Percheron Therapeutics Limited Corporate Presentation
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ASX Announcement
Percheron Therapeutics Limited
ABN 41 095 060 745
ASX: PER | FSE: AWY | US OTC: ATHJY
PERCHERON THERAPEUTICS CORPORATE PRESENTATION
Melbourne, Australia – 5 November 2025: 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 an updated non-
confidential corporate presentation for the information of shareholders and investors.
The attached presentation supersedes the previous corporate presentation that was
released to the market on 27 June 2025.
The updated presentation offers additional information on the company’s new program,
HMBD-002, as well as an overview of its financial position.
~ 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
Developing High Impact
Therapies for Cancer and Rare Diseases
Corporate Overview
November 2025
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.
ASX-Listed Biotech
Company, Competitively
Valued Against Peers
Percheron (ASX:PER) trades
at a discount to comparable
ASX-listed biotech companies,
suggesting upside potential
Potential First-in-Class
Drug Candidate with
Well-Validated
Mechanism of Action
HMBD-002 targets VISTA, one
of the most promising new
immuno-oncology targets
There are currently no
approved therapies targeting
VISTA, meaning that HMBD-
002 has first-in-class potential
Positive Phase I Trial
Completed in
United States
under FDA Oversight
HMBD-002 has an open IND
application with the
US FDA
A phase I clinical trial has
been completed at leading
cancer centres in the United
States, demonstrating a very
favourable safety profile and
signals of efficacy
Near-Term
Value-Driving Catalysts
Percheron aims to commence
a phase II trial of HMBD-002
in CY2026, moving it rapidly
towards potential
commercialisation
Ongoing preclinical and
regulatory work is expected to
provide additional news flow
Large Commercial
Opportunity with
Substantial Partnering
Potential
If successful, HMBD-002
would enter an immuno-
oncology therapeutics market
worth >US$ 40 billion, in
which high-value partnering
transactions are common
Percheron presents a compelling, differentiated, competitively valued investment
opportunity in the emerging field of immuno-oncology
Percheron’s pipeline includes HMBD-002, a potential first-in-class immuno-oncology
asset with applicability to a wide range of cancer indications
3
Note: VISTA = v-domain immunoglobulin suppressor of T-cell activation
All timelines are indicative and subject to ongoing review
HMBD-002
Monoclonal antibody to VISTA
Triple-Negative Breast Cancer
PRECLIN. PHASE 1 PHASE 2 PHASE 3 MARKET
Phase II Trial Expected
CY2026
Solid Tumours
TBD
COMBINATION THERAPY
EGFR-Mutant Non-Small-Cell Lung Cancer
Phase II Trial Expected
CY2026
TBD
HER2-Negative Oesophageal Cancer
Phase II Trial Expected
CY2026
TBD
Endometrial Cancer
Phase II Trial Expected
CY2026
TBD
Triple-Negative Breast Cancer &
Squamous Cell Cancer of Head & Neck
Radiotherapy
PARTNERSHIPS & COLLABORATIONS
(preclinical research)
Other target indications under evaluation
(preclinical research)
The Percheron Board and Management Team bring extensive international experience
in drug development, commercialisation, and partnering
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
Chief Executive Officer
& Managing Director
20+ 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
5
Percheron benefits from an ultra-lean operating model, with the majority of cash
outlays being applied directly to R&D
-7,000
-6,000
-5,000
-4,000
-3,000
-2,000
-1,000
0
1,000
2,000
3,000
Q2 (Dec) Q3 (Mar) Q4 (Jun) Q1 (Sep) Q2 (Dec) Q3 (Mar) Q4 (Jun) Q1 (Sep)
Income R&D G&A Partnering
FY2026
Cashflows (AU$ ,000)
Net Quarterly Cash Burn
17,194 14,939 11,867 8,014 17,388 12,916 10,168 5,683
Cash at
end of
period:
Corporate Fundamentals (as at 31 October 2025)
Market Capitalisation: AU$ 9M
Primary Listing: ASX: PER
