 forwardlooking statements as a result of new information, future events or otherwise.

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Novel, small molecule medicines focused on cancer and fibrotic disease

In House Discovery and Development capability

Experienced team delivering stream of novel drugs to the clinic

Platform technology drives pipeline of clinical assets

Multiple opportunities from global leadership in amine oxidase enzymes

Cash flow positive manufacturing business

FDA approval for Cystic Fibrosis drug transformative with Pharmaxis manufacturing business now cash flow positive

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Clinical stage
medicines
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Targeting Fibrosis
and Cancer
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Lead asset PXS-5505 in phase 2 trial

Breakthrough clinical program with disease modifying potential in Myelofibrosis

Broad potential for PXS-5505 in oncology

Global scientific and clinical collaborations to extend value of PXS5505 in further oncology indications

Anti scarring drug in phase 2 trial in 2021

PXS-6302 to enter patient studies in commercially important dermatology indications

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Experienced Scientific Leadership Team Significant global experience in drug development, commercialisation and partnering

In senior management

On the board

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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

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Gary Phillips – CEO and Managing Director

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

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Dieter Hamprecht – Head of Chemistry

  • more than 20 years experience with small molecule and peptide experience, contributed to greater than 10 drug candidates brought to development and co-inventor of 50 patent families, co-author of 30+ scientific publications

 previously Managing Director – Boehringer Ingelheim’s research group in Milan

  • senior medicinal chemistry positions at GSK

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Kathleen Metters – Non Executive Director

former Senior Vice President and Head of Worldwide Basic Research for Merck & Co. with oversight of all the company’s global research projects. in a subsequent role at Merck &Co she led work on External Discovery and Preclinical Sciences

former CEO of biopharmaceutical company Lycera Corp

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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

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Neil Graham – Non Executive Director

  • former VP of immunology and inflammation responsible for strategic program direction overseeing pipeline development and clinical programs at Regeneron (REGN:US)

  • former SVP program and portfolio management at Vertex Pharmaceuticals

  • former Chief Medical Officer at Trimeris Inc and Tibotec Pharmaceuticals

Read more on the Pharmaxis website

Drug development capability

Established team in Drug Discovery and Clinical Trials with broad experience across multiple regulatory agencies

2015-2020

Organisation

  • Leadership with extensive drug discovery/development experience from big pharma and biotech

  • Full In house capabilities o On site laboratories

  • Leveraged with international network of external contract organisations

  • Numerous collaborations with leading academic institutions in Australia and world-wide in pharmacology and medicinal chemistry

  • High scientific reputation through peer-reviewed publications

  • Direct management of Regulatory interaction with FDA, EMA, etc.

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7 Drugs
entered
pre-clinical
12
testing
publications
4 Drugs
entered
phase 1
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3 Drugs cleared phase 1 / IND ready

Strategy

  • Focus on inflammation and fibrosis/cancer driven diseases with high unmet medical need

  • Leverage global leading position in amine oxidase chemistry and biology

  • Create first / best in class small molecule inhibitors with biomarker assays for early validation of clinical hypothesis in phase 1 trials

  • Protect intellectual property by focused chemical matter, use and biomarker patents

  • Capture advantages of Australian location:

  • Accelerated (and lower cost) Phase 1 entry

  • Australian Government R&D tax credit system

2 Drugs in phase 2

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Multiple potential value inflection points over next two years Pipeline creates multiple opportunities

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Product Candidate 2021 2022 2023
Myelofibrosis Phase 1c Myelofibrosis Phase 2
PXS-5505
MDS pre-clinical MDS Phase 2
Pan-LOX Oncology
Hepatocellular Carcinoma Phase 2
Phase 1 Established / keloid scars Phase 2
PXS-6302
Pan-LOX topical scarring
Post surgical burns scarring Phase 2
PXS-4699 preclinical
Research
assessment by DMD TACT
committee
Phase 2 ready programs
Evaluating grant and partnering
SSAO; PXS-4728
options
LOXL2; PXS-5382
Value inflection point Grant pending
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DMD: Duchenne Muscular Dystrophy

MDS: Myelodysplastic Syndrome

Pipeline opportunities
in fibrosis and inflammation

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PXS-5505 Breakthrough clinical program in
myelofibrosis prioritised into phase 2

