Percheron Therapeutics Limited Corporate Presentation
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Percheron Therapeutics Limited
ABN 41 095 060 745
ASX: PER | FSE: AWY | US OTC: ATHJY
PERCHERON THERAPEUTICS CORPORATE PRESENTATION
Melbourne, Australia – 1 September 2026: Percheron Therapeutics Limited (ASX: PER)
(‘the Company’), an international biotechnology company focused on the development
of novel therapies for oncology and rare diseases, is pleased to provide shareholders
with a copy of an updated non-confidential corporate presentation providing further
information on the recently announced investigator sponsored clinical trial to be
conducted by Vanderbilt Health.
~ ENDS ~
About Percheron Therapeutics Limited
Percheron Therapeutics Limited [ASX: PER | US OTC: PERCF] is a publicly listed biotechnology company
focused on the development and commercialisation of novel therapies for oncology and rare diseases. The
company’s lead program is HMBD-002, a monoclonal antibody targeting the immune checkpoint regulator,
VISTA. HMBD-002 has completed a phase I clinical trial in patients with advanced cancer, which has shown
the drug to be generally safe and well-tolerated, and Percheron aims to commence further clinical trials in
CY2026.
For more information, please contact info@PercheronTx.com.
This announcement has been authorized for release to the Australian Securities Exchange
by the Board of Directors.
www.PercheronTx.com ASX: PER | US OTC: PERCF
Advancing a Next Generation Immune
Checkpoint Inhibitor into Blood Cancers
Investor Update
September 2026
1
Forward-Looking Statements
This presentation contains forward-looking statements within the meaning of the safe-harbor provisions such as the Private Securities Litigation Reform Act of 1995. These
statements do not relate strictly to historical or current facts and may be accompanied by words such as ‘could,’ ‘would,’ ‘may,’ ‘potentially,’ ‘suggest,’ ‘believes,’ ‘expects,’
‘should,’ ‘intends,’ ‘plans,’ ‘forecasts,’ and similar words or expressions.
Such statements involve substantial risks and uncertainties, not all of which may be known at the time. All statements contained in this presentation, other than statements of
historical fact, including without limitation statements regarding our strategy, research and development plans, collaborations, future operations, future financial position, future
revenues, projected costs, pricing, prospects, plans, and objectives of management, are forward-looking statements. Not all forward-looking statements in this presentation are
explicitly identified as such.
The Company does not warrant any of the forward-looking statements in this presentation, and investors are advised to interpret such statements in the context of other
available sources of information and with the assistance of expert advisors as appropriate.
Many factors could cause the actual results of the Company to differ materially from the results expressed or implied herein, and you should not place undue reliance on the
forward-looking statements. Drug development is inherently risky, and only a small proportion of research and development programs lead to a marketed product. Factors which
could change the Company’s expected outcomes include, without limitation, our ability to: advance the development of our programs, and to do so within any timelines that may
be indicated herein; the safety and efficacy of our drug development candidates; our ability to replicate experimental data; the ongoing validity of patents covering our drug
development candidates, and our freedom to operate under third party intellectual property; our ability to obtain necessary regulatory approvals; our ability to enter into and
maintain partnerships, collaborations, and other business relationships necessary to the progression of our drug development candidates; changes in the competitive landscape
pertaining to our drug development candidates; the timely availability of necessary capital to pursue our business objectives; changes in the public policy environment in one or
more countries in which we operate or may seek to operate which disfavour our business; our ability to attract and retain qualified personnel; changes from anticipated levels of
customer acceptance of existing and new products and services; and other factors.
Although the Company believes that the expectations reflected in such forward-looking statements are reasonable, and although they reflect our current views as at the date of
this presentation, there can therefore be no assurance that such expectations will prove to be correct. The Company has no obligation as a result of this presentation to pursue
any specific strategy or plan outlined herein, or to deliver any specific outcome that may be implied or inferred.
