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Arming the patient’s immune system to fight cancer
Nordic-American Life Science Days
New York City – November 14th 2017
www.targovax.com
The five year survival rate for pancreatic cancer
patients has not improved since the 1970s
2
SOURCE: Cancer Research UK, graphic adapted from The Economist September 16 2017
No improvement in long-
term survival over the
past 45 years
www.targovax.com
The RAS gene is mutated in 90% of pancreatic
cancer patients, making it an ideal target
3
RAS mutations are found
in 90% of pancreatic
cancer patients
RAS mutations result in
uncontrolled cell
division
There are no existing
therapies targeting RAS
Frequency of RAS mutations
www.targovax.com
Targovax’ TG vaccine gears the immune system to
recognize and destroy RAS mutated cancer cells
4
1. Activate immune system
o TG vaccine injected
intradermally and
picked up by APCs
2. Induce mutRAS T-cells
o CD4+ and CD8+ mut-
RAS T-cells induced in
the lymph node
3. Attack the cancer
o mutRAS T-cells identify
and destroy RAS
mutated cancer cells
www.targovax.com
In previous trials in resected pancreatic cancer,
TG vaccination has shown 20% 10 year survival
5
1 Wedén et al., 2011 3 Oettle H et al., JAMA 2013, vol 310, no 14
0
10
20
30
40
50
60
70
80
90
100
0 6 12 18 24 30 36 42 48 54 60 66 72 78 84 90 96 102 108 114 120
o 4/20 (20%) of treated patients
alive after 10 years
o 0/87 untreated patients alive in
a similar cohort from the same
period, at the same hospitals
Survival(%)
Months from resection
10 year survival in historical TG trials in resected pancreatic cancer (n=20, TG monotherapy)
Historical control:
7.7% 10 year survival2
www.targovax.com
These promising results are now being validated in an
ongoing phase I/II trial with adjuvant chemotherapy
6
2nd cohort
(13 patients)
13 of 13 patients (100%) alive 1 year after surgery
mutRAS immune
response (1 yr)
90% of patients (29/32) had RAS-specific immune activation
Safety
TG01 and gemcitabine combination treatment is well-tolerated
Four allergic reactions reported in 1st cohort, none in 2nd
cohort (up to 1 year)
1st cohort
(19 patients)
Median survival 33.1 months vs. 27.6 for historical control
13 of 19 patients (68%) alive 2 years after surgery,
vs. 30-53% in historical controls
www.targovax.com
How TG is different from other peptide vaccines,
and may succeed where others have failed
Target often poorly defined and not
cancer specific
Mutated RAS is a well-defined neo-
antigen, and a driving cause of cancer
Lessons Learned The TG approach
Depot-forming adjuvants not
suitable, as activated T-cells return to
depot instead of tumor site
✓
Insufficient immune activation of
CD4+ helper and CD8+ killer T-cells
Non depot-forming immune modulator
GM-CSF used as adjuvant to stimulate
strong, systemic T-cell response
TG peptides are designed and proven
to induce both CD4+ helper and
CD8+ killer mutRAS-specific T-cells
✓
✓
7
www.targovax.com
Resected pancreatic cancer is the lead indication, but
all RAS mutated cancers are potential TG targets
8
1 2 3 4
Pancreatic
cancer (resected)
Colorectal
cancer
Lung cancer
(NSCLC)
All mutRAS
cancers
TG01 lead indication
Completing phase I/II
Planning phase IIb/III
TG02 lead indication
Phase I trial recruiting
50% RAS mutated
Up to 500.000
patients1
40.000 patients1
TG02 potential
future indication
30% RAS mutated
Up to 500.000
patients1
TG02 + TG03 ultimate
long-term potential
30% of all cancers
Up to 30% of all
cancer patients1
Source: Global data, Riva et al. Plos One 2017 1: Estimated total addressable patient number with RAS mutations in US, EU and China
ONCOS TG
www.targovax.com
Targovax has a broad clinical program with several
important upcoming data read-outs
9
Cancer
indication
Combined
with
ONCOS-102
Melanoma CPI
Mesothelioma Chemo* Orphan ind.
Ovarian &
Colorectal
CPI
Orphan ind.
Sponsor: Ludwig
Prostate DC therapy Sponsor: SOTIO
TG
Resected
Pancreatic
Chemo* Orphan ind.