Secondary Quotations: OTC: PERCF
Shares on Issue: 1,087 Million
Average Daily Trading (YTD): ~AU$ 74K
Key Shareholders
Mutual Investments Limited 5.4%
Non-Correlated Capital Limited 4.6%
Powerhouse Ventures Limited 2.3%
Board of Directors 5.6%
Financial Position (as at 31 October 2025)
Cash Balance: AU$ 5.7 million
Runway (per Appendix 4C): 4.0 quarters
AS AT 31 OCT 2025
FY2025
FY2024
6
Percheron is competitively valued in comparison to peer ASX-listed companies
Company Ticker Number of
Programs
Highest Stage of
Development
Cash at
30 June 25
Market Cap at
30 June 25
IMM 4 Phase III $67.4M $350.5M
RAC 1 Phase II $13.6M $204.1M
ALA 2 Preclinical $20.9M $118.9M
IMU 5 Phase II $21.9M $97.1M
ATX 1 Phase II $7.0M $77.6M
RAD 6 Phase II $29.1M $52.0M
PTX 3 Phase II $6.9M $35.4M
PER 1 Phase II – CY2026 $10.2M $10.9M
Source: ASX; Company filings
Indicative List of Comparator ASX-Listed Oncology Companies
7
CY2025
Release of full phase I data 2H CY2025 ✓
Initiate manufacture of new batch of drug substance for clinical trial use 2H CY2025 ✓
Publication of new preclinical data in head & neck cancer and in triple-negative breast cancer 2H CY2025 ✓
Clinician consultation to determine optimal path(s) forward for HMBD-002 in phase II 2H CY2025 ✓
Disclosure of phase II study design and target tumours 2H CY2025 ✓
CY2026
Initiation of phase II trial(s) for HMBD-002 CY2026
Potential application for orphan drug designation with FDA CY2026
Potential application for international non-proprietary name for HMBD-002 CY2026
Potential licensing or acquisition of further pipeline expansion opportunities CY2026 - 27
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
HMBD-002
Monoclonal Antibody to VISTA
HMBD-002 establishes Percheron as an immuno-oncology company, one of the most
exciting areas at the forefront of cancer drug development
9
IMMUNE
CELL
CANCER
CELL
Cancer cell is recognsed by the
immune system and activates
an immune response
The immune system
neutralises and removes
the cancer cell
Ordinarily, the immune system is our first line of
defence against newly-formed cancer cells, just
as it protects from bacteria and viruses.
IMMUNE
CELL
CANCER
CELL
To become established, the cancer cell must
evade the immune system, which it does via
several different mechanisms
Cancer cell evades immune
detection or inhibits the
immune response
The immune response is
ineffective and the tumour
grows unimpeded
IMMUNE
CELL
CANCER
CELL
Immuno-oncology therapies stimulate the
activities of the immune system, including
recognition and removal of tumour cells
Cancer Vaccines
Increase recognition of
cancer by immune system
CAR-T
Augment the native
immune response by
adding active immune cells
Checkpoint Inhibitors
Counter the immune
inhibitory effects of the
tumour
Immune checkpoint inhibitors have been one of the fastest growing classes of
medicine in the immuno-oncology field, with more than US$ 40 billion in annual sales
10
Immune
Cell
Cancer
Cell
CTLA4
PD-1
LAG3
VISTA
CD80 / CD86
PDL-1
Gal3
VSIG-3
eftilagimod
HMBD-002
Immuno-oncology therapies target the interaction between tumours and the
immune system to enhance the immunological response to cancer
Approved Mar 2011
Approved Sept 2014
Approved Dec 2014 Approved May 2016
0
5
10
15
20
25
30
35
40
45
2019 2020 2021 2022 2023
Yervoy (ipilimumab) Tecentriq (atezolizumab)
Keytruda (pembrolizumab) Opdivo (nivolumab)
Immuno-oncology has grown to a
~US$ 40+ billion market
US$, billions
21.4
27.0
30.3
34.6
39.6
Approved
In Development
Source: Company filings
Note: list of approved products and available targets is non-exhaustive
VISTA is a highly attractive target for novel oncology therapies, showing high
expression on many tumours and clear correlation with prognosis
11
Sources: D Nishizaki et al. (2024) ESMO Open 9(4):102942 (panels 1 & 3); LF Kuklinski et al. (2018) Cancer Immunol. Immunother. 67:1113-1121 (panel 2)
VISTA is expressed on a wide range of