Any forward-looking statements contained in this presentation speak only as of the date this presentation is made, and we expressly disclaim any obligation to update any
forward-looking statements, whether because of new information, future events or otherwise, except as may be required by law.
2
Executive Summary
• Percheron has entered into an agreement with Vanderbilt Health (VH) to conduct a multi-centre clinical trial of HMBD-002 in
patients with acute myeloid leukaemia (AML)
• AML is the most common form of leukaemia, with about 20,000 patients diagnosed annually in the United States
• Existing therapies are initially effective in a majority of patients, but >50% will relapse, and long-term outcomes remain challenging
• VH study is an investigator-sponsored trial (IST), with Percheron’s obligations limited to a financial grant, study drug, and advisory support
• Following a $2.3 million capital raise in August 2026, Percheron’s commitments associated with the AML study are fully funded
• In preparation for new clinical trial work, the Company has successfully completed drug substance manufacture, with new clinical
material expected to be available for shipping to sites in 2H CY2026
• Dr Michael Baker has been appointed CEO and Managing Director, commencing on 5 October 2026
• Dr James Garner has stepped down as CEO but will remain a non-executive director to provide continuity and support
• World Health Organisation has selected ‘minperstobart’ as the provisional international non-proprietary name (pINN) for HMBD-
002, with confirmation during CY2027
Percheron is developing HMBD-002, a novel, mid-clinical-stage, immuno-oncology
therapy with applicability to multiple forms of cancer and high combination potential
3
THE SCIENCE THE COMPANY THE OPPORTUNITY
Percheron is developing HMBD-002,
a next generation immune checkpoint inhibitor,
with the potential to treat many forms of cancer
Percheron is listed on the ASX (ASX: PER),
with a market cap of ~$8 million,
and cash at 30JUN26 of ~$4.1 million
With phase 1 successfully completed in CY2025,
Percheron aims to take HMBD-002 into
an international phase 2 human trial in CY2026
HMBD-002 targets VISTA, a novel immune checkpoint,
similar in many respects to PD-1, the target of Keytruda®
(pembrolizumab)
Previous attempts to drug VISTA have been limited by
toxicity; HMBD-002 is built on a different antibody scaffold,
substantially reducing potential toxicity
HMBD-002 has potential as a monotherapy, but also
appears synergistic with other checkpoint inhibitors such as
Keytruda®
The company trades at a substantial discount to ASX-listed
peers, following a corporate transformation during CY2025
Enterprise value largely reflects acquisition cost of
HMBD-002 (~$4.5 million), currently imputing no value to
future positive data
Percheron’s cash reserves and low burn imply 3.9 quarters
of funding, per June 2026 Appendix 4C filing
US-based phase 1 study, conducted under IND with US FDA,
showed very favourable safety profile, both as monotherapy
and in combination with Keytruda® (pembrolizumab)
Adaptive, modular phase 2 study design can be launched
with very modest cash resources and is designed to provide
early validating read-outs
Keytruda® patent expiry in 2028 creates substantial
opportunity for novel immune checkpoint inhibitors to
expand and extend US$ ~100 billion market
Immune checkpoint inhibitors such as HMBD-002 work by reactivating the body’s
immune response so that it can attack the tumour
4
Cancer
cells
Immune
cells
• Ordinarily, our immune system
protects us against tumour cells in the
same way that it protects against
bacteria and viruses
