Annual General Meeting - Management Presentation
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ASX Announcement
Percheron Therapeutics Limited
ABN 41 095 060 745
ASX: PER | FSE: AWY | US OTC: ATHJY
AGM PRESENTATION
Melbourne, Australia – 7 October 2026: In advance of this morning’s Annual General
Meeting of shareholders to be held at 10:00am, and in accordance with ASX Listing Rule
3.13.3, Percheron Therapeutics Limited (ASX: PER or ‘the Company’) is pleased to
provide a copy of the presentation that will be made to shareholders at the Annual
General Meeting.
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 further
information, please see our website at www.PercheronTx.com, or email
info@PercheronTx.com.
This announcement has been authorised for release to the Australian Securities Exchange
by the Board of Directors.
ASX: PER US OTC: PERCF
WWW.PERCHERONTX.COM
Annual General
Meeting 2026
CEO and MD address
Dr Michael Baker | October 2026
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.
© Percheron Therapeutics 1
Large Market Opportunity
Strong Team
Percheron Investment Highlights
2
© Percheron Therapeutics
Strong Science Interest For Investigator Led Studies
Clinically Validated Safety
Multiple Partnership Opportunities
HMBD-002 targets VISTA using a differentiated mAb
scaffold supported by compelling preclinical data
US FDA IND-enabled phase 1 study showed favourable
safety profile, alone and in combination with Keytruda
(αPD-1 check point)
Synergy between VISTA and other anti-cancer agents
may make HMBD-002 attractive to potential partners
Investigator initiated clinical studies may expand the
clinical data set at modest cost to Percheron and yield
important data
VISTA is implicated across multiple tumour types,
opening a broad set of potential indications to target
Leadership with deep, hands-on experience in antibody
drug development, regulatory strategy and deal-making
Corporate and Financial Overview
3
© Percheron Therapeutics
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PER Price and Volume - 12 months1
1. As of 2 October 2026; 2. Does not include the proceeds from the R&D
Tax rebate announced 31 August 2026 and the gross proceeds from the
$2.3 million Placement announced 1 September 2026; 3. Holding
percentages reflect holdings post-completion of tranche 1 and pre-
completion of tranche 2 of the Placement announced 1 September 2026.
Corporate Fundamentals
Market Capitalisation1 AU$ 7.3 million
Primary Listing: ASX: PER
Secondary Quotations: OTC: PERCF
Shares on Issue: 1,822 million
Cash Balance (June 30)2 $4.1 million
Key Shareholders
Mutual Investments Limited 4.5%
Glen Bull 4.0%
Non-correlated Capital Limited 3.8%
Directors and Management 4.7%
Checkpoint
inhibitors play a
key role in cancer
treatment
5
© Percheron Therapeutics
Checkpoint inhibitors (i.e. α-PD-1) reactivate T
cells resulting in tumour cell killing
Checkpoint inhibitors can activate T cells or influence
immunosuppressive myeloid cells
Checkpoints can contribute to an immunosuppressive
tumour microenvironment (TME)
Myeloid derived suppressor cells (MDSCs) and tumour associated macrophages (TAMs) express VISTA
and can make the TME immunosuppressive supporting tumour growth
Blocking immunosuppressive myeloid cells can re-
program the TME
Checkpoints act as an inhibitory signal and
block T cell killing
Immune checkpoint inhibitors have been one of the fastest growing
classes of medicine in the immuno-oncology field, with close to US$
50 billion in annual sales
6
© Percheron Therapeutics
Immuno-oncology therapies have had a profound
impact on the cancer treatment landscape, but patent
cliffs are looming
0
10
20
30
40
50
60
2019 2020 2021 2022 2023 2024 2025
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
Source: Company filings; * - primary market exclusivity
45.4
48.6
Yervoy Tecentriq Keytruda Opdivo
Approval 2011 2016 2014 2014
Target CTLA-4 PD-L1 PD-1 PD-1
Patent
Expiration*
2025/2026 2028 2028 2028
Successful immuno-oncology assets are highly partnerable, with
