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www.targovax.com
Arming the patient’s immune system to fight cancer
DnB Healthcare conference
Øystein Soug, CEO
15 December 2016
www.targovax.com
Important notice and disclaimer
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.
2
www.targovax.com
Enables the immune system to kill cancer cells:
o Oncolytic viruses
• Release cancer antigens
• Imlygic, ONCOS-102
o Peptide vaccines
• Mimic cancer antigens
• TG01, TG02
o Cell therapies
• Load T-cells with antigen receptors
• Chimeric antigen receptors, CARs
o Checkpoint inhibitors
• General upgrade of immune system
• Yervoy, Keytruda, Opdivo, Tecentriq
3
Immunotherapy – enables the immune system to kill cancer cells
Surgery
Chemo
Radio-
therapy
Targeted
therapies
Traditional cancer treatment New approach - Immunotherapy
www.targovax.com
The goal is to make cancer a chronic disease by combining
immuno-oncology therapies
4
o Yervoy started the revolution
in cancer treatment in 2011
o Due to immuno-oncology
combination the number of
addressable cancers is
expected to increase to at
least 60%
Untreated
Time from treatment
Proportionalive
Long term survival
Long term survival
Chemotherapy / TKI
Immunotherapy
combination
Immunotherapy
monotherapy
www.targovax.com
Checkpoint inhibitors show signs of “curing” some cancers
- example of Yervoy treated melanoma
5
Week 108: complete remissionWeek 72: complete remission
1 year20 weeks 8 months
Prior to Yervoy 4 weeks 8 weeks
www.targovax.com
Bladder
6
Response rate to checkpoint inhibitors (CPIs)
ONCOS-102 can
potentially activate
non-responders to
become susceptible
to CPI's
~80%
~84%
~80%
~70%
~70%-80%
~50%
Head and Neck
Lung Carcinoma (NSCLC)
Triple Negative Breast
Renal Cell carcinoma
Melanoma
Large unmet need for checkpoint inhibitor refractory patients
Non-respondersResponders
www.targovax.com
ONCOS-102: CPI refractory melanoma trial details
7
Setting
Background
Key endpoints
Advanced malignant melanoma patients not responsing to CPIs
Immune activate CPI non-responders with ONCOS-102, then re-
challenge with a CPI (Keytruda)
No standard of care for patients not responding to CPI
Safety
Immune activation and clinical response data
Correlation of immune activation and clinical response data
Cohorts
Six patients with prior PD1 monotherapy
Six patients with prior PD1 plus Yervoy combination therapy
www.targovax.com 8
ONCOS-102: CPI refractory melanoma trial details
ONCOS-102 Checkpoint inhibitor (Keytruda)
Weeks3 6 9 12 15 18 24
DAYS
1 4 8
CT CT
PBMC
PBMC
PBMC
3 biopsies per patient
DAY 22 DAY 64Baseline
PBMC
PBMC
Open-label Phase I trial
ONCOS-102: 3 injections at day 1, 4 & 8
CPI (Keytruda) at day 22, then every 3 weeks for 5 months
CT CT
21 27
www.targovax.com 9
At the tumor:
Virus injected directly into tumor,
replicates, lyses cells and releases
antigens. Immune system picks up
antigens
At the lymph node:
Immune system starts production
of tumor specific T-cells
At the tumor lesions:
T-cells find tumor lesions with
corresponding tumor antigens
and kill the cancer cells
Lymph node
How does ONCOS-102 work?
www.targovax.com
Adaptive immune system (biopsy) Anti-tumor immune response (blood)Innate Immune System (biopsy)
Initial ONCOS-102 trial showed strong T-cell response
10
OvCa.
patient
(FI1-19)
o Increase in T-cell infiltration into tumors
(including CD8+ killer T-cells) in 11 out
of 12 patients
o Observation in one non-injected distant
metastasis
o Induction of proinflammatory cytokines
+ fever (all patients)
o Infiltration of innate immune cells into
tumors in 11 out of 12 patients
o Systemic induction of tumor-specific
CD8+ T-cells
Correlation between post-treatment
increase in innate immune cells and OS
Associated with clinical benefit
Correlation between post-treatment
increase in CD8+ T-cells and OS
(p=0.008, R=0.74)
Evidence that immune system
recognizes tumor threat
Evidence that T-cells find the
tumor and are cell killing
Evidence that newly produced
T-cells are tumor specific
Mesothelioma patient:
MAGE-A3 specific CD8+ cells
Ovarian patient:
NY-ESO-1, MAGE-A1, MAGE-A3, and
Mesothelin specific CD8+ cells
Overall survival
Scatterplot of ranks
IncreaseinCD68+
cellspost-treatment
p=0.0004, R=0.86
www.targovax.com
Six shots on goal
11
Cancer
indication
Combo
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
H2
2016
Interim data Clinical, immune and
safety data
www.targovax.com
TG01 upcoming data: Two-year survival in resected pancreatic
cancer
12
First half 2017
Two-year survival
First cohort: 19 patients
TG has potential to enable the immune system to identify and
destroy tumors bearing any RAS mutations.
