AsiDNA is an experimental therapy being developed by Onxeo as a cancer treatment. Originally being tested in Phase 1 clinical trials for melanoma and other solid tumors, it now also will be assessed for its effectiveness in women with relapsed ovarian cancer.

Onxeo has announced that it will be working with Gustave Roussy, the leading European cancer center, to conduct a Phase 1b/2 study to determine the effect of AsiDNA on acquired resistance to the DNA damage repair treatment niraparib in ovarian cancer patients.

The study should begin in early 2020, with preliminary results expected by the end of the year.

How AsiDNA works

AsiDNA contains an artificial DNA fragment that mimics a particular type of DNA damage called a double-strand break. By mimicking DNA damage, the therapy molecule attracts all of the repair machinery of the cell that acts to repair DNA damage onto itself. As a result, real DNA damage in the tumor cells is not repaired. Because cancerous cells divide very quickly, unrepaired DNA damage rapidly accumulates in them. Ultimately, this cumulative damage causes the cancer cells to die.

Healthy cells that receive the treatment will halt cell division as long as the compound is present. Therefore, the treatment should not damage healthy cells.

Because AsiDNA affects all of the components of DNA repair machinery, it should be harder for cancerous cells to develop resistance to the compound.

AsiDNA in clinical trials

The experimental treatment was evaluated in combination with radiotherapy in a Phase 1 clinical trial called the DRIIM study. A total of 23 patients with skin metastases from melanoma were treated, with AsiDNA administered directly into the tumors.

The results of the study were published in the British Journal of Cancer. All patients were evaluated for safety throughout the study. Dose-limiting toxicity was not observed and the maximum-tolerated dose was not reached. The most frequent adverse events were mild reactions at the site of injection. A total of 76 lesions in 21 patients were assessed for efficacy. An objective, or measurable response was observed in 59% of lesions, which included 23 complete responses (30% of lesions). A complete response is the disappearance of all signs of cancer following treatment.

A second Phase 1 trial (NCT03579628) called DRIIV-1 is evaluating AsiDNA in treating solid tumors. That study is still recruiting patients in Belgium and France.

The preliminary results from 22 participants were presented at the International Conference on Molecular Targets and Cancer Therapeutics, held last year in Boston. The first part of the study established the active dose that will be used in future applications and provided further confirmation of the safety profile of the treatment. The second half of the study will assess the safety and efficacy of AsiDNA in combination with carboplatin — a medication that interferes with the growth of cancer cells and slows their spread in the body — with or without paclitaxel. Paclitaxel can lower blood cells that help the body fight infections and help the blood to clot.


Last updated: Feb. 3, 2020


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