comparable deals suggesting attractive valuations1
7
Date Acquirer Target
Surface
target
Lead asset
stage
Tumour types Upfront Milestone payments
Jan-24 TIGIT Phase 3
Lung cancer, GI
cancer
$320m equity
investment
NA
Jun-23
IL-27 &
CCR8
Phase 2
Lung, liver cancer,
solid tumours
Stock transaction
value of $65m
Acquisition
Mar-23 CTLA-4 Phase 2 / 3
Solid tumours,
NSCLC
$200m
Development, regulatory and commercial
milestones, tiered royalties
Jun-22 PD-1
Commercial /
Phase 3
Skin cancers, NSCLC $900m Milestone payments and royalties
Jun-21 TIGIT Phase 1 Solid tumours $625m
Up to $1.45 billion in milestones; tiered
royalties on Ex-US sales; co-
commercialisation and profit share in US
Dec-21 TIGIT Phase 3
Solid tumours and
lung cancer
$300m
Up to $700 million if the option is exercised;
royalties
Feb-21 PD-1
Commercial /
BLA
Nasopharyngeal
carcinoma, NSCLC
$150m
$380 million in milestone payments and 20%
royalty on net sales
Dec-20 PVRIG Phase 1
Advanced solid
tumours
$85m
Up to $730 million in milestones; tiered
royalties on net sales
Sep-20 CD47 Phase 1 / 2
Solid tumours and
AML
$180m
Up to $1.94 billion; tiered royalties on global
net sales
Mar-20 CD47 Phase 1 / 1b AML, DLBCL $4,900m Acquisition
ILLUSTRATIVE
1. Deal references are provided on the final slide; GI – gastrointestinal; NSCLC – non small cell lung carcinoma; BLA – Biologics License Application; AML – Acute Myeloid Leukaemia; DLBCL – Diffuse Large B-cell Lymphoma.
© Percheron Therapeutics
VISTA is a novel
checkpoint
© Percheron Therapeutics
VISTA is an attractive target across a range of solid tumours
1. https://doi.org/10.1016/j.esmoop.2024.102942; 2. https://doi.org/10.1038/s41598-021-00835-0.
9
© Percheron Therapeutics
VISTA is expressed in tumour
samples from a wide range of
cancer types1
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
A relevant case study: VISTA is the highest checkpoint gene expressed
in late-stage glioma and is prognostic for survival2
High prevalence by IHC
82.4%
High grade gliomas
(III/IV) express VISTA
(14/17
30.8%
Low grade gliomas
(I/II) express VISTA
(4/13)
Highest expression
of all check points
Associated with poor
survival
Targeting VISTA is expected to enhance immune cell mediated tumour
killing1
10
© Percheron Therapeutics
1. VISTA mechanism of action is still being full elucidated; iNOS – Inducible Nitric Oxide Synthase;
ROS – Reactive Oxygen Species; NO – Nitric Oxide
Targeting VISTA enhances the activity of other anti-tumour strategies
11
RT – Radiotherapy; 1. https://jitc.bmj.com/content/10/2/e003382; 2. https://jitc.bmj.com/content/10/Suppl_2/A558; 3. https://doi.org/10.3389/fimmu.2023.1311658; 4. 10.1016/j.celrep.2025.115893
CT26 (colon cancer) –
Monotherapy1
CT26 (colon cancer) –
αVISTA + αPD-12
CT26 (colon cancer) –
αVISTA + αCTLA-43
MOC2 (oral carcinoma) –
αVISTA + Radiotherapy4
© Percheron Therapeutics
HMBD-002 is arguably the most advanced VISTA antibody in clinical
development, and likely the only active member of the IgG4 class
12
© Percheron Therapeutics
Program HMBD-002 TBS-2025 CI-8993 JNJ-61610588 W0180
Company
Isotype IgG4
Optimised
IgG1
IgG1 IgG1 IgG1
Stage
Phase 1
Complete
Phase 1
Phase 1b for
AML
Phase 1/2
Terminated
Phase 1
Terminated
Phase 1
Terminated
Exposure to
Date
48 patients 41 patients 64 patients 12 patients 33 patients
Combination
Data Available
Monotherapy &
Combination with
Pembrolizumab
Monotherapy &
combination with
Pembrolizumab
Monotherapy &
Combination with
Cemiplimab
Monotherapy
Monotherapy &
Combination with
Pembrolizumab
Status Ongoing Ongoing Discontinued Discontinued Discontinued
Notes
Starting an IST
with Vanderbilt
Health for AML
patients
Commencing
phase 1b trial in
AML in CY 2026
pH-sensitive
Antibody
Sources: Company filings; Clinicaltrials.gov; AML – Acute Myeloid Leukemia
HMBD-002
Development to
date
© Percheron Therapeutics
Phase 1 data revealed that HMBD-002 has a compelling safety profile
14
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
Key Takeaways
HMBD-002 safety profile is
favourable for an anti-cancer
therapeutic.