2015-16 data demonstrated
• 14 of 15 patients alive after 1 year
(19 ITT, 15 evaluable patients)
• DTH response 15 of 18 patients
• RAS specific T-cell response: 6 of
first 8 patients
www.targovax.com
Multiple near term value inflection points
2015 2016 2017 2018H1 H2H1 H2
Phase ll initiated
TG01
Immune activation
and MoA demo
ONCOS-102
Interim data
pancreas
TG01 (1st cohort)
Immune activation
pancreas
TG01 (2nd cohort)
2-year survival
pancreas
TG01 (1st cohort)
2-year survival
pancreas
TG01 (2nd cohort)
Initiate Phase l/ll
mesothelioma
ONCOS-102
Initiate Phase l
prostate
ONCOS-102
Initiate Phase l/ll
melanoma
ONCOS-102
Interim data
ovarian
ONCOS-102
Phase l/ll data
melanoma
ONCOS-102
Interim data
melanoma
ONCOS-102
Interim data
mesothelioma
ONCOS-102
Initiate immune
testing colorectal
TG02
Interim data
colorectal
TG02 (mono)
Initiate Phase l
ovarian
ONCOS-102
Interim data
prostate
ONCOS-102
Phase I data/
PoC colorectal
TG02 (combo)
H1 H2
13
www.targovax.com
Financial summary
Operations
Cash NOK 193m at 30 September 2016
Cash run rate NOK 121m (last four quarters)
Annual opex NOK 129m (last four quarters)
14
The Targovax share OSE: TRVX
Daily liquidity NOK 4m (last month’s avg.)
Market Cap NOK 600m
Number of shares 42,2m (44,4m fully diluted)
Analyst coverage DNB, ABGSC, Arctic, Redeye, Norske
Aksjeanalyser
www.targovax.com 15
Clinical trials
TG
ONCOS
1
2
3
 Six shots on goal
 Two year survival data of TG01 in resected pancreatic cancer
 Data in 1H17
 Important proof of concept trial in CPI refractory melanoma
 Data in 2H17
Arming the patient’s immune system to fight cancer

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Arming the patient's immune system to fight cancer

  • 1. www.targovax.com Arming the patient’s immune system to fight cancer DnB Healthcare conference Øystein Soug, CEO 15 December 2016
  • 2. www.targovax.com Important notice and disclaimer 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. 2
  • 3. www.targovax.com Enables the immune system to kill cancer cells: o Oncolytic viruses • Release cancer antigens • Imlygic, ONCOS-102 o Peptide vaccines • Mimic cancer antigens • TG01, TG02 o Cell therapies • Load T-cells with antigen receptors • Chimeric antigen receptors, CARs o Checkpoint inhibitors • General upgrade of immune system • Yervoy, Keytruda, Opdivo, Tecentriq 3 Immunotherapy – enables the immune system to kill cancer cells Surgery Chemo Radio- therapy Targeted therapies Traditional cancer treatment New approach - Immunotherapy
  • 4. www.targovax.com The goal is to make cancer a chronic disease by combining immuno-oncology therapies 4 o Yervoy started the revolution in cancer treatment in 2011 o Due to immuno-oncology combination the number of addressable cancers is expected to increase to at least 60% Untreated Time from treatment Proportionalive Long term survival Long term survival Chemotherapy / TKI Immunotherapy combination Immunotherapy monotherapy
  • 5. www.targovax.com Checkpoint inhibitors show signs of “curing” some cancers - example of Yervoy treated melanoma 5 Week 108: complete remissionWeek 72: complete remission 1 year20 weeks 8 months Prior to Yervoy 4 weeks 8 weeks
  • 6. www.targovax.com Bladder 6 Response rate to checkpoint inhibitors (CPIs) ONCOS-102 can potentially activate non-responders to become susceptible to CPI's ~80% ~84% ~80% ~70% ~70%-80% ~50% Head and Neck Lung Carcinoma (NSCLC) Triple Negative Breast Renal Cell carcinoma Melanoma Large unmet need for checkpoint inhibitor refractory patients Non-respondersResponders
  • 7. www.targovax.com ONCOS-102: CPI refractory melanoma trial details 7 Setting Background Key endpoints Advanced malignant melanoma patients not responsing to CPIs Immune activate CPI non-responders with ONCOS-102, then re- challenge with a CPI (Keytruda) No standard of care for patients not responding to CPI Safety Immune activation and clinical response data Correlation of immune activation and clinical response data Cohorts Six patients with prior PD1 monotherapy Six patients with prior PD1 plus Yervoy combination therapy
  • 8. www.targovax.com 8 ONCOS-102: CPI refractory melanoma trial details ONCOS-102 Checkpoint inhibitor (Keytruda) Weeks3 6 9 12 15 18 24 DAYS 1 4 8 CT CT PBMC PBMC PBMC 3 biopsies per patient DAY 22 DAY 64Baseline PBMC PBMC Open-label Phase I trial ONCOS-102: 3 injections at day 1, 4 & 8 CPI (Keytruda) at day 22, then every 3 weeks for 5 months CT CT 21 27
  • 9. www.targovax.com 9 At the tumor: Virus injected directly into tumor, replicates, lyses cells and releases antigens. Immune system picks up antigens At the lymph node: Immune system starts production of tumor specific T-cells At the tumor lesions: T-cells find tumor lesions with corresponding tumor antigens and kill the cancer cells Lymph node How does ONCOS-102 work?