• Growing tumours need to inhibit the
patient’s immune system to avoid
being attacked by normal defences
• One of the ways tumours accomplish
this is via ‘immune checkpoints’ such
as PD-1 (the target of Keytruda®) and
VISTA (the target of HMBD-002)
HMBD-002
• Immune checkpoint inhibitors such as
HMBD-002 block these inhibitory
signals on the surface of the cancer
cell
• A number of such immune checkpoint
inhibitor drugs have been approved by
FDA in recent years, including
Keytruda®, Opdivo®, Tecentriq®,
Yervoy® and others
• There are not yet any commercial
VISTA inhibitors, partly because the
target is relatively newly discovered
• Removal of the inhibitory signals
allows the body’s own immune system
to attack the cancer cells
• The reinvigorated immune system can
be potent in its own right, but can also
work synergistically with other cancer
treatments such as chemotherapy and
radiotherapy
• In some cancers, inhibiting multiple
immune checkpoints is more effective
than just one (e.g. PD-1 and CTLA-4 in
melanoma)
• In addition to making the cancer cells
vulnerable to the immune system,
VISTA inhibition helps to attract more
immune cells into the ‘tumour
microenvironment,’ potentially
amplifying its effects by turning ‘cold
tumours’ into ‘hot tumours’
• In particular, VISTA inhibition appears
to attract myeloid cells, which are less
susceptible to other immune
checkpoint inhibitors
HMBD-002 is arguably the most advanced VISTA antibody in clinical development, and
likely the only active member of the IgG4 class
5
Program HMBD-002 CI-8993 JNJ-61610588 SNS-101 W0180
Company
Isotype IgG4 IgG1 IgG1 IgG1 IgG1
Stage Phase I Complete Phase I Complete Phase I
Terminated
Phase I
Terminated
Phase I
Terminated
Exposure to Date 48 patients 26 patients 12 patients - 33 patients
Combination
Data Available
Monotherapy &
Combination with
Pembrolizumab
Monotherapy Monotherapy Monotherapy &
Combination with
Cemiplimab
Monotherapy &
Combination with
Pembrolizumab
Status Ongoing Inactive Discontinued Discontinued Discontinued
Notes pH-sensitive
antibody
INDICATIVE
Sources: Company filings; Clinicaltrials.gov
In general, IgG1 antibodies activate
antibody-dependent cellular
cytotoxicity (ADCC), recruiting immune
cells such as natural killer (NK) cells to
destroy the target.
This can be a very effective approach
in cancer, but can also lead to
significant toxicity, including cytokine
release syndrome (CRS).
Expert feedback indicates that the key
challenge with other VISTA antibodies,
which are almost exclusively IgG1, is
that they have often proven toxic in
clinical trials.
HMBD-002 is an IgG4 antibody, so it
blocks VISTA signalling, but does not
necessarily destroy VISTA-positive
cells. This is likely to make it
significantly less toxic in clinical use.
Acute myeloid leukaemia (AML) is the most common form of leukaemia in adults
Pathology Clinical Picture
• AML is a cancer of the progenitor cells
which ordinarily give rise to myeloid
immune cells in the blood
• The cancerous cells multiply in an
uncontrolled way and ‘crowd out’
healthy cells in bone marrow and blood
• Common symptoms of AML include
fatigue, bruising, bleeding, and infection
• Untreated, AML is typically fatal within
months
• Approximately 20,000 patients are
diagnosed annually in the US; most
common in males over 60 years of age
• Five-year survival is estimated at 10% -
35%, depending mainly on the general
health of the patient at diagnosis
Standard of Care
• First-line treatment is with intensive
chemotherapy.
• Approximately 60-80% of patients are
initially responsive. However, roughly
half of those will eventually relapse.
