1) The document discusses Targovax's clinical programs using oncolytic viruses and neoantigen vaccines to activate a patient's immune system to fight cancer.
2) Targovax has two lead programs - ONCOS, an oncolytic virus, and TG, a neoantigen vaccine. ONCOS has several ongoing clinical trials and TG has one ongoing trial in colorectal cancer.
3) Preliminary data from a TG trial in pancreatic cancer showed increased median overall and disease-free survival compared to historical controls, suggesting efficacy.
Targovax is developing two complementary and highly targeted approaches to cancer immunotherapy: a peptide-based targeted immunotherapy platform for patients with RAS-mutated cancers and a virus-based oncolytic immunotherapy platform based on engineered oncolytic viruses armed with potent immune-stimulating transgenes for patients with solid tumors.
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Global Cancer Vaccine Clinical Pipeline: 298 Vaccines
Marketed Cancer Vaccines: 15 Vaccines
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Global Cancer Tyrosine Kinase Inhibitors Clinical Pipeline by Company, Indication & Phase
Global Cancer Tyrosine Kinase Inhibitors Clinical Pipeline: 411 Drugs
Majority of Cancer Tyrosine Kinase Inhibitors in Preclinical Phase: 164 Drugs
Marketed Cancer Tyrosine Kinase Inhibitors: 32 Drugs
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Targovax is developing two complementary and highly targeted approaches to cancer immunotherapy: a peptide-based targeted immunotherapy platform for patients with RAS-mutated cancers and a virus-based oncolytic immunotherapy platform based on engineered oncolytic viruses armed with potent immune-stimulating transgenes for patients with solid tumors.
Global cancer vaccine market & clinical trial insightKuicK Research
“Global Cancer Vaccine Market & Clinical Trial Insight” Report Highlights:
Global Cancer Market Overview
Emergence of Personalized Cancer Vaccines
Platforms for Cancer Vaccines Delivery
Mechanism of Cancer Vaccines
Global Cancer Vaccines Clinical Pipeline by Phase, Indication, Company & Country
Global Cancer Vaccine Clinical Pipeline: 298 Vaccines
Marketed Cancer Vaccines: 15 Vaccines
Regulatory Framework for Cancer Vaccines Development & Marketing
Global cancer tyrosine kinase inhibitors market & clinical pipeline outlo...KuicK Research
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Introduction to Cancer Tyrosine Kinase Inhibitors
Signaling Pathway of Receptor Tyrosine Kinase
Advantage of Tyrosine Kinase Inhibitors for Treatment of Cancer
Applications of Tyrosine Kinase Inhibitor in Cancer Therapy
Global Tyrosine Kinase Inhibitors Market Overview
Global Cancer Tyrosine Kinase Inhibitors Clinical Pipeline by Company, Indication & Phase
Global Cancer Tyrosine Kinase Inhibitors Clinical Pipeline: 411 Drugs
Majority of Cancer Tyrosine Kinase Inhibitors in Preclinical Phase: 164 Drugs
Marketed Cancer Tyrosine Kinase Inhibitors: 32 Drugs
Global cancer immunotherapy market outlook 2020KuicK Research
"Global Cancer Immunotherapy Market Outlook 2020" Report Highlight:
Introduction & Classification of Cancer Immunotherapy
Global Cancer Immunotherapy Pipeline by Company, Indication & Phase
Marketed Cancer Immunotherapies Clinical Insight & Patent Analysis by Company & Indication
Global Cancer Immunotherapy Pipeline: 1834 Drugs
Marketed Cancer Immunotherapies: 113 Drugs
Cancer Monoclonal Antibodies Pipeline: 622 Cancer mAb
Cancer Vaccines Pipeline: 312 Vaccines
Marketed Cancer mAb: 36 mAb
Marketed Cancer Vaccines: 12 Vaccines
Targovax Next generation immune activators for solid tumorsRoarFredriksen1
Targovax (OSE:TRVX) is a clinical stage immuno-oncology company developing immune activators to target hard-to-treat solid tumors. Targovax’s focus is to activate the patient’s immune system to fight cancer, and to bring benefit to cancer patients with few available treatment alternatives. Targovax is developing its product candidates in different cancer indications, including melanoma, mesothelioma, and multiple myeloma, and has demonstrated a favorable safety and tolerability profile.