tumours
High expression of VISTA is
associated with worse prognosis
0% 25% 50% 75% 100%
ALL CANCERS
Small Bowel
Pancreas
Sarcoma
Colorectal
Ovarian
Liver & Bile Duct
Oesophageal
Breast
Head & Neck
Uterus
Lung
High Medium Low
High VISTA expression is associated
with resistance to PD-1 inhibition
0.28
0.14
1.21
0.64
0.0
0.5
1.0
1.5
Overall Survival (OS) Progression-Free
Survival (PFS)
High VISTA Expression Low VISTA Expression
Survival of 85 melanoma patients comparing
those with VISTA positive tumour-infiltrating
inflammatory cells (TIICs) and those with VISTA
negative TIICs
Hazard Ratio: 3.57
p = 0.005
Survival of 16 pancreatic cancer patients, all
treated with immunotherapy, comparing those
with high and low VISTA expression
Survival (years)
HMBD-002 has shown compelling evidence of activity in a range of preclinical tumour
models, and is synergistic with PD-1 inhibition
12
Monotherapy administration yields up to 80% tumour growth inhibition,
depending on tumour type
Tumour Growth
Inhibition (TGI):
~84%
Tumour Growth
Inhibition (TGI):
~65%
Tumour Growth
Inhibition (TGI):
~40%
Combination with PD-1 inhibition
provides substantial synergy
Tumour Growth
Inhibition (TGI):
~94%
CT26 (Colon cancer)
PDX
Source: B Dharmadhikari et al. (2022) J Immunother. Cancer 10(Suppl 2):A558-A558 (poster presentation to SITC 37th Annual Meeting)
HMBD-002 is arguably the most advanced VISTA antibody in clinical development, and
likely the only active member of the IgG4 class
13
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 Ongoing 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 Ongoing 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.
Process Optimisation
Manufacturing
Process Development
HMBD-002 is well-advanced in clinical development, with a clear path towards
commercialisation
14
Preclinical
Validation
IND-Enabling
Toxicology
NDA-Enabling
Toxicology
Phase I
Clinical Trial
Phase II
Clinical Trial
Phase III
Clinical Trial
✓ ✓ ✓
• Extensive preclinical
package shows HMBD-
002 binds to VISTA and
modulates tumour
immune environment
• Percheron may explore
additional cancer types
in animal models to
inform future clinical
work
• HMBD-002 has an
open IND, indicating
acceptance by FDA of
manufacturing and
toxicology, and has
already conducted a
48-patient study in the
United States
• Possibility that FDA
may require 13-week
toxicology data prior to
NDA approval; for
discussion with
advisors and FDA in
FY2026
• 48-patient multiple
ascending dose phase I
study already
completed at top
centers in US; provides
data both as
monotherapy and in
combination with
pembrolizumab
• Percheron aims to
commence phase II
trial in multiple tumour
types in CY2026
• In oncology, phase III
studies are often
performed as a ‘post-
approval commitment’,
with accelerated
approval granted after
phase II
✓
• Robust, high-yield
process
• 48-month stability data • Routine ‘fine tuning’ to be conducted in parallel to clinical
trials
New
Drug
Application
to
FDA
Preclinical Development Clinical Development
Manufacturing & Technical Development
• Multiple batches
manufactured at 500L
scale
• Favourable cost of
goods, with scope for
further optimisation
PRELIMINARY
HMBD-002
Phase I Clinical Trial Data
The HMBD-002 phase I study adopted a standard ‘3+3’ design, with an initial
monotherapy group (n=28) followed by a pembrolizumab combination group (n=20)
16
Patient Population & Study Design
• Advanced cancer patients with no available
treatment options known to confer clinical
benefit
• ECOG performance status ≤ 1
• 3+3 dose escalation design, with a 21-day
observation period for dose limiting toxicities
• HMBD-002 administered weekly via 60-
minute iv infusion
• 28 patients on monotherapy and 20 patients
in combination with pembrolizumab
Dose Escalation
Cohort 7
(n=3)
1,400
mg QW
Cohort 1
(n=4)
20
mg QW
Cohort 6
(n=3)
720
mg QW
Cohort 5
(n=7)
360
mg QW
Cohort 4
(n=3)
180