Only few patients experienced
treatment-related adverse events
≥ grade 3 in severity.
Less than half of patients in the
study experienced any treatment-
related adverse events at all.
HMBD-002 and pembrolizumab
combination appeared to cause
no additional toxicity or
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
Source: Unpublished data; TRAE – Treatment Related Adverse Event, QW – Once Weekly; DLT – Dose Limiting Toxicity; CRS – cytokine release syndrome
© Percheron Therapeutics
Patients treated with HMBD-002 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
© Percheron Therapeutics 15
HMBD-002
Development
Strategy
© Percheron Therapeutics
Process Optimisation
Manufacturing
Process Development
HMBD-002 has excellent potential to enhance the activity of a range
of anti-cancer therapeutics
17
Preclinical
Validation
IND-Enabling
Toxicology
NDA-Enabling
Toxicology
Phase 1
Clinical Trial
Phase 2
Clinical Trial
Phase 3
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
48-patient multiple
ascending dose phase
1 study already
completed at top
centers in US; provides
data both as
monotherapy and in
combination with
pembrolizumab
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
Define the best combination for
development – Venetoclax/chemo
(AML), Oncolytic Virus, Photodynamic
Therapy, Radiotherapy,
Sufficient HMBD-002 has been manufactured
to support Investigator Sponsored Trials
(ISTs)
© Percheron Therapeutics
IND – Investigational New Drug; NDA – New Drug Application
18
Strategies to determine the best phase 2 study to test HMBD-002
© Percheron Therapeutics
Initiate Investigator
Sponsored Trials (IST) to
find best combination
• Identify leading cancer centres interested in combining HMBD-002
with standard of care treatments.
• Support completion of clinical studies and assess the efficacy of the
combination
• If the data supports it, commence phase 2 study
• i.e. Vanderbilt – HMBD-002 + venetoclax + chemo for AML
Review the landscape for
new technologies to
in-license
• Engage with research institutes and universities across the globe to
screen for technologies expected to be synergistic with HMBD-002
• Enter into Option Agreements to assess the activity of the
combination
• If promising, execute license agreement
• Focus on cytotoxic therapies and biologics
Complete preclinical
combination studies to
define best combination
approaches
• Identify companies with promising anti-cancer therapies that are
expected to be synergistic with HMBD-002
• Enter into a research collaboration and perform preclinical
studies to assess the synergistic benefit
• i.e. oncolytic viruses, kinase inhibitors, RAS inhibitors,
photodynamic therapy, cell therapy
What are the best combination strategies for HMBD-002
19
Checkpoint Inhibitors (CPIs)
• i.e. PD-1/PD-L1, CTLA-4
• VISTA blocks T cell activation, PD-1
promotes T cell exhaustion
• VISTA upregulation is implicated in
resistance to PD-1 and CTLA-4
therapy
• Preclinical data supports approach
for combination CPIs1,2
• Opens up commercial opportunities
with large pharma
Radiotherapy (RT)
• Causes immunogenic cell death and
increases antigen release
• Induces suppressive myeloid cell
populations, where a VISTA-
targeting mAb should be most
effective3
• Preclinical data supports the
approach4
• Cost effective approach given RT is
widely adopted
Immunogenic Cell Death
Modalities
• i.e. Oncolytic viruses, some antibody
drug conjugates, photodynamic
therapy
• Therapeutics that are directedly
cytotoxic to tumour cells, and also
have the potential to illicit an anti-
tumour response through the
patient’s immune system
© Percheron Therapeutics
1. https://jitc.bmj.com/content/10/Suppl_2/A558; 2. https://doi.org/10.3389/fimmu.2023.1311658; 3. 10.1016/j.ijrobp.2023.11.05 4. 10.1016/j.celrep.2025.115893
What are the most promising tumour types to target
20
Acute myeloid
leukemia (AML)1
Triple negative breast
cancer (TNBC)2 Late-stage glioma3 Non-small cell lung
carcinoma (NSCLC)4,5
VISTA expression on
immunosuppressive
cells in the TME
Yes, highly expressed on
myeloid cells, including
blast cells
Yes, expressed on
infiltrating macrophages
and neutrophils
Yes, and was the highest
checkpoint gene expressed
Yes, expressed on myeloid
cells and also expressed on
some lymphocytes
Prognostic indicator
Yes, increased expression
correlates with disease
progression and reduced
survival
No, but blocking VISTA
reverts immunosuppressive
macrophages, increases T
cell infiltration and
enhances an anti-tumour
response
Yes, increased VISTA
transcripts was associated
with poor prognosis of
glioma patients
Varied, but data suggests
that the VISTA pathway
may contribute to adaptive
resistance mechanisms in
the tumour
microenvironment
Market Size (US
p.a.)