Targovax’s lead clinical candidate, ONCOS-102, is a genetically modified oncolytic adenovirus, which has been engineered to selectively infect cancer cells and activate the immune system against the tumor. Following very encouraging clinical data in several indications, both as monotherapy and in combinations, ONCOS-102 is progressing into a randomized phase 2 trial in melanoma patients resistant to PD-1 checkpoint inhibitor treatment.
Building on successful clinical studies which have provided deep mechanistic insights into the tumor biology and the human immune systems, Targovax is researching circular RNA (circRNA) as novel cancer medicines. In addition, Targovax has a KRAS immunotherapy program, with lead cancer vaccine candidate, TG01, expected to enter the clinic in an enhanced format in the second half of 2022. Together this provides Targovax with a rich pipeline of innovative future immunotherapy product candidates to follow ONCOS-102.
Snam 2023-27 Industrial Plan - Financial Presentation
1810 3 q-2018-presentation-v7
1. Activating the
immune
system to
fight cancer
Company presentation
August
2018
Activating the patient’s
immune system to fight
cancer
3Q 2018 Report
1 November 2018
2. This report contains certain forward-looking statements based on uncertainty, since they relate to events and depend on
circumstances that will occur in future and which, by their nature, will have an impact on the results of operations and the
financial condition of Targovax. Such forward-looking statements reflect the current views of Targovax and are based on the
information currently available to the company. Targovax cannot give any assurance as to the correctness of such statements.
There are a number of factors that could cause actual results and developments to differ materially from those expressed or
implied in these forward-looking statements. These factors include, among other things, risks or uncertainties associated with the
success of future clinical trials; risks relating to personal injury or death in connection with clinical trials or following
commercialization of the company’s products, and liability in connection therewith; risks relating to the company’s freedom to
operate (competitors patents) in respect of the products it develops; risks of non-approval of patents not yet granted and the
company’s ability to adequately protect its intellectual property and know-how; risks relating to obtaining regulatory approval and
other regulatory risks relating to the development and future commercialization of the company’s products; risks that research
and development will not yield new products that achieve commercial success; risks relating to the company’s ability to
successfully commercialize and gain market acceptance for Targovax’s products; risks relating to the future development of the
pricing environment and/or regulations for pharmaceutical products; risks relating to the company’s ability to secure additional
financing in the future, which may not be available on favorable terms or at all; risks relating to currency fluctuations; risks
associated with technological development, growth management, general economic and business conditions; risks relating to the
company’s ability to retain key personnel; and risks relating to the impact of competition.
Important
NOTICE AND DISCLAIMER
2
3. Status & Highlights
2. TG neo-antigen vaccine
3. ONCOS oncolytic virus program
4. 3Q 2018 Financials
3
5. 5
Two programs in clinical development, with an
ONCOLYTIC VIRUS LEAD PRODUCT CANDIDATE
ONCOS
Oncolytic virus
TG
Neoantigen
vaccine