  • 10. www.targovax.com Adaptive immune system (biopsy) Anti-tumor immune response (blood)Innate Immune System (biopsy) Initial ONCOS-102 trial showed strong T-cell response 10 OvCa. patient (FI1-19) o Increase in T-cell infiltration into tumors (including CD8+ killer T-cells) in 11 out of 12 patients o Observation in one non-injected distant metastasis o Induction of proinflammatory cytokines + fever (all patients) o Infiltration of innate immune cells into tumors in 11 out of 12 patients o Systemic induction of tumor-specific CD8+ T-cells Correlation between post-treatment increase in innate immune cells and OS Associated with clinical benefit Correlation between post-treatment increase in CD8+ T-cells and OS (p=0.008, R=0.74) Evidence that immune system recognizes tumor threat Evidence that T-cells find the tumor and are cell killing Evidence that newly produced T-cells are tumor specific Mesothelioma patient: MAGE-A3 specific CD8+ cells Ovarian patient: NY-ESO-1, MAGE-A1, MAGE-A3, and Mesothelin specific CD8+ cells Overall survival Scatterplot of ranks IncreaseinCD68+ cellspost-treatment p=0.0004, R=0.86
  • 11. www.targovax.com Six shots on goal 11 Cancer indication Combo 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 H2 2016 Interim data Clinical, immune and safety data
  • 12. www.targovax.com TG01 upcoming data: Two-year survival in resected pancreatic cancer 12 First half 2017 Two-year survival First cohort: 19 patients TG has potential to enable the immune system to identify and destroy tumors bearing any RAS mutations. 2015-16 data demonstrated • 14 of 15 patients alive after 1 year (19 ITT, 15 evaluable patients) • DTH response 15 of 18 patients • RAS specific T-cell response: 6 of first 8 patients
  • 13. www.targovax.com Multiple near term value inflection points 2015 2016 2017 2018H1 H2H1 H2 Phase ll initiated TG01 Immune activation and MoA demo ONCOS-102 Interim data pancreas TG01 (1st cohort) Immune activation pancreas TG01 (2nd cohort) 2-year survival pancreas TG01 (1st cohort) 2-year survival pancreas TG01 (2nd cohort) Initiate Phase l/ll mesothelioma ONCOS-102 Initiate Phase l prostate ONCOS-102 Initiate Phase l/ll melanoma ONCOS-102 Interim data ovarian ONCOS-102 Phase l/ll data melanoma ONCOS-102 Interim data melanoma ONCOS-102 Interim data mesothelioma ONCOS-102 Initiate immune testing colorectal TG02 Interim data colorectal TG02 (mono) Initiate Phase l ovarian ONCOS-102 Interim data prostate ONCOS-102 Phase I data/ PoC colorectal TG02 (combo) H1 H2 13
  • 14. www.targovax.com Financial summary Operations Cash NOK 193m at 30 September 2016 Cash run rate NOK 121m (last four quarters) Annual opex NOK 129m (last four quarters) 14 The Targovax share OSE: TRVX Daily liquidity NOK 4m (last month’s avg.) Market Cap NOK 600m Number of shares 42,2m (44,4m fully diluted) Analyst coverage DNB, ABGSC, Arctic, Redeye, Norske Aksjeanalyser
  • 15. www.targovax.com 15 Clinical trials TG ONCOS 1 2 3  Six shots on goal  Two year survival data of TG01 in resected pancreatic cancer  Data in 1H17  Important proof of concept trial in CPI refractory melanoma  Data in 2H17 Arming the patient’s immune system to fight cancer