mg QW
Cohort 3
(n=3)
120
mg QW
Cohort 2
(n=5)
60
mg QW
Cohort 1
(n=4)
180
mg QW
Cohort 4
(n=5)
1400
mg QW
Cohort 3
(n=8)
720
mg QW
Cohort 2
(n=3)
360
mg QW
Monotherapy
Combination with
pembrolizumab
200mg Q3W
The study population was highly mixed in terms of tumour type, and comprised very
late-stage, heavily pre-treated patients
Notes: PDAC = pancreatic ductal adenocarcinoma; CRC = colorectal carcinoma; TNBC = triple-negative breast cancer; SCC = squamous cell carcinoma
TMB is usually only assessed in certain tumour types, so was not available for all patients
Breakdown of Tumour Types Baseline Disease Parameters
0%
20%
40%
60%
80%
100%
Monotherapy Combination with Pembrolizumab
PDAC CRC TNBC NSCLC SCC Other
n=28 n=20
17
Age (mean) 56.6
Age Range 27 – 81
Time Since Diagnosis (mean) 4.1 years
Time Since Metastasis (mean) 2.6 years
Prior Lines of Systemic Therapy (median) 4
Prior Surgical Treatments (median) 1
Proportion of Patients Who Had Failed
Prior Immune Checkpoint Therapy
69%
Common treatment-related adverse events (TRAEs) included infusion reactions,
gastrointestinal disorders, fatigue, and rash
18
n=28 n=20
• Clinical experience comprises 48 patients
receiving doses up to 1,400mg QW, for up
to ~1 year, as monotherapy or in
combination
• Most common TRAEs were infusion
reactions, gastrointestinal disorders (e.g.
nausea, diarrhea), fatigue, and rash
• 1 dose-limiting toxicity (DLT) observed at
360mg dose: a patient with grade 3
hepatitis which resolved with
corticosteroids
• Only one treatment-related discontinuation:
a combination therapy patient with
pneumonitis (a common side effect of PD-1
inhibition)
• No cases of cytokine release syndrome
(CRS) seen in monotherapy or combination
therapy cohorts
Commentary
• The safety profile of HMBD-002 appears
very favourable in the context of a drug
intended for use in advanced cancer. Only a
handful of patients experienced treatment-
related adverse events grade 3 in severity,
and less than half of patients in the study
experienced any treatment-related adverse
events at all.
• Combination of HMBD-002 with
pembrolizumab appeared in general to be
associated with no additional toxicity and no
potentiation of anti-PD1 toxicity.
0%
20%
40%
60%
80%
100%
Monotherapy Combination with
Pembrolizumab
No TRAEs TRAEs Grade 1-2 TRAEs Grade 3+
Few patients with TRAEs at Grade ≥3;
no incremental toxicity in combination
19
28% of patients in the study showed a best observed response of ‘stable disease’ (SD)
5
6
18
11
5 3
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
100%
Monotherapy Combination Therapy
Commentary
• 18% of patients in the monotherapy group
and 30% of patients in the combination
therapy group had a best observed
response of stable disease (SD), meaning
that their tumour substantially ceased
growing for a period of time.
n=28 n=20
Best Observed Response (%)
per RECIST 1.1
Stable Disease
A change in tumour size
between -30% and +20%
Progressive Disease
A change in tumour size
greater than +20%
Not Evaluable
e.g. due to early loss of
patient from study
20
Overall response rates such as were seen in this study are common for targeted and
cytostatic therapies
Commentary
• Many targeted therapies have shown similar
response profiles in early-phase studies,
and some have gone on to become
blockbuster commercial products.
• There are several reasons why ORR in phase
I sometimes primarily demonstrates a
stabilisation effect:
1. Phase I studies are typically performed
in very advanced patients, who may be
resistant to almost any therapeutic
intervention.
2. ORR can be a reasonable measure of
cytotoxicity and is therefore more useful
in the context of cytotoxic
chemotherapy, but it often performs
less well when measuring the primarily
cytostatic effects of targeted therapies.
3. Immunotherapies in particular can take
longer to show benefit, whereas ORR is
typically a relatively early-focused
measure.
