Onxeo Announces Positive Preclinical Proof of Concept Results Confirming AsiDNA™ Activity via Systemic Administration

Strong data confirms near-term plan to initiate clinical trial for AsiDNA™, Onxeo’s breakthrough DNA Repair Inhibitor

Paris (France), July 05 2017 – 07h30 am CEST – Onxeo S.A. (Euronext Paris, NASDAQ Copenhagen: ONXEO), (“Onxeo” or the “Company”), a clinical-stage biotechnology company specializing in the development of innovative drugs for the treatment of orphan diseases, in particular in oncology, today announced positive preclinical proof of concept results confirming the activity via systemic (intravenous, IV) administration of AsiDNA™, the company’s first-in-class DNA repair inhibitor.

A first phase I trial (DRIMM[1]) of AsiDNA™ via local administration in melanoma previously demonstrated good tolerance and a beneficial safety profile, as well as a strong signal of efficacy. The objective of these most recent preclinical studies was to show that AsiDNA™ is also effective when administered via an IV route, which would open a wide potential of tumor types for treatment with AsiDNA™.

The generated data confirms the activity of AsiDNA™ administered intravenously, alone and in combination, as shown by the prevention of tumor growth in a murine model of triple negative breast cancer (TNBC). These data also showed a significant synergistic effect of AsiDNA™ when