ONCOS-102 is an experimental adenovirus-based cancer therapy. Targovax is developing it for treating patients with solid tumors.

How does ONCOS-102 work?

Researchers engineered the treatment to replicate inside cancer cells only, leaving healthy cells unharmed. They inserted into the virus genome a gene coding for GM-CSF (granulocyte-macrophage-colony-stimulating factor) that tumor cells will make during replication of the virus. GM-CSF is a protein that stimulates the immune system.

ONCOS-102 particles bind and enter tumor cells specifically, resulting in their death. When the tumor cells die, they release tumor antigens or molecules that are capable of inducing an immune response, as well as new ONCOS-102 particles. This, together with the production of the co-stimulatory molecule GM-CSF, functions as a danger signal that the immune system recognizes and leads to the death of more tumor cells.

ONCOS-102 in clinical trials

A Phase 1 clinical trial (NCT01598129) assessed the safety, tolerability, and recommended dose of ONCOS-102 in 12 patients with advanced cancers. Results of the trial that were published in the Journal for ImmunoTherapy of Cancer, revealed that treatment with ONCOS-102 in conjunction with low-dose Cytoxan (cyclophosphamide injection) was safe and well-tolerated at the tested doses. The treatment also was able to induce a tumor-specific immune response both at the injection site and throughout the body. More importantly, anti-tumor immunity came with signals of clinical benefit.

Ongoing clinical trials

A Phase 1/2 study (NCT02879669) is evaluating the safety, immune activation, and clinical response of ONCOS-102 in combination with Alimta (pemetrexed) and Platinol (cisplatin) in 31 patients with malignant mesothelioma. In the Phase 1 portion of the trial, researchers are evaluating the safety of ONCOS-102 and Alimta/Platinol. In the Phase 2 portion, patients receive either ONCOS-102 plus Alimta/Platinol, or Alimta/Platinol alone. Data from this study indicate that patients tolerate ONCOS-102 well. Moreover, the addition of ONCOS-102 elevates immune system activity and extends the duration after treatment in which the disease does not worsen.

A Phase 1 trial (NCT03003676) is evaluating ONCOS-102 followed by Keytruda (pembrolizumab) in 12 patients with advanced melanoma whose disease has progressed despite prior treatment. The main goal of the study is to determine the safety of the sequential treatment. Secondary goals include progression-free survival, objective response rate, and change in the size of individual lesions. Researchers will also evaluate changes in blood and tumor immune parameters. Early results from the study suggested that patients tolerate ONCOS-102 well. Moreover, the treatment may induce an immune response, based on data from four patients treated to date.

Finally, a Phase 1/2 study (NCT02963831) is assessing the safety and anti-tumor activity of ONCOS-102 in combination with imfinzi (durvalumab) in people with advanced peritoneal malignancies. The study is currently enrolling up to 78 participants in the U.S.

Other details

The most common adverse side effects of ONCOS-102 are flu-like symptoms and fever.


Last updated: May 7, 2020


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