Lead product candidate
o Genetically armed adenovirus
o Alerts the immune system to
recognize cancer antigens
o Induces T-cells specific to
the patients’ tumor
o 4 ongoing trials
Pipeline product
o Shared neoantigen, therapeutic
cancer vaccine
o Triggers the immune system to
recognize mutant RAS cancers
o 1 ongoing trial
Triggers patient-
specific responses
No need for
individualization
6. 3Q 2018 HIGHLIGHTS
During period
Corporate development:
Torbjørn Furuseth appointed Chief Financial Officer, with Erik Digman
Wiklund transitioning to the role of Chief Business Officer
Post-period
Publications:
New ONCOS-102 in vivo data in the Journal of Medical Virology, showing
T-cell activation in a mesothelioma model
ONCOS-102 Peritoneal cancer phase I/II trial in combination w/ Imfinzi®:
Safety evaluation of first dose cohort completed without any concerns
ONCOS-102 Prostate cancer phase I trial in combination w/ DCVAC:
First patient has been dosed
ONCOS-102 Melanoma CPI refractory phase I trial:
One complete response among first six patients
Innate and adaptive immune activation in all four evaluated patients
6
Full data set from the TG01 trial in resected pancreatic cancer, including
encouraging disease-free survival data was announced
7. 7
Large deals in the last year show strong
INDUSTRY INTEREST IN ONCOLYTIC VIRUSES
Type of deal Deal value
M&A
Phase I/II
oncolytic virus
BD partnership
Pre-clinical
oncolytic virus
Acquirer Target
USD 400m
up-front cash
USD 140m
up-front cash
Up to USD 1b
total value
M&A
Pre-clinical
oncolytic virus
USD 15m
milestone payment
Up to USD 1b
total value
M&A
Phase I/II
oncolytic virus
USD 250m
up-front cash
9. Frequency of RAS mutations
Global cancer incidents per 10,000
(xx) = no. of cancer patients
9
The RAS gene is mutated in
90% OF PANCREATIC AND 50% OF COLORECTAL CANCERS
0
High
Med
1 2 3 4 5 6 7 8 9 10 11 12 13 14 15
Low
Pancreas
(340,000)
Gallbladder
(180,000)
Melanoma of skin
(230,000)
Prostate
(1,130,000)
Colorectal
(1,360,000)
Lung
(1,820,000)
o RAS mutations are oncogenic and
result in uncontrolled cell
division
o There are no existing therapies
targeting RAS mutations
o Targovax’ TG program is a unique
vaccine approach for mutant
RAS cancer
Fernandez-Medarde; RAS in Cancer and Developmental Diseases; Genes & Cancer. 2011;2(3)
10. 1010
mutRAS immune
activation
Dosing and safety
30 out of 32 patients (94%) had RAS-specific immune activation
Dosing regimen defined and TG01 is well-tolerated in combination with
chemotherapy
TG01 IN RESECTED PANCREATIC CANCER
SIGNAL OF EFFICACY SEEN IN PHASE I/II TRIAL
Median overall
survival (mOS)
(N=32)
33.4 vs. 27.6 months reported in the ESPAC4 trial for gemcitabine alone
(from time of surgery)
o First cohort: 33.1 months mOS (n=19)
o Second cohort: not yet reached (n=13)
Median disease free
survival (mDFS)
16.1 vs. 13.1 months reported in the ESPAC4 trial for gemcitabine alone
(from time of surgery)
o First cohort 13.9 months mDFS (n=19)
o Second cohort 19.5 months mDFS (n=13)
11. 11
TG01 resected pancreas cancer trial overall survival
SECOND PATIENT COHORT PERFORMING BETTER
o 2nd cohort; optimized dosing regimen
o 77% 2-year survival rate (10/13)
o mOS not yet reached
o 9 patients alive at time of analysis
o 1st cohort; full dosing regimen
o 68% 2-year survival rate (13/19)
o mOS 33.1 months (from surgery)
o ESPAC4 mOS 27.6 months
o 5 patients alive at time of analysis
12. DISEASE FREE SURVIVAL FROM SURGERY
Censored= No progression on latest scan collected
o 2nd cohort (n=13)
o Median DFS 19.5 months
o ESPAC4 mDFS 13.1
months
o 1st cohort (n=19)
o Median DFS 13.9 months
12
- - - 1st cohort
