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www.targovax.com
Arming the patient’s immune system to fight cancer
1Q 2018 presentation
3 May 2018
First quarter results
2018
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
Agenda
3
1Q 2018 Highlights
ONCOS-102 Mesothelioma update
1Q 2018 Financials
www.targovax.com
Highlights from the first quarter 2018
4
Dr. Michael Bogenstätter was appointed Chief Business Officer
Research &
Development
ONCOS-102 generated immune activation in 4/4 patients treated
in the checkpoint inhibitor refractory melanoma trial
Corporate
Post-period
The safety lead-in cohort of ONCOS-102 in mesothelioma was
completed without any concerns
ONCOS-102 generated immune activation in the first treated
patients in the mesothelioma trial
Dr. Catherine Wheeler was elected new member of the Board
A new site in Philadelphia was opened for the melanoma trial
Early signal of efficacy was detected in the ONCOS-102 meso-
thelioma trial, with responses observed in 3 out of 6 patients
www.targovax.com
Targovax has two immuno-oncology programs in
clinical development
5
ONCOS
Oncolytic virus
TG
RAS neoantigen
vaccine
o Shared neoantigen, off-the-shelf cancer vaccine
o Targets oncogenic, mutated RAS neoepitopes
o Induces T-cells specific to RAS mutations
o Genetically armed adenovirus
o Makes cancer antigens visible to immune system
o Induces T-cells specific to patients’ tumor
ONCOS TG
www.targovax.com
Overview of Targovax’ full clinical program
Cancer Indication
TG
Resected Pancreas
Resected Pancreas
Planned registration program
Colorectal
ONCOS-102
Melanoma
Mesothelioma
Ovarian & Colorectal
Collab. w/CRI & MedImmune
Prostate
Collab. w/Sotio
6
2018 2019
H1 H2 H1 H2
2020
H1
Ph l/II
Phase lb
Planned Phase II (lead-in)
Phase l
Phase lb/II
Dose escalating Ph I Ph II
Phase l
Interim data
Clinical, immune
and safety data
ONCOS TG
www.targovax.com
ONCOS clinical program overview
7
Initial Phase I trial
Solid tumors
7 indications
Mesothelioma
Phase I/II - controlled
30 patients
Melanoma
Phase I
12 patients
Prostate
Phase I
10 patients
Ovarian / colorectal
Phase I/II
up to 78 patients
o Combination with PD-1
CPI in refractory patients
o Proof-of-concept
o Memorial Sloan Kettering
o Combination with chemo SoC
o Randomized trial
o Orphan indication
o Collaboration with Ludwig & CRI
o Combination with Medimmune’s
durvalumab (Imfinzi™)
o Collaboration with SOTIO
o Combination with DC therapy
Compassionate
use program
Finland
115 patients
ONCOS TG
www.targovax.com
Agenda
8
1Q 2018 Highlights
ONCOS-102 Mesothelioma update
1Q 2018 Financials
www.targovax.com
ONCOS clinical program overview
9
Initial Phase I trial
Solid tumors
7 indications
Mesothelioma
Phase I/II - controlled
30 patients
Melanoma
Phase I
12 patients
Prostate
Phase I
10 patients
Ovarian / colorectal
Phase I/II
up to 78 patients
o Combination with PD-1
CPI in refractory patients
o Proof-of-concept
o Memorial Sloan Kettering
o Combination with chemo SoC
o Randomized trial
o Orphan indication
o Collaboration with Ludwig & CRI
o Combination with Medimmune’s
durvalumab (Imfinzi™)
o Collaboration with SOTIO
o Combination with DC therapy
Compassionate
use program
Finland
115 patients
ONCOS TG
www.targovax.com
Malignant pleural mesothelioma is a difficult-to-treat
cancer of the lining of the lung
ONCOS TG
10
o Orphan disease, estimated 15,000 new
cases per year (EU, USA, Australia)
o Often caused by asbestos exposure
o Diagnosis usually too late for surgery
o No significant treatment advance in