Colorectal CPI
4 readouts
2017
5 readouts
2018
2017 2018
H1 H2 H1 H2
2019
H1
Phase l
Phase l/ll
Phase l
Phase lb/ll
Phase I/II
Phase Ib
Interim data Clinical, immune and
safety data
* In combination with Standard of Care Chemotherapy. Pemetrexed/cisplatin
for Mesothelioma and Gemcitabine for Resected Pancreatic
Indicative timing of:
www.targovax.com
Targovax has an experienced senior management
team in place to execute the development program
CEO - Øystein Soug
o Former CFO of Algeta ASA
o Track record in bringing an
oncology asset from phase I
through to market launch
o Oversaw the sale of Algeta to
Bayer Healthcare
CMO - Magnus Jäderberg, MD
o Former CMO of Bristol Myers
Squibb Europe
o Brought Yervoy to market as first-
in-class checkpoint inhibitor
o 30 years of experience in R&D
CFO - Erik Digman Wiklund
o Former consultant in McKinsey &
Co Pharma practice
o PhD in cancer epigenetics
o Various commercial and R&D roles
in biotech, including at Algeta
CTIO – Jon Amund Eriksen
o Original inventor of the RAS TG
peptide platform
o Pioneer in immuno-oncology
o 35 years of experience in pharma
R&D and product development
10

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  • 1. www.targovax.com Arming the patient’s immune system to fight cancer Nordic-American Life Science Days New York City – November 14th 2017
  • 2. www.targovax.com The five year survival rate for pancreatic cancer patients has not improved since the 1970s 2 SOURCE: Cancer Research UK, graphic adapted from The Economist September 16 2017 No improvement in long- term survival over the past 45 years
  • 3. www.targovax.com The RAS gene is mutated in 90% of pancreatic cancer patients, making it an ideal target 3 RAS mutations are found in 90% of pancreatic cancer patients RAS mutations result in uncontrolled cell division There are no existing therapies targeting RAS Frequency of RAS mutations
  • 4. www.targovax.com Targovax’ TG vaccine gears the immune system to recognize and destroy RAS mutated cancer cells 4 1. Activate immune system o TG vaccine injected intradermally and picked up by APCs 2. Induce mutRAS T-cells o CD4+ and CD8+ mut- RAS T-cells induced in the lymph node 3. Attack the cancer o mutRAS T-cells identify and destroy RAS mutated cancer cells
  • 5. www.targovax.com In previous trials in resected pancreatic cancer, TG vaccination has shown 20% 10 year survival 5 1 Wedén et al., 2011 3 Oettle H et al., JAMA 2013, vol 310, no 14 0 10 20 30 40 50 60 70 80 90 100 0 6 12 18 24 30 36 42 48 54 60 66 72 78 84 90 96 102 108 114 120 o 4/20 (20%) of treated patients alive after 10 years o 0/87 untreated patients alive in a similar cohort from the same period, at the same hospitals Survival(%) Months from resection 10 year survival in historical TG trials in resected pancreatic cancer (n=20, TG monotherapy) Historical control: 7.7% 10 year survival2
  • 6. www.targovax.com These promising results are now being validated in an ongoing phase I/II trial with adjuvant chemotherapy 6 2nd cohort (13 patients) 13 of 13 patients (100%) alive 1 year after surgery mutRAS immune response (1 yr) 90% of patients (29/32) had RAS-specific immune activation Safety TG01 and gemcitabine combination treatment is well-tolerated Four allergic reactions reported in 1st cohort, none in 2nd cohort (up to 1 year) 1st cohort (19 patients) Median survival 33.1 months vs. 27.6 for historical control 13 of 19 patients (68%) alive 2 years after surgery, vs. 30-53% in historical controls
  • 7. www.targovax.com How TG is different from other peptide vaccines, and may succeed where others have failed Target often poorly defined and not cancer specific Mutated RAS is a well-defined neo- antigen, and a driving cause of cancer Lessons Learned The TG approach Depot-forming adjuvants not suitable, as activated T-cells return to depot instead of tumor site ✓ Insufficient immune activation of CD4+ helper and CD8+ killer T-cells Non depot-forming immune modulator GM-CSF used as adjuvant to stimulate strong, systemic T-cell response TG peptides are designed and proven to induce both CD4+ helper and CD8+ killer mutRAS-specific T-cells ✓ ✓ 7
  • 8. www.targovax.com Resected pancreatic cancer is the lead indication, but all RAS mutated cancers are potential TG targets 8 1 2 3 4 Pancreatic cancer (resected) Colorectal cancer Lung cancer (NSCLC) All mutRAS cancers TG01 lead indication Completing phase I/II Planning phase IIb/III TG02 lead indication Phase I trial recruiting 50% RAS mutated Up to 500.000 patients1 40.000 patients1 TG02 potential future indication 30% RAS mutated Up to 500.000 patients1 TG02 + TG03 ultimate long-term potential 30% of all cancers Up to 30% of all cancer patients1 Source: Global data, Riva et al. Plos One 2017 1: Estimated total addressable patient number with RAS mutations in US, EU and China ONCOS TG
  • 9. www.targovax.com Targovax has a broad clinical program with several important upcoming data read-outs 9 Cancer indication Combined with ONCOS-102 Melanoma CPI Mesothelioma Chemo* Orphan ind. Ovarian & Colorectal CPI Orphan ind. Sponsor: Ludwig Prostate DC therapy Sponsor: SOTIO TG Resected Pancreatic Chemo* Orphan ind. Colorectal CPI 4 readouts 2017 5 readouts 2018 2017 2018 H1 H2 H1 H2 2019 H1 Phase l Phase l/ll Phase l Phase lb/ll Phase I/II Phase Ib Interim data Clinical, immune and safety data * In combination with Standard of Care Chemotherapy. Pemetrexed/cisplatin for Mesothelioma and Gemcitabine for Resected Pancreatic Indicative timing of:
  • 10. www.targovax.com Targovax has an experienced senior management team in place to execute the development program CEO - Øystein Soug o Former CFO of Algeta ASA o Track record in bringing an oncology asset from phase I through to market launch o Oversaw the sale of Algeta to Bayer Healthcare CMO - Magnus Jäderberg, MD o Former CMO of Bristol Myers Squibb Europe o Brought Yervoy to market as first- in-class checkpoint inhibitor o 30 years of experience in R&D CFO - Erik Digman Wiklund o Former consultant in McKinsey & Co Pharma practice o PhD in cancer epigenetics o Various commercial and R&D roles in biotech, including at Algeta CTIO – Jon Amund Eriksen o Original inventor of the RAS TG peptide platform o Pioneer in immuno-oncology o 35 years of experience in pharma R&D and product development 10