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First in class PXS-5505 IND approved and in the clinic

Novel anti fibrotic approach with broad applications in difficult to treat cancers

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Myelofibrosis: Orphan Disease with high unmet need forecast to exceed US$1b

  • Drug with disease modifying potential patented 2018

  • Long term tox and phase 1 studies completed 1H 2020

  • FDA orphan status granted July 2020

  • IND approved August 2020

  • Phase 1/2a proof of concept myelofibrosis study commenced recruitment Q1 21

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Adjunct to best standard of care in multiple cancers

  • Pan-LOX inhibition synergistic with current standard of care and pharma development pipeline in many stromal cancers

  • Academic and clinical interest in additional indications including;

  • Myeloproliferative disorders (e.g. MDS)

  • liver carcinoma (HCC)

  • glioblastoma

  • International studies facilitated by IND approval and availability of drug product

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Myelofibrosis background

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

KEY FACTS

Primary Myelofibrosis is caused by a build up of scar tissue (fibrosis) in bone marrow reducing the production of blood cells:

  • Driven by clonal mutations of a hematopoietic stem cell (JAK, MPL, CALR genes)

Affects 15 in 1m people worldwide

  • Reduced red blood cells can cause extreme tiredness (fatigue) or shortness of breath

  • Reduced white blood cells can lead to an increased number of infections

  • 5 Years Median survival

  • Reduced platelets can promote bleeding and/or bruising

  • Spleen increases blood cell production and becomes enlarged

Age of onset 50 – 80

  • Other common symptoms include fever, night sweats, and bone pain

Standard of Care; JAK inhibition

11% transformation to leukemia

  • Symptomatic relief plus some limited survival improvement. 75% discontinuation at 5 years

  • Median overall survival is 14 – 16 months after discontinuation

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Mode of action in MF

Disease modifying potential as monotherapy and on top of standard of care

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Unique mechanism of action
targeting the extracellular matrix
Disease modifying
potential
Efficacy on top of
existing standard of care -
AND development pipeline drugs
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“Specific targeting of ECM dysregulation to prevent and diminish MF may prove the frontline of research and therapy development in PMF with the greatest promise of relieving symptoms and extending life expectancy of patients” Blood Cancer Journal (2017) 7, e525; doi:10.1038/bcj.2017.6

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PXS-5505; Pan-LOX inhibitor with promising profile Pre clinical and clinical studies strongly support entry into patient studies

PXS-5505 attenuates hallmarks of primary myelofibrosis in mice.

PXS-5505 – Phase 1 SAD

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Treatment with
PXS-5505
significantly
reduced reticulin
fibrosis
( = p<0.001)
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“JAK inhibition alone is insufficient in the treatment of patients with myelofibrosis; it is not associated with changes in underlying disease biology and it can worsen blood counts, leading to high drug discontinuation rates over time. The trial utilizing PX-5505 is supported by a sound scientific rationale, based on pre-clinical work demonstrating the importance of lysyl oxidase in the development of myelofibrosis. PXS-5505 has a unique mechanism of action that has the potential for disease modification. I am looking forward to seeing the effect of this drug in clinical trials.”[1]

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  • Good safety profile with 6 month tox studies complete

  • Dose dependant 24 hour inhibition of LOX enzymes from single once a day dose in humans

  • No safety signal seen in phase 1 trials

  • 2018 priority patent date

Ref Graph1: Leiva et al. Intl J Hemat 2019. https://doi.org/10.1007/s12185-019-02751-6

PXS-5505 Phase 1/2a Trial in myelofibrosis

6 month monotherapy study with meaningful safety and efficacy endpoints

STUDY POPULATION

DESIGN

JAK-inhibitor unsuitable* Phase 1/2a open primary MF or post-ET/PV label study to MF patients with: evaluate safety, PK/PD, and efficacy

  • INT-2 or High risk MF requiring therapy

  • Symptomatic

  • BMF Grade 2 or greater

TREATMENT COHORT

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Dose escalation: PXS-5505 3 ascending doses, 4 weeks (n = 3 to 6 subjects/dose)

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Cohort expansion: PXS-5505 (n = 24 subjects) 26 weeks