the last decade
o Median survival 12 months with current
standard of care (SoC), 5 year OS <10%
o Chemotherapy (pemetrexed / cisplatin)
is the SoC in non-resected disease
SOURCE: Global Data
www.targovax.com
Mesothelioma treatment options
ONCOS TG
11
o Offers best prognosis, but only about 10% of
patients are resectable
o Technically challenging due to location
Chemotherapy
Immunotherapy
Surgery
Radiotherapy
o Rarely used as the shape of mesothlioma
tumors make them hard to target
o Mainly palliative care
o Pem/Cis only approved SoC
o 40% response in 1st line, <10% in 2nd line
o 6 month PFS and 12 month median OS
o Mixed signals from early CPI trials
o Nivo+ipi phase III trial reading out 20211
o Currently no/few other oncolytic viruses
1: BMS trial CheckMate743
www.targovax.com
ONCOS-102 in Mesothelioma – Phase I/II study design
Patient population
Advanced malignant
pleural mesothelioma
1st line / 2nd line
Non-randomized
Safety lead-in (n=6)
ONCOS-102
plus SoC chemotherapy
(6 cycles)
Experimental group (n=14)
ONCOS-102 (6 administrations)
SoC (6 cycles)
Control group (n=10)
SoC (6 cycles)
GO
Randomized
2:1
ONCOS TG
Safety lead-in
completed
12
www.targovax.com 13
Mesothelioma trial: Early signal of clinical efficacy in the 6
patient safety lead-in cohort
ONCOS TG
✓ Phase Ib safety lead-in cohort of six patients completed with
no safety concerns
✓ ONCOS-102 first time in combination with chemotherapy
✓ Now recruiting patients into the randomized phase II part
Safety
Innate immune
activation
Adaptive immune
activation
Clinical efficacy
✓ Systemic increase of several pro-inflammatory cytokines
in 6/6 patients (IL-6, TNFα and IFNγ)
✓ Increase in infiltration of CD4+ helper and CD8+ cytotoxic
T cells into lesions (3/4 patients with pre- and post-biopsies
available)
✓ 3 of 6 patients in the safety lead-in cohort showed clinical
response after 6 months (50% response rate, RECIST 1.1)
www.targovax.com 14
Mesothelioma trial: Safety cohort patient split and
clinical responses
ONCOS TG
Safety lead-in
cohort
6 patients
1st line treatment
3 patients
2nd / 3rd line
treatment
3 patients
o Safety & tolerability
o Immune activation
o Clinical reponse rate
Clinical response
2 patients
Progression of
disease
1 patient
Clinical response
1 patient
Progression of
disease
2 patients
o No previous chemo-
therapy treatment
o Previous chemo-
therapy treatment
o 1 partial reponse
o 1 stable disease
o 1 stable disease
www.targovax.com 15
ONCOS-102 opportunity in malignant mesothelioma
ONCOS TG
o ONCOS-102 has the opportunity to become standard of care in 1st line
treatment
- In combination with current SoC chemotherapy
o ONCOS-102 has orphan drug designation in mesothelioma in both the
US and EU
- 7 year market exclusivity in the US and 10 years in the EU
o Few direct IO competitors in current development
- CPIs are potential combination therapies with ONCOS, rather than
competitors
- No/few competing viruses and vaccines in clinical development
Rationale for ONCOS-102 opportunity in mesothelioma
www.targovax.com
Agenda
16
1Q 2018 Highlights
ONCOS-102 Mesothelioma update
1Q 2018 Financials
www.targovax.com
Targovax has a sound financial position, with cash to
complete the planned clinical program well into 2019
Operations
Cash end of Q1 NOK 229m USD 29m Mar 31st 2018
Net cash flow NOK -32m USD -4m Total Q1
Annual run rate NOK 113m USD 15m Last four quarters
17
The share OSE: TRVX
Market Cap NOK 850m USD ~110m At share price NOK ~16
Daily turnover NOK 3m USD 0.4m Rolling 6 month avg.