0%
20%
40%
60%
80%
100%
HMBD-002
Not Evaluable
Progressive Disease
Stable Disease
Partial Response
Complete Response
Sources: D Strumberg et al. (2007) Oncologist 12(4):426-437; MS Gordon et al. (2001) J Clin Oncol. 19(3); A O’Donnell et al. (2008) J Clin Oncol. 26(10);
JS Weber et al. (2008) J Clin Oncol. 26(36); PM Fracasso et al. (2007) Clin Cancer Res 13(3):986-993; company and analyst reports
n=48 n=173 n=23 n=25 n=92 n=39
Reported Best Observed Response in Key Phase I Studies (%)
$7.1B
peak
sales
$1.0B
peak
sales
$2.2B
peak
sales
$1.3B
peak
sales
$1.9B
peak
sales
21
A number of patients in the study showed evidence of clinical benefit, providing a
positive signal of potential future efficacy
Patient Diagnosis Time Since
Diagnosis
Prior Lines
of Therapy
Treatment Group Maximum Tumour
Reduction
Time on Therapy
27M Metastatic dedifferentiated liposarcoma 5.1 years 3 360mg Monotherapy ↓ 17.2% 53 weeks
67F Metastatic triple-negative breast cancer 2.8 years 3 360mg Monotherapy ↓ 6.2% 26 weeks
49M Metastatic non-small-cell lung cancer 2.5 years 5 360mg Combination ↓ 1.1% 30 weeks
45F Metastatic triple-negative breast cancer 2.7 years 5 720mg Combination ↓ 26.9% 5 weeks
59M Metastatic squamous cell carcinoma of the head & neck 2.9 years 4 720mg Combination ↓ 13.2% 15 weeks
Patients in the study were very
late-stage and heavily pre-treated
Tumour shrinkage and prolonged periods
of stable disease are positive markers of
potential future efficacy
HMBD-002
Phase II Clinical Trial Design
The planned phase II trial for HMBD-002 is designed to achieve five strategic objectives
Provide multiple shots on goal
to reduce ‘all or nothing’
binary risk
Maintain operational flexibility
to accommodate Percheron’s
financial capacity
Generate frequent read-outs
to guide ongoing development
and inform investors
Adapt trial dynamically to
focus on most promising
patient populations
Optimise opportunities to seek
accelerated approval and
engage with potential partners
1 2 3 4 5
The study will explore several
tumour types in parallel arms
to determine which are most
responsive to the drug
The study will start with one or
two arms, subsequently
adding others according to
emerging data and funding
capacity
Study will likely be conducted
in Australia and US, with
potential to include other
countries
The study will recruit patients
in defined blocks, allowing for
regular read-outs
Not all arms will be pursued to
the conclusion of the study;
unpromising arms will be
dropped in favour of those
showing superior response
The study will be performed at
reputable trial sites under
oversight provided by existing
IND with US FDA
Minimise time, cost, and risk to clinical proof-of-concept, while generating maximally valuable data
23
The phase II study will evaluate multiple tumour types through independent arms in a
single study, a well-established approach that provides efficiency and flexibility
24
Arm 1
Arm 2
Arm 3
Arm 4
Each arm resembles a standalone
study in a specific patient population
Combining the arms into a single
study achieves very substantial
efficiencies and economies of scale
Arms may start and finish at different
times, allowing substantial
operational flexibility
1
2
3
Results of each arm are delivered as
they emerge, independently of the
other arms
4
PRELIMINARY
Each arm will potentially have two stages, allowing for an early read-out of activity
before investing in confirmatory data
25
Arm 1
Arm 2
Arm 3
Arm 4
Arm 4
Exploratory Stage
(single arm)
~20-30 patients
provides early indication of efficacy
before Percheron invests in
confirmatory stage
1
Confirmatory Stage
(randomised)
~40-100 patients
provides more comprehensive
quantification of efficacy versus
comparator
2
Decision to ‘graduate’ from
exploratory to confirmatory stage is
under company’s control and may
take many inputs into consideration
3
Not all arms will graduate – the study
is designed to select only the most
promising patient populations
4
PRELIMINARY
Four arms have been identified as high priority targets, with the potential for others to
be added in future