ENDPOINTS

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Primary: Safety TEAEs

Secondary: PK/PD BMF Grade IWG Response SVR Haematology Symptom score

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FDA granted orphan drug designation July 20 and IND approved August 2020

Multiple sites across 3 countries to enhance trial recruitment (USA, Korea, Australia)

Study budget (~US$5m)

Study recruitment commenced Q1 2021, study concludes H2 2022

*Unsuitable = ineligible for JAKi treatment, intolerant of JAKi treatment, relapsed during JAKi treatment, or refractory to JAKi treatment. JAKi – Janus Kinase inhibitor, MF myelofibrosis, ET Essential Thrombocythaemia, PV polycythaemia vera, INT intermediate,

BMF bone marrow fibrosis, RP2D recommended phase 2 dose, TEAE treatment emergent adverse event, PK pharmacokinetics, PD pharmacodynamics, SVR spleen volume response, IWG International Working Group Myeloproliferative Neoplasms

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Myelofibrosis – other programs

PXS-5505 unique mechanism of action promises disease modification and good tolerability

Company Market cap(1) Bourse Asset Description Clinical phase
$1.2bn Nasdaq KER-050 TGF-β ligand trap Phase 2
$1.1bn Nasdaq CPI-0610 BET inhibitor Phase 3
$0.7bn(2) n.a. – private KRT-232 MDM2 antagonist Phase 3
$0.5bn Nasdaq Imetelstat Telomerase inhibitor Phase 3
$27m (A$35m) ASX PXS-5505 Pan-LOX inhibitor Phase 1c/2
commenced
Existing pipeline in
development all have
challenging safety /
side effect profiles
PXS-5505 unique
mechanism of action with
expected good efficacy
AND tolerability
PXS-5505 mechanism of action expected to
deliver additional efficacy on top of existing
standard of care and/or known pipeline
drugs without adding to tolerability issues

Notes: (1) Market cap as at 1[st] October 2020

Pipeline opportunities
in fibrosis and inflammation

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PXS-5505 Significant opportunity in other cancers;
-
 Myelodysplastic Syndrome
-
 Hepatocellular Carcinoma

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PXS-5505: Significant opportunity in other cancers

Global academic and clinical interest in LOX inhibition drives development plan

Normal tissue

Pharmaxis Research Collaborations

Other Myeloproliferative Disorders; e.g.

Myelodysplastic syndrome

Germany

Liver Cancer

Rochester (NY)

Pancreatic Cancer

Sydney, Rochester (NY)

Melanoma and glioblastoma Houston

Head and Neck Cancer

Boston

Mesothelioma

Dublin

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Collagen
Tumour with fibrotic tissue has
• increased tissue stiffness
• Increased interstitial pressure
Increased Increased
EMT angiogenesis
Increased Decreased
Increased
Invasion drug perfusion
tumour growth
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Multiple benefits from anti-fibrotic mechanism of action

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EMT: epithelial to mesenchymal transition 15
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Myelodysplastic Syndrome (MDS)

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

KEY FACTS

 A group of malignant hematopoietic neoplasms characterized by Bone marrow failure with resultant cytopenia and related complications

 Current standard of care

Affects ~40 in 100,000 people > 70 years

  • Allogeneic stem cell transplantation

  • Immunomodulatory drug lenalidomide,

  • Advanced disease: DNA hypomethylating agents (HMA), azacitidine (AZA), and decitabine

50% to 60% of the patients will die from complications of the disease

New US diagnoses per annum ~50,000

30% transformation to Acute Myeloid Leukemia

 Pre clinical evidence

  • Unpublished data from Pharmaxis scientific collaborations demonstrating strong proof of concept

  • – Treatment with PXS-5505 and AZA significantly increases red cell count

 Clinical strategy

  • 6 month open label study in patients non responsive to AZA / current standard of care

  • Multi centre approach to follow on from myelofibrosis study

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Hepatocellular Carcinoma (HCC)

4th leading cause of cancer-related mortality worldwide with a 19.6% 5-year relative survival

  • Primary liver malignancies have doubled in incidence over the last two decades.

 HCC is a stromal (fibrotic) tumour

  • Accumulation of collagen crosslinks increases stromal stiffening and interstitial fluid pressure (IFP) reducing delivery of chemotherapy and immunotherapy.