Analyst
coverage
DNB, ABG Sundal Collier, Arctic, Redeye, Norske Aksjeanalyser,
Edison
CORPORATE
www.targovax.com
The increase in run rate over the last 12 months is
driven by higher spend on R&D
18
CORPORATE
Other45.6m
39%
70.0m
61%R&D
Other
45.6m
36%
80.8m
64%
R&D
R&D spend (NOK million)
2Q16 -
1Q17
2Q17 -
1Q18
Total: 115.6m Total: 126.4m
(Amounts in NOK thousands)
Total of which R&D Total of which R&D
External R&D expenses 43.0 43.0 48.0 48.0
Payroll and related expenses 47.1 25.8 52.8 31.8
Other operating expenses 25.5 1.3 25.6 1.0
Total operating expenses 115.6 70.0 126.4 80.8
12 months rolling
2Q 2017 - 1Q 20182Q 2016 - 1Q 2017
www.targovax.com
Targovax is listed on the Oslo Stock Exchange, and
included in the OSEBX index as of December 2017
19
TRVX share turnover (% of share capital, rolling 12 month)
• NOK ~850 m
market cap
• NOK 3m avg. daily
turnover in last 6
months
• NOK 213m total
turnover in 1Q
• 190k shares avg.
daily volume in 1Q
• >4,100 owners
• 52.6m shares*
(57.4 fully diluted)
CORPORATE
www.targovax.com
News flow – Multiple near-term value inflection pointsInitiationRead-outsCorp
20
2018 2019
H1 H2 H1 H2
2020
H1
2017
H1 H2
Pancreas
full data
Prostate
ph I
Mesothl.
interim data
Melanoma
interim data
Pancreas
ph II
Melanoma
full data
Colorectal
full data
Mesothl.
full data
Pancreas
2yr survival
Melanoma
safety data
Mesothl.
safety data
Colorectal
ph Ib
Colorectal
safety data
OvCa / Col.
ph l
Melanoma
ph l/ll
OSE
main list
OSEBX
index
www.targovax.com 21
Broad clinical
program
TG
ONCOS
✓ Six shots on goal
✓ Several upcoming data points
✓ Unique approach for targeting RAS mutations
✓ Potential to benefit up to 1/3 of all cancer patients
✓ Demonstrated ability to increase T-cell count
✓ Early signal of efficacy in difficult-to-treat tumors
Arming the patient’s immune system to fight cancer

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180504 1 q18 presentation_v7

  • 1. www.targovax.com Arming the patient’s immune system to fight cancer 1Q 2018 presentation 3 May 2018 First quarter results 2018
  • 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 Agenda 3 1Q 2018 Highlights ONCOS-102 Mesothelioma update 1Q 2018 Financials
  • 4. www.targovax.com Highlights from the first quarter 2018 4 Dr. Michael Bogenstätter was appointed Chief Business Officer Research & Development ONCOS-102 generated immune activation in 4/4 patients treated in the checkpoint inhibitor refractory melanoma trial Corporate Post-period The safety lead-in cohort of ONCOS-102 in mesothelioma was completed without any concerns ONCOS-102 generated immune activation in the first treated patients in the mesothelioma trial Dr. Catherine Wheeler was elected new member of the Board A new site in Philadelphia was opened for the melanoma trial Early signal of efficacy was detected in the ONCOS-102 meso- thelioma trial, with responses observed in 3 out of 6 patients
  • 5. www.targovax.com Targovax has two immuno-oncology programs in clinical development 5 ONCOS Oncolytic virus TG RAS neoantigen vaccine o Shared neoantigen, off-the-shelf cancer vaccine o Targets oncogenic, mutated RAS neoepitopes o Induces T-cells specific to RAS mutations o Genetically armed adenovirus o Makes cancer antigens visible to immune system o Induces T-cells specific to patients’ tumor ONCOS TG
  • 6. www.targovax.com Overview of Targovax’ full clinical program Cancer Indication TG Resected Pancreas Resected Pancreas Planned registration program Colorectal ONCOS-102 Melanoma Mesothelioma Ovarian & Colorectal Collab. w/CRI & MedImmune Prostate Collab. w/Sotio 6 2018 2019 H1 H2 H1 H2 2020 H1 Ph l/II Phase lb Planned Phase II (lead-in) Phase l Phase lb/II Dose escalating Ph I Ph II Phase l Interim data Clinical, immune and safety data ONCOS TG