26
PRELIMINARY
Triple-Negative
Breast Cancer
(TNBC)
EGFR-Mutant
Non-Small Cell Lung Cancer
(NSCLC)
HER2-Negative
Oesophageal Adenocarcinoma
Endometrial
Cancer
~36,000 patients
per annum in United States
~15,000 patients
per annum in United States
~10,000 patients
per annum in United States
up to ~65,000 patients
per annum in United States*
• TNBC lacks surface receptors for
estrogen, progesterone, or HER2,
which are common targets for
therapies, making it very resistant to
drug treatment
• TNBC disproportionately affects
younger women
• Pembrolizumab provides only
marginal survival benefit
• EGFR-mutant NSCLC is driven by a
mutation in a growth signaling
pathway
• It is more common in women, non-
smokers, and Asian patients
• EGFR-mutant NSCLC is relatively less
responsive to immunotherapy such
as pembrolizumab
• Adenocarcinoma is the most
common form of oesophageal
cancer in the Western world
• HER2-negative disease is generally
unresponsive to trastuzumab, a
common therapy used in HER2-
positive cases
• Endometrial cancer is the most
common form of uterine cancer
• Pembrolizumab is approved but only
partially effective, with typical
progression-free survival of 6-11
months
Note: endometrial cancer population subject to further refinement in consultation with advisors
The plan provides the potential for significant news flow from CY2026 onwards
27
2026 2027 2028
Design and set-up
of study; selection
of CRO & sites
Complete
manufacture of
study drug
Enrol patients to open-label
exploratory stage for initial arm(s)
Graduate one or more initial arms
to randomised confirmatory stage
Initiate additional arms, guided by scientific rationale, and in line with company’s resources
1H 26 Selection of CRO
Mid 26 Initiation of sites
Mid 26 First patient in to Arm 1
2H 26 First patient in to subsequent arms
Mid 26 Potential grant of orphan designation
2H 26 Award of international non-prop. name
Late 26 Possible initial data
1H 27 Efficacy data from Arm 1
Mid 27 Efficacy data from subsequent arms
2H 27 Graduation of arm(s) to confirmatory
Mid 27 Potential initiation of new arms
2H 27 Potential grant of Fast Track
Mid 28 Confirmatory data from Arm 1
Mid 28 Confirmatory data from other arms
Mid 28 Efficacy data from other arms
2H 28 Potential grant of Breakthrough
2H 28 Possible New Drug Application
Potential timeframe for partnering
discussions with ‘big pharma’
Potential timeframe for accelerated
approval discussions with FDA
Indicative Cost Estimates
Approximate Cost per Patient
~US$ 150,000
(industry-wide benchmark)
Estimated Number of Patients
in Exploratory Stage
20 - 25
Estimated Cost of Exploratory
Stage for Each Arm
~US$ 3.0 - 3.5 million
(~AU$ 5 million)
(not including impact of R&D
Tax Incentive Rebate)
Note: all timelines, costs, forecasts, and other estimates are preliminary, and subject to ongoing revision
INDICATIVE
PRELIMINARY
HMBD-002
Commercial Opportunity
29
A high-level estimate of market opportunity suggests enormous commercial potential
for HMBD-002; only one indication needs to be successful for a profitable product
Triple-Negative
Breast Cancer
EGFR-Mutant
Non-Small-Cell
Lung Cancer
HER2-Negative
Oesophageal Cancer Endometrial Cancer
Estimated incident patients in
United States, pa
36,000 15,000 10,000 6,500*
Annualised treatment cost, US$ $250K $250K $250K $250K
Median treatment cost in 2020-24
was $350K; reduced to $250K for
conservative estimate
Estimated time on treatment,
months
6 months 4 months 4 months 6 months
Earlier stage patients, and patients
in combination, may result in longer
treatment durations
US addressable market, US$ pa $4.5 billion $1.2 billion $0.8 billion $0.8 billion
Global addressable market, US$
pa
$9.0 billion $2.4 billion $1.6 billion $1.6 billion
Assume that US represents 50% of
global sales
* Endometrial cancer patients estimated at 10% of total pending further analysis of target sub-population
ILLUSTRATIVE
Successful immuno-oncology assets are highly partnerable, with comparable deals
suggesting attractive va
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