2nd cohort
13. 13
OPPORTUNITIES TO DEVELOP THE TG PROGRAM
Rationale for further development of TG
13
Clinical relevance
o Meaningful clinical benefit
(DFS and OS) data in
resected pancreatic cancer
o Immune activation with
generation of mutRAS
specific T-cells
o Good safety profile
Well-defined target
o Cancer neoantigens are
immunogenic and can drive
anti-tumor immunity
o RAS mutations are known
trunk neoantigens present
in large patient populations
Growing interest
o Combinations with CPI
might fully release the
therapeutic potential of
neoantigen vaccines
o Several academic groups
have contacted Targovax to
run trials with TG
“With the emergence of immune checkpoint inhibitors, therapeutic vaccine strategies are primed for a rebirth”
14. 14
TG01 indication
o Ph I/II completed
o Next steps being
reassessed
o ~40 000 incidents
TG02 lead indication
o Ph I trial ongoing
o 50% mutRAS
o ~0.5m incidents
TG02 potential
future indication
o 30% mutRAS
o ~0.5m incidents
TG02 + TG03 long-
term potential
o Up to 30% of all
cancer patients
1
Resected panc-
reatic cancer
2
Colorectal
cancer
3
Lung cancer
(NSCLC)
4
All mutRAS
cancers
Source: Global data, Riva et al. Plos One 2017 Estimated total addressable patient number with RAS mutations in US, EU and China
TG
CLINICAL DEVELOPMENT STRATEGY
15. Sc
15
TG DEVELOPMENT STRATEGY OVERVIEW
TG01-01 phase I/II data in pancreatic cancer
TG02-01 ongoing phase I trial in colorectal cancer
TG01 historical Hydro Pharma data
TG pivotal development
Future indication TBD
Collaborative
pancreas trial
CPI combination
clinical trial
Pre-clinical
package
Pursue
opportunities for
investigator-led
trials in pancreatic
cancer
Evaluate clinical
benefit of TG
vaccination in
combination with
PD-1/L1 blockade
Generate support-
ing pre-clinical TG
data package, incl.
CPI and ONCOS
combination
17. 17
ONCOS
CLINICAL DEVELOPMENT STRATEGY
Target launch
indication
o Ongoing Phase I/II
o Orphan drug status
o Aim to become addition
to SoC
o 15,000 patients per year
Indications with no/
limited effect of CPIs
o Ongoing melanoma
Phase I
o Combo w/PD-1
o >100,000 patients per
year
Peritoneal
malignancies
o Ongoing Phase I/II in
ovarian and colorectal
o Combo w/PD-L1
o >100,000 patients
per year
Platform expansion
with new targets
o Ongoing pre-clinical
testing
o Novel targets and
mode-of-action
o Broad spectrum of solid
tumors
1
Path-to-market
Mesothelioma
2
Proof-of-concept
CPI refractory
3
Proof-of-concept
New CPI indication
4
Next generation
oncolytic viruses
SOURCE: Global Data, EU big 5 + US
18. 18
Compassionate use
program
115 patients o Ovarian and colorectal cancers
o Combination with Imfinzi®
o Intraperitoneal administration
o Collaboration with MedImmune /
AZ, CRI, & Ludwig
Phase I trial
12 patients
7 indications
Peritoneal cancer
Phase I/II
up to 78 patients
Melanoma
Phase I
up to 12 + 12 patients
Mesothelioma
Phase I/II -
randomized
30 patients
o PoC in CPI refractory patients
o Combination with Keytruda®
o Memorial Sloan Kettering
o Shortest path-to-market
o Orphan drug designation
o Combination with SoC chemo
o Randomized vs. SoC
ONCOS
CLINICAL PROGRAM OVERVIEW
Completed trials
Ongoing trials sponsored by Targovax
Ongoing trials sponsored by partner
Prostate cancer
Phase I
up to 15 patients
o Combination with dendritic cell
vaccine (DCVAC)
o Collaboration with Sotio
19. 19
ONCOS-102 in malignant pleural mesothelioma
DEVELOPMENT STRATEGY AND INDICATIVE TIMELINES
2018 2019 2020 2021 2022
o Randomized ORR and OS
data 30 patients
o Decide on possible CPI
combination arm
o EMA & FDA advisory
meetings
o Randomized ORR and OS