  • 7. www.targovax.com ONCOS clinical program overview 7 Initial Phase I trial Solid tumors 7 indications Mesothelioma Phase I/II - controlled 30 patients Melanoma Phase I 12 patients Prostate Phase I 10 patients Ovarian / colorectal Phase I/II up to 78 patients o Combination with PD-1 CPI in refractory patients o Proof-of-concept o Memorial Sloan Kettering o Combination with chemo SoC o Randomized trial o Orphan indication o Collaboration with Ludwig & CRI o Combination with Medimmune’s durvalumab (Imfinzi™) o Collaboration with SOTIO o Combination with DC therapy Compassionate use program Finland 115 patients ONCOS TG
  • 8. www.targovax.com Agenda 8 1Q 2018 Highlights ONCOS-102 Mesothelioma update 1Q 2018 Financials
  • 9. www.targovax.com ONCOS clinical program overview 9 Initial Phase I trial Solid tumors 7 indications Mesothelioma Phase I/II - controlled 30 patients Melanoma Phase I 12 patients Prostate Phase I 10 patients Ovarian / colorectal Phase I/II up to 78 patients o Combination with PD-1 CPI in refractory patients o Proof-of-concept o Memorial Sloan Kettering o Combination with chemo SoC o Randomized trial o Orphan indication o Collaboration with Ludwig & CRI o Combination with Medimmune’s durvalumab (Imfinzi™) o Collaboration with SOTIO o Combination with DC therapy Compassionate use program Finland 115 patients ONCOS TG
  • 10. www.targovax.com Malignant pleural mesothelioma is a difficult-to-treat cancer of the lining of the lung ONCOS TG 10 o Orphan disease, estimated 15,000 new cases per year (EU, USA, Australia) o Often caused by asbestos exposure o Diagnosis usually too late for surgery o No significant treatment advance in the last decade o Median survival 12 months with current standard of care (SoC), 5 year OS <10% o Chemotherapy (pemetrexed / cisplatin) is the SoC in non-resected disease SOURCE: Global Data
  • 11. www.targovax.com Mesothelioma treatment options ONCOS TG 11 o Offers best prognosis, but only about 10% of patients are resectable o Technically challenging due to location Chemotherapy Immunotherapy Surgery Radiotherapy o Rarely used as the shape of mesothlioma tumors make them hard to target o Mainly palliative care o Pem/Cis only approved SoC o 40% response in 1st line, <10% in 2nd line o 6 month PFS and 12 month median OS o Mixed signals from early CPI trials o Nivo+ipi phase III trial reading out 20211 o Currently no/few other oncolytic viruses 1: BMS trial CheckMate743
  • 12. www.targovax.com ONCOS-102 in Mesothelioma – Phase I/II study design Patient population Advanced malignant pleural mesothelioma 1st line / 2nd line Non-randomized Safety lead-in (n=6) ONCOS-102 plus SoC chemotherapy (6 cycles) Experimental group (n=14) ONCOS-102 (6 administrations) SoC (6 cycles) Control group (n=10) SoC (6 cycles) GO Randomized 2:1 ONCOS TG Safety lead-in completed 12
  • 13. www.targovax.com 13 Mesothelioma trial: Early signal of clinical efficacy in the 6 patient safety lead-in cohort ONCOS TG ✓ Phase Ib safety lead-in cohort of six patients completed with no safety concerns ✓ ONCOS-102 first time in combination with chemotherapy ✓ Now recruiting patients into the randomized phase II part Safety Innate immune activation Adaptive immune activation Clinical efficacy ✓ Systemic increase of several pro-inflammatory cytokines in 6/6 patients (IL-6, TNFα and IFNγ) ✓ Increase in infiltration of CD4+ helper and CD8+ cytotoxic T cells into lesions (3/4 patients with pre- and post-biopsies available) ✓ 3 of 6 patients in the safety lead-in cohort showed clinical response after 6 months (50% response rate, RECIST 1.1)