 Etiology

  • Extrinsic factors e.g. Virus infections

  • Intrinsic factors e.g. auto immune diseases, fatty infiltration, genetics

 Current standard of care

  • Tyrosine kinase inhibitors

  • PD-L1 inhibitors + anti-VEGF

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 Pre-clinical data

  • High LOX expression associated with reduced survival

  • LOX is up-stream regulator of VEGF expression and inhibition of this enzyme could potentiate the intratumoral effects of anti-VEGF therapy

  • Combination anti-PD-1 therapy with LOX inhibition has demonstrated synergistic decrease in tumor growth

 Clinical strategy

Enhance the intratumoral response to VEGF therapy and anti-PD-L1 blockade through the addition of LOX inhibition in human HCC

  • Open label 6 month study combination PXS-5505 with Atezolizumab and Bevacizumab compared to historical standard of care in newly diagnosed unresectable or metastatic hepatocellular carcinoma

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Pipeline opportunities
in fibrosis and inflammation

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PXS-6302 Topical Pan-LOX Inhibition for
scarring

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Hypertrophic and keloid scarring

Cutaneous scarring following skin trauma or a wound is a major cause of morbidity and disfigurement

KEY FACTS

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Collagen turnover in keloid
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  • Mechanisms underlying scar formation are not well established, and prophylactic and treatment strategies remain unsatisfactory

 Current standard of care

100m patients develop scars in the developed world alone each year as a result of elective operations and operations after trauma

Hypertrophic scars and keloids are fibroproliferative disorders that may arise after any deep cutaneous injury caused by trauma, burns, surgery, etc.

Hypertrophic scars and keloids are cosmetically and functionally problematic significantly affecting patients’ quality of life

The increase in extracellular matrix is a key factor and this depends on collagen and elastin cross-linking to make them less degradable.

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  • Corticosteroids

  • Surgical revision

  • Cryotherapy

  • Laser therapy

  • 5-fluorouracil

 Pre clinical evidence

  • Unpublished data from Pharmaxis scientific collaborations demonstrating strong proof of concept

  • – Treatment with PXS-6302 monotherapy and tool compound demonstrates cosmetic and functional improvements to the scar

 Clinical strategy

  • 3 month placebo controlled study in patients versus current standard of care.

  • Initial patient groups will either have established scars or scarring subsequent to burn injury.

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Further non core pipeline opportunities in fibrosis and inflammation Leveraging global leadership position in amine oxidase enzymes to deliver targeted drugs for cancer and fibrosis

Product Candidate
Indications
Pre-
clinical
Product Candidate
Indications
Pre-
clinical
Product Candidate
Indications
Pre-
clinical
Phase 1 Phase 2
Next Steps
Phase 2
Next Steps
SSAO; PXS-4728 Repurposed for neuro
inflammatory disease

Partnering discussions; phase 2
protocol and funding discussions with
independent investigators
LOXL-2; PXS-5382 Chronic fibrotic disease
e.g. chronic kidney disease,
idiopathic pulmonary fibrosis

Partnering discussions; phase 2
protocol and funding discussions with
independent investigators
SSAO/MAOB; PXS-4699 Anti inflammatory
Muscular Dystrophy

$1m matched funding grant

DMD TACT committee Q2 2021

Advance to the clinic H1 2022
SSAO/MPO; PXS-5370 Anti inflammatory
Multiple indications

Grant identification in process

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IIS: Independent Investigator Studies 20
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Mannitol respiratory business (Bronchitol® and Aridol®)

Transformational impact of FDA Bronchitol approval (Oct 2020) - cash flow positive from FY 2021 onwards

Sales

  • Mannitol respiratory sales forecast to double by FY 2022 with Bronchitol > 75% of sales

  • Strong longer term growth contribution from US

  • Growth in Ex-US markets including Russia

Expenses

  • Relatively fixed production cost base

  • Potential for simplified business

  • model to reduce costs

EBITDA

  • Positive EBITDA from FY 2021 onwards (before potential cost savings).