• In patients who relapse, or who are unfit
for intensive chemotherapy, a range of
other agents are used, including
venetoclax (discovered at the Walter &
Eliza Hill Institute in Melbourne,
Australia)
• Stem cell transplants are increasingly
used in patients who are not suitable for
pharmacological therapies
VISTA is a highly relevant and attractive target for treatment of AML
7
Source: TK Kim et al. (2024) J Clin Invest. 134(3):e164325
VISTA is highly expressed
on AML blasts
VISTA expression correlates with
survival in AML models
Blocking VISTA improves clinical
outcome in AML models
High VISTA expression (n=40)
Low VISTA expression (n=40)
Healthy Donor AML
Control
VISTA
Anti-VISTA antibody
Control
• Tumour cells express much higher
levels of VISTA than those drawn from
healthy donors
• Tumours with high VISTA expression
have a worse prognosis than those
with low VISTA expression
• An anti-VISTA antibody significantly
slows tumour growth in an animal
model of AML
The study is planned to be conducted in two stages, under an open label design
8
Stage 1
Confirm dose of
HMBD-002 in
combination with
azacitidine and
venetoclax
Stage 2
Identify signals of
potential efficacy
Expected recruitment: 29 – 38 patients
Patient population: initially relapsed / refractory high-
risk AML / MDS patients, with potential extension to
newly-diagnosed patients in Stage 2
PRELIMINARY
Venetoclax
• Inhibits Bcl-2 protein, implicated in
several blood cancers
• Discovered at Walter & Eliza Hill
Institute (Melbourne, VIC)
• Global sales ~US$ 1.4 billion (2025)
Azacitidine
• Chemotherapy agent that impedes
DNA synthesis in tumours
The Percheron Board and Management Team bring extensive international experience
in drug development, commercialisation, and partnering
9
Dr Charmaine Gittleson
Non-Executive Chair of the
Board
25 years of drug
development experience,
including 15-year tenure with
CSL in international roles
Dr Gil Price
Non-Executive Director
& Chair of Audit
Experienced biotech
executive and entrepreneur
with extensive experience in
drug development
Dr James Garner
Non-Executive Director
25-year track record of
international drug
development, including ten
years as a public company
life sciences CEO
Deborah Ambrosini
Chief Financial Officer
& Company Secretary
Over 20 years of strategic
financial experience with a
diverse range of ASX-listed
companies
Valentina Dubljevic
Chief Technical Officer
20-year track record of
international drug
development in multinational
companies
Dr Eugene Kennedy
Chief Medical Officer
Johns Hopkins-trained cancer
surgeon with over ten years
of experience in immuno-
oncology drug development
* Dr Michael Baker
Chief Executive Officer
& Managing Director
Scientist, investor, and
company-builder, including
six years with Arovella
Therapeutics
* Note: Dr Baker commences his appointment on 5 October 2026
10
Percheron benefits from an ultra-lean operating model, with the majority of cash
outlays being applied directly to R&D
Share Price (AU$)
Corporate Fundamentals (as at 30 June 2026)
Market Capitalisation: AU$ 4.6M
Primary Listing: ASX: PER
Secondary Quotations: OTC: PERCF
Shares on Issue: 1,522 Million
Key Shareholders
Mutual Investments Limited 5.4%
Directors and Management 5.6%
Financial Position (as at 30 June 2026)
Cash Balance: AU$ 4.05 million
Runway (per Appendix 4C): 3.9 quarters
AS AT 30 JUN 2026
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31-Dec-25 31-Mar-26 30-Jun-26
Millions
Volume
Asset Economics
Acquisition Cost ~$4.5 Million
(includes manufacture of one batch of drug substance)
Confidential, substantially back-ended participation for
licensor in success of product
11
CY2026
Presentation of data at scientific conferences 1H CY2026 ✓
Grant of provisional international nonproprietary name (INN) for HMBD-002 by WHO 1H CY2026 ✓
Completion of drug substance manufacture for new clinical trials 1H CY2026 ✓
Engagement with Vanderbilt Health to execute clinical trial in AML 2H CY2026 ✓
Release of completed HMBD-002 drug product for use in clinical trials 2H CY2026
Commencement of new CEO 2H CY2026
FDA approval of Vanderbilt AML study 2H CY2026
First patient in to Vanderbilt AML study 2H CY2026
Potential further clinical trial(s) in additional patient populations 2H CY2026
CY2027
Initial data from AML study CY2027
Percheron is focused on moving its pipeline forward expeditiously, with potential for
rich, value-generating news flow over FY2026
PRELIMINARY
Note: All timelines are indicative and subject to ongoing review
info@PercheronTx.com
www.PercheronTx.com
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