  • 14. www.targovax.com 14 Mesothelioma trial: Safety cohort patient split and clinical responses ONCOS TG Safety lead-in cohort 6 patients 1st line treatment 3 patients 2nd / 3rd line treatment 3 patients o Safety & tolerability o Immune activation o Clinical reponse rate Clinical response 2 patients Progression of disease 1 patient Clinical response 1 patient Progression of disease 2 patients o No previous chemo- therapy treatment o Previous chemo- therapy treatment o 1 partial reponse o 1 stable disease o 1 stable disease
  • 15. www.targovax.com 15 ONCOS-102 opportunity in malignant mesothelioma ONCOS TG o ONCOS-102 has the opportunity to become standard of care in 1st line treatment - In combination with current SoC chemotherapy o ONCOS-102 has orphan drug designation in mesothelioma in both the US and EU - 7 year market exclusivity in the US and 10 years in the EU o Few direct IO competitors in current development - CPIs are potential combination therapies with ONCOS, rather than competitors - No/few competing viruses and vaccines in clinical development Rationale for ONCOS-102 opportunity in mesothelioma
  • 16. www.targovax.com Agenda 16 1Q 2018 Highlights ONCOS-102 Mesothelioma update 1Q 2018 Financials
  • 17. www.targovax.com Targovax has a sound financial position, with cash to complete the planned clinical program well into 2019 Operations Cash end of Q1 NOK 229m USD 29m Mar 31st 2018 Net cash flow NOK -32m USD -4m Total Q1 Annual run rate NOK 113m USD 15m Last four quarters 17 The share OSE: TRVX Market Cap NOK 850m USD ~110m At share price NOK ~16 Daily turnover NOK 3m USD 0.4m Rolling 6 month avg. Analyst coverage DNB, ABG Sundal Collier, Arctic, Redeye, Norske Aksjeanalyser, Edison CORPORATE
  • 18. www.targovax.com The increase in run rate over the last 12 months is driven by higher spend on R&D 18 CORPORATE Other45.6m 39% 70.0m 61%R&D Other 45.6m 36% 80.8m 64% R&D R&D spend (NOK million) 2Q16 - 1Q17 2Q17 - 1Q18 Total: 115.6m Total: 126.4m (Amounts in NOK thousands) Total of which R&D Total of which R&D External R&D expenses 43.0 43.0 48.0 48.0 Payroll and related expenses 47.1 25.8 52.8 31.8 Other operating expenses 25.5 1.3 25.6 1.0 Total operating expenses 115.6 70.0 126.4 80.8 12 months rolling 2Q 2017 - 1Q 20182Q 2016 - 1Q 2017
  • 19. www.targovax.com Targovax is listed on the Oslo Stock Exchange, and included in the OSEBX index as of December 2017 19 TRVX share turnover (% of share capital, rolling 12 month) • NOK ~850 m market cap • NOK 3m avg. daily turnover in last 6 months • NOK 213m total turnover in 1Q • 190k shares avg. daily volume in 1Q • >4,100 owners • 52.6m shares* (57.4 fully diluted) CORPORATE
  • 20. www.targovax.com News flow – Multiple near-term value inflection pointsInitiationRead-outsCorp 20 2018 2019 H1 H2 H1 H2 2020 H1 2017 H1 H2 Pancreas full data Prostate ph I Mesothl. interim data Melanoma interim data Pancreas ph II Melanoma full data Colorectal full data Mesothl. full data Pancreas 2yr survival Melanoma safety data Mesothl. safety data Colorectal ph Ib Colorectal safety data OvCa / Col. ph l Melanoma ph l/ll OSE main list OSEBX index
  • 21. www.targovax.com 21 Broad clinical program TG ONCOS ✓ Six shots on goal ✓ Several upcoming data points ✓ Unique approach for targeting RAS mutations ✓ Potential to benefit up to 1/3 of all cancer patients ✓ Demonstrated ability to increase T-cell count ✓ Early signal of efficacy in difficult-to-treat tumors Arming the patient’s immune system to fight cancer