20,000 30,000-40,000 14,000-20,000 190,000-200,000
© Percheron Therapeutics
1. TK Kim et al. (2024) J Clin Invest. 134(3):e164325; 2. https://doi.org/10.1038/s41416-025-03013-5; 3. 10.1038/s41598-021-00835-0; 4. 10.17305/bjbms.2021.6531 5. 10.1158/1078-0432.CCR-17-2542
Investigator
Sponsored Trial
(IST) for AML
© Percheron Therapeutics
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
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
Standard of Care
© Percheron Therapeutics 22
50% RELAPSE
MALES 60+
10% – 35%
VISTA is a highly relevant and attractive target for treatment
for AML1
23
AML – Acute Myeloid Leukamia; 1. 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
© Percheron Therapeutics
The study is planned to be conducted in two stages, under an open
label design1
24
© Percheron Therapeutics
Confirm dose of
HMBD-002 in
combination with
azacitidine and venetoclax
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
Stage 1 Stage 2
1. Final clinical study design will incorporate FDA feedback
CY2026
Release of completed HMBD-002 drug substance (DS) 2H CY2026
New CEO and MD commenced 2H CY2026
Release of completed HMBD-002 drug product (DP) for use in clinical trials 2H CY2026
IND Study May Proceed for Vanderbilt study 2H CY2026
Potential further clinical trial(s) in additional patient populations 2H CY2026
CY2027
First patient recruited into the Vanderbilt AML study 1H CY2027
Initial data from AML study CY2027
Formation of new partnerships to test combinations with HMBD-002 CY2027
Assessment of additional technologies to in-license for combination with HMBD-002 CY2027
Percheron is focused on moving its pipeline forward expeditiously,
with potential for rich, value-generating news flow over FY2027
Note: All timelines are indicative and subject to ongoing review
© Percheron Therapeutics 25
ASX: PER US OTC: PERCF
WWW.PERCHERONTX.COM
Thank you
L30, Collins Place
35 Collins Street Melbourne,
VIC 3000 Australia
+61 3 9827 8999
info@PercheronTx.com
Glossary
27
© Percheron Therapeutics
Cold Tumours
Expression (of an
immune checkpoint)
Immune Checkpoint
Immune Checkpoint
Inhibitor (ICI)
Ligand
Monoclonal Antibody
Myeloid Cells
Tumours with few immune cells in the tumour microenvironment. They are
typically relatively resistant to immuno-oncology therapies. Some cancers, such as
ovarian cancer and brain cancer, are usually considered to be generally 'cold'
tumours. By implicit contrast, a 'hot tumour' or inflammatory tumour is one with a
large and active population of immune cells in the tumour microenvironment.
In the context of immuno-oncology, a tumour with, on average, a lot of a given
immune checkpoint on the surface of its cells is said to highly express that
immune checkpoint. For example, a tumour with high PD-1 expression has a lot of
PD-1 on the surface of its constituent cancer cells.
A control mechanism that allows cells, including tumour cells, to inhibit the local
activity of the immune system. Analogous to the brake pedal on a car. Examples
of immune checkpoints include PD-1, CTLA-4, LAG3, and VISTA.
A drug which inhibits an immune checkpoint, releasing the 'brake' on the local
activity of the immune system. Examples of immune checkpoint inhibitors include
Keytruda(R) (pembrolizumab), Opdivo(R) (nivolumab), Yervoy(R) (ipilimumab), and
Tecentriq(R) (atezolizumab).