data 90 patients
o Potentially use as basis
for a submission for
conditional approval
o Potentially start Phase III
OS trial for full MAA
Ongoing
Phase I/II, randomized
30 patients
Planned
Expansion of randomized Phase II
~60 additional patients (N=90)
Future
Phase III
n=TBD
20. 20
ONCOS-102 + KEYTRUDA MELANOMA TRIAL
One complete response observed in the first six patients
20
1
Safety
2
Innate immune
activation
3
Adaptive immune
activation
4
Efficacy
✓ First safety review
completed with no
safety concerns
✓ ONCOS-102 first
time in melanoma
treatment
✓ Systemic increase
of several pro-
inflammatory
cytokines (6/6
patients)
✓ Complete response
in one out of six
patients
Expected <5% CR in
this CPI refractory
population*
✓ Increase in the
relative level of
cytotoxic CD8+ T-
cells (4/4 patients)
✓ Increase in PD-1
expression on
CD8+ T-cells (4/4
patients)
*SOURCE: Checkmate Pharmaceutical, press release 17 April 2018
21. MELANOMA COMPLETE RESPONSE CASE EXAMPLE
Received 3 injections of ONCOS-102 and 2 Keytruda infusions
Baseline Day 22 Day 63
21 Two immunotherapies received prior to study (Yervoy and Keytruda)
23. Operations The share
TARGOVAX HAS A SOUND FINANCIAL POSITION
with cash to complete the planned clinical program into 2020
173
NOK million
21
USD million
Cash end of 3Q - Sep 30th 2018
-27
NOK million
-3
USD million
Net cash flow - total 3Q
112
NOK million
14
USD million
Annual run rate - last four quarters
470
NOK million
57
USD million
Market Cap - at share price NOK ~9
2.5
NOK million
0.3
USD million
Daily turnover - rolling 6 month avg.
DNB, ABG Sundal Collier, Arctic,
Redeye, Edison
Analyst coverage
23
0.5
% of share capital
24. 24
TARGOVAX IS LISTED ON THE OSLO STOCK EXCHANGE
and included in the OSEBX index as of December 2017
TRVX share turnover (% of
share capital, rolling 12 month)
o USD ~57 m market cap
o USD 0,3m avg. daily turnover
in last 6 months
o USD 13m total turnover in 3Q
o 150k shares avg. daily volume
in 3Q
o ~ 4,000 owners
o 52.6m shares*
(57.4 fully diluted)
* Up until 30th Sep
25. 25
R&D PIPELINE OVERVIEW AND MILESTONES
1 Current standard of care chemotherapy for patients with unresectable malignant pleural mesothelioma
2 Patients with advanced peritoneal disease, who have failed prior standard chemotherapy and have histologically confirmed platinum-resistant or
refractory epithelial ovarian or colorectal cancer
3 Trials sponsored by collaborators
Ongoing collaborator sponsored trials
Mesothelioma
Comb. w/ pemetrexed/cisplatin1
Melanoma
Comb. w/KEYTRUDA®
Peritoneal cancers2,3
Collab: Ludwig, CRI & AZ
Comb. w/IMFINZI®
Prostate3
Collab: Sotio
Comb. w/DCVAC
3 viruses
undisclosed
Pancreatic cancer
Comb. w/gemcitabine
Colorectal cancer
Proof-of-mechanism
Comb. w/KEYTRUDA®
Platform
Product
candidate
Preclinical
Phase
I
Phase
II
Phase
III
Last event
Next expected
event
ONCOS
oncolytic
adenovirus
ONCOS-102
Next-gen
ONCOS
TG01
TG02
TG
neo-
antigen
cancer
vaccine
Phase Ib safety lead-in cohort,
incl. immune activation and
ORR data (6 pts)
ORR and immune activation
(6 pts), 1/6 CR
First dose escalation cohort
safety review (4 pts)
First patient dosed
Virus construct cloning and in
vitro validation
First safety review, incl.
immune activation data (3 pts)
1H 2020
Randomized ORR data
30 pts
1H 2019
ORR and immune data
first cohort
Update by collaborator,
expected 2019
Update by collaborator,
expected 2019
2H 2019
Target disclosure and in
vivo data
1H 2019
Immune activation and
mechanistic data (mono)
1H 2019
TG02 + in vivo data
CPI synergy
TG + PD-1
TG02
mOS 33.4 months
Demonstrated mutant RAS-
specific immune activation
TBD
25