Targovax AS

Arming the patient’s immune system to fight cancer

Targovax – an innovation driven specialist in immuno-oncology

Targovax is a clinical stage immuno-oncology company dedicated to the development of highly targeted immunotherapies for cancer

Targovax is developing two complementary and highly targeted approaches in immuno-oncology:

Targovax2 a virus-based immunotherapy platform based on engineered oncolytic viruses armed with potent immune stimulating transgenes for patients with solid tumors.

a peptide-based immunotherapy platform for patients with difficult to treat RAS-mutated cancers


Two unique and complementary technologies – designed to harness the patient’s own immune system



The immunotherapy market – forecast for significant growth over next decade

Immunotherapy is now an important additional treatment modality in the fight against many types of cancer and represents one of the
fastest growing and most promising biotech segments today. The percentage of immuno-oncology addressable cancers is expected to
increase to at least 60% by 2023 due to combination strategies.
According to a report by Citi Research, the immunotherapy market will experience considerable growth over the next years, increasing
from USD 1.1 billion in 2012 to an estimated USD 35 billion in 2023. According to a report by Decision Resources Group, the market
will reach USB 13.3 billion in 2023. This is a lower number than Citi Research’s estimate, but altogether, significant growth is expected.

The Targovax Pipeline – differentiated assets with orphan drug designations


* Indication where orphan drug designation has been granted

ONCOS-102 – Promising early clinical data with virus-based immunotherapy

ONCOS-102 is Targovax’s lead adenovirus-based pipeline product and the oncolytic virus with the most comprehensively mapped
mechanism of action as well as the first to show induction of tumor-specific CD8+ killer T-cells.
ONCOS-102 has demonstrated encouraging Phase I efficacy data in a challenging patient population. In its Phase I clinical trial in
heavily-pretreated patients with multiple types of solid tumors, ONCOS-102 demonstrated a stable disease (SD) rate of 40% in 10
evaluable patients.

Multiple immunotherapeutic mechanisms: Phase I data showed a dramatic increase in tumor-infiltrating cytotoxic CD8+ T cells,
innate immune system activation, and cancer specific CD8+ T-cells in blood indicating systemic activation of the immune system.
Both the increase of innate immune cell infiltration and the increase of CD8+ T cells were correlated with overall survival.

Encouraging safety profile (in >100 patient safety database): A five-year compassionate use program has demonstrated an
encouraging safety profile in 115 ONCOS-102-treated patients. Adverse events were mostly of grade 1 or 2 (mild flu-like symptoms).
The ONCOS-102 Phase I study did not identify a dose-limiting toxicity.

Synergistic potential in combination usage. Preclinical data has shown synergistic effects between ONCOS-102 and chemotherapy.
There is also a compelling mechanistic rationale for combining ONCOS-102 with immune checkpoint inhibitors, which would
benefit from ONCOS-102’s innate immune system activation, co-stimulatory effects and T cell activation, yielding a complete
immunotherapeutic arsenal.

TG01 – Encouraging early clinical data with RAS-targeted immunotherapy

TG01 is Targovax’s lead RAS immunotherapy product and the only RAS-specific cancer vaccine in development. It is targeted at the
difficult to treat RAS mutations found in more than 85% of pancreatic cancer, 50% of colorectal cancer and 20-30% of all cancers.
Results of the Phase I part of a Phase I/II trial of TG01 and gemcitabine as adjuvant therapy for treating patients with resected
adenocarcinoma of the pancreas have demonstrated:

• TG01 induced immune responses in nearly all patients

• TG01 had a favorable safety and tolerability profile.

• 14 out of 15 evaluable patients were still alive after one year.
(Contrasting favorably with historical 1-year overall survival rate of only 75%)

Future clinical development plans – focused on combination therapy

The Targovax pipeline is built on a robust knowledge of the immunotherapy area and its unique technologies. It includes a number of
product candidates targeted at a wide range of cancer types, such as mesothelioma, melanoma, ovarian, prostate and pancreatic cancers.
Targovax is committed to a development plan designed to optimize patient response and maximize efficacy. Following initial promising
data in its Phase 1 monotherapy studies, it has now expanded its development to focus on combination studies.
Several reports, including the Immunotherapies Report by Decision Resources highlight the future role of immune checkpoint inhibitors
in combination with current cancer treatment regimens or other immunotherapies rather than as monotherapy.


1 http://www.cancer.org/treatment/treatmentsandsideeffects/treatmenttypes/immunotherapy/immunotherapy-what-is-immunotherapy (10 July 2015)
2 Citi Research 2013
3 Decision Resource Special Report – Cancer Immunotherapies May 2015, projections based on seven major pharmaceutical markets; United States, France, Germany,
Italy, Spain, United Kingdom, and Japan


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