Generally, a molecule which connects to another molecule to bring about a
biological effect. In the context of immuno-oncology, a ligand is typically the
molecule on the immune cell to which the immune checkpoint binds (e.g. PD-1 to
PDL-1, CTLA-4 to CD80, LAG3 to Gal3, and VISTA to VSIG-3).
A class of genetically engineered drugs which utilise the structure of natural
human antibodies to generate new molecules which can either block signalling or
attack cells. Approximately 40% of cancer drugs are monoclonal antibodies.
Monoclonal antibodies are manufactured in living cells under carefully controlled
laboratory conditions. They are more challenging to manufacture than 'small
molecule' (chemical) drugs but generally simpler than cell therapies.
Myeloid cells are one type of cells involved in the immune system. They exert a
range of effects, some of which can be immunosuppressive.
Overall Survival (OS)
Progression-Free
Survival (PFS)
RECIST / Overall
Response Rate (ORR)
Resistance
T-Cell / T-Lymphocyte
Tumour
Microenvironment
(TME)
VISTA
A measurement of the average time to death from any cause in a clinical trial.
When treated patients are compared to a control group, an improvement in OS is
considered the 'gold standard' for FDA approval.
A measurement of the average time to disease progression (20% increase in size
of tumour) or death in a clinical trial. When treated patients are compared to a
control group, PFS is often a swifter and more sensitive measure of efficacy than
OS, and can often provide a basis for approval.
A standardised way of measuring the early activity of cancer therapies. A
complete response (CR) describes 100% reduction in a specific lesion on MRI or
CT scan. A partial response (PR) means a 30% or greater reduction in size. Stable
disease (SD) means no material change in the size of the tumour. Progressive
disease (PD) occurs when a lesion grows by more than 20%. ORR is traditionally a
useful way to assess early-stage cancer therapies but rarely provides a basis for
FDA approval and is often less sensitive for targeted therapies such as immune
checkpoint inhibitors.
In oncology, a tumour is said to be resistant to a given therapy if it does not
meaningfully respond to that therapy. Resistance may be primary, if the tumour is
unresponsive from the outset, or secondary / acquired if the tumour becomes
unresponsive after a period of initially successful treatment.
T-cells are one type of cells involved in the immune system. There are many
subtypes, and their effects range from control and coordination ('helper T-cells')to
direct attack of infectious organisms and cancer cells ('cytotoxic T-cells').
The TME includes the tumour itself, along with the surrounding tissues, including
structural support ('stroma'), small blood vessels, etc. The TME is typically also
infiltrated with immune cells. When considering the immune cells specifically, it is
sometimes referred to as the tumour immune microenvironment (TIME).
V-domain immunoglobulin suppressor of T-cell activation. A novel immune
checkpoint, first identified in the 2010s, and the target of HMBD-002.
Deal References
28
1. https://www.gilead.com/news/news-details/2024/gilead-and-arcus-announce-amended-collaboration-and-equity-investment
2. https://investors.coherus.com/news-releases/news-release-details/coherus-acquire-surface-oncology
3. https://www.biontech.com/au/en/home/mediaroom/news/press-releases/2023/03/biontech-and-oncoc4-announce-strategic-collaboration-co-develop.html
4. https://investor.regeneron.com/news-releases/news-release-details/regeneron-strengthens-commitment-oncology-through-purchase
5. https://www.gsk.com/en-gb/media/press-releases/gsk-and-iteos-therapeutics-announce-development/
6. https://www.novartis.com/news/media-releases/novartis-strengthens-immunotherapy-pipeline-option-collaboration-and-license-agreement-beigene-tigit-inhibitor-ociperlimab
7. https://investors.coherus.com/news-releases/news-release-details/coherus-biosciences-expand-late-stage-pipeline-immuno-oncology
8. https://www.pharmalive.com/surface-oncology-gsk-enter-agreement-to-advance-immunotherapy-agent/
9. https://news.abbvie.com/2020-09-04-AbbVie-and-I-Mab-Enter-Into-Global-Strategic-Partnership-for-Differentiated-Immuno-oncology-Therapy
10. https://www.gilead.com/news/news-details/2020/gilead-to-acquire-forty-seven-for-49-billion
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