  • US volumes enable mannitol segment to generate profit

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Bronchitol in US

  • US CF market >65% of global market

US market doubles global cystic fibrosis patient opportunity with attractive pricing

  • Chiesi approval /launch milestone payments US$10m received FY 2021

  • US sales commence in Q2 CY 2021

  • High teens % of Chiesi sales + long term supply contract - ~20% of Chiesi US Bronchitol net sales flow directly to the Pharmaxis bottom line

  • Three sales milestones totaling US$15m payable on achieving annual sales thresholds

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21
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Strengthened cash position

Further opportunities to extend cash runway ahead

  • Dec 20 proforma cash balance of A$22m

  • Cash Dec 2020: A$18m (includes $4m Chiesi milestone received March 2021)

  • US Bronchitol sales commence in Q2 CY 2021

  • High teens % of Chiesi sales + long term supply contract - ~20% of Chiesi US Bronchitol net sales flow directly to the Pharmaxis bottom line

  • Mannitol business to go from cash burn (FY 20: A$4m) to cash flow positive from FY 21 onwards (FY 26: A$10m+)

Year 2019 2020 From 2021 E
EBITDA
(A$m)*
($5.0) ($4.0) Cash Flow
Positive
  • Further opportunities to extend cash runway

  • Mannitol potential cost savings

  • Distribution license fees from additional Aridol and Bronchitol territories

  • Pipeline supported by grants and R&D tax credit (~A$5m 2020)

  • Partnering deals with pipeline assets

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Proforma Cash Usage [1] A$m
20
18
16 ($5m)
14 Mannitol makes a
($4m)
positive cash
12
contribution from
10
FY 21 onwards
8
6
4
2
0
2019 2020 Future
Other Mannitol
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  1. Proforma cash usage is the total of segment EBITDA (mannitol business, new drug discovery and corporate), finance lease payments, capex and financing agreement payments. Refer financial slides for further detail.

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Mannitol segment EBITDA only 22
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Anticipated news flow: 2021 – 2022 Multiple value inflection points over next two years

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H1 2021

  • Feb 22: Breakthrough drug PXS-5505 phase 1c/2a myelofibrosis study commenced recruitment

  • Mar 19: Chiesi pays US$3m milestone on Pharmaxis shipment of US launch

  • Mar 31: LOX topical drug PXS-6302 commenced independent investigator studies - safety

  • Mannitol business simplification completed – realising annual cost savings

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H2 2021

CY 2022

  • PXS-5505 phase 1c/2a myelofibrosis study dose expansion stage commence

  • PXS-5505 phase 2a myelofibrosis study safety and efficacy data

  • First collaborations to progress PXS-5505 into clinical trials in other myeloproliferative diseases and/or cancer indications

  • PXS-5505 dose escalation data in myelodysplastic syndrome

  • PXS-5505 dose escalation data in hepatocellular carcinoma

  • Ongoing cash receipts from supply of Bronchitol for US sales

  • LOX topical drug phase 2 studies burns and established scars safety and efficacy data

  • LOX topical drug progresses into independent investigator patient studies - burns and established scars

  • Feedback from global advisory committee on development fast tracking for Duchenne muscular dystrophy clinical trials.

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Appendices

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Experienced senior management team

Significant experience in drug development, commercialisation and partnering

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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

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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

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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

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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

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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

Non Executive Directors

Malcolm McComas Kathleen Metters Will Delaat Neil GrahamChair, former former head of former CEO of Merck former strategic investment banker; worldwide basic Australia; former chair program director at former MD Citi Group research at Merck; of Medicines Australia Regeneron Inc; former CEO of extensive career in biopharmaceutical pipeline development company Lycera Corp and clinical development

Read more on the Pharmaxis website

Board

Significant international pharmaceutical experience

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Malcolm McComas – Chair

  • former investment banker and commercial lawyer

  • former MD Citi Group

  • has worked with many high growth companies across various industry sectors and has experience in equity and debt finance, acquisitions and divestments and privatisations.

  • joined Pharmaxis Board in 2003

  • chair since 2012

Will Delaat – Non-Executive Director

  • more than 35 years’ experience in the global pharmaceutical industry

  • former CEO of Merck Australia

  • former chair of Medicines Australia and Pharmaceuticals Industry Council

  • joined Pharmaxis Board in 2008

Dr Kathleen Metters – Non-Executive Director

  • former Senior Vice President and Head of Worldwide Basic Research for Merck & Co. with oversight of all the company’s global research projects.

  • in a subsequent role at Merck &Co she led work on External Discovery and Preclinical Sciences

  • former CEO of biopharmaceutical company Lycera Corp

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Gary Phillips – Chief Executive Officer

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

Dr Neil Graham – Non-Executive Director

former VP of immunology and inflammation responsible for strategic program direction overseeing pipeline development and clinical programs at Regeneron (REGN:US)

former SVP program and portfolio management at Vertex Pharmaceuticals former Chief Medical Officer at Trimeris Inc and Tibotec Pharmaceuticals

Read more on the Pharmaxis website

Financials

Cash

Periods ended (A$’000) Dec 2020
HY
Dec 2019
HY
Jun 2020
FY
Jun 2019
FY
Proforma cash
Cashperiod end 18,249 25,864 14,764 31,124
R&D tax credit - - 5,048 5,962
Chiesi milestonepayments ~4,0001 - ~14,000 -
~$22,249 $25,864 ~$33,812 $37,086
Cash Flow Statement Highlights
Operations
Receipts from customers 3,602 3,973 7,775 6,893
R&D tax incentive 5,099 6,221 6,271 -
Chiesi milestone 9,949 - - -
Payments to suppliers,employees etc (13,602) (13,886) (27,330) (26,691)
Total operations 5,048 (3,692) (13,284) (19,798)
Investing (capex) (281) (328) (574) (981)
Finance leasepayments2 (1,147) (1,111) (2,232) (1,593)
Financingagreementpayments3 (135) (129) (270) (254)
Share issue - net - - - 22,677
Net increase(decrease)in cash $3,485 ($5,260) ($16,360) $51
  1. US$3m milestone earned February 2021

  2. Lease over 20 Rodborough Rd (to 2024) – total liability at 31 December 2020: $7.1 million

  3. NovaQuest financing – not repayable other than as % of US & EU Bronchitol revenue – up to 7 years

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Refer to Quarterly Shareholder Updates, 2020 Annual Report and December 2020 Half Year Report for complete financial information 27
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Financials

Income statement highlights

Periods ended (A$’000) Dec 2020
HY
Dec 2019
HY
Jun 2020
FY
Jun 2019
FY
Segment Financials
New drug development
Oral Pan-LOX (external costs) (1,323) (1,400) (3,124) (3,833)
Otherprogram external costs(net ofgrants) (775) (1,078) (3,315) (5,108)
Employee costs (1,799) (1,529) (3,373) (2,837)
Overhead (238) (281) (460) (606)
R&D tax credit 148 259 5,159 5,962
EBITDA (3,987) (4,029) (5,113) (6,764)
Mannitol respiratory business
Sales 3,086 3,259 7,027 5,676
Other income 10,098 10 20 27
13,184 3,269 7,047 5,703
Expenses – employee costs (2,914) (3,037) (5,855) (6,083)
Expenses– manufacturing purchases (1,172) (746) (1,456) (1,689)
Expenses – other (2,374) (1,755) (3,713) (2,944)
EBITDA 6,724 (2,269) (3,977) (5,013)
Corporate – EBITDA (2,024) (1,701) (2,990) (3,874)
Total Adjusted EBITDA 713 (7,999) (12,080) (15,651)
Net profit(loss) $46 ($10,319) ($13,943) ($20,058)

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Refer to Quarterly Shareholder Updates, 2020 Annual Report and December 2020 Half Year Report for complete financial information 28
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Shareholders & trading

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Financial Information Institutional Ownership 31 Dec 20
ASX Code PXS
BVF Partners (US) 19%
Market Cap1 A$32m
Shares on Issue1 397m
D&A Income Limited 7%
Employee Options1 19m
Liquidity (turnover last 12 months)1 369m shares Other Institutions 8%
Share price1 A$0.081
Proforma cash balance (31 December 2020) A$22m Total Institutional Ownership 34%

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1. As at 31 March 2021
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developing breakthrough treatments for fibrosis and inflammation www.pharmaxis.com.au

Contacts
Gary Phillips
Chief Executive Officer
[email protected]
David McGarvey
Chief Financial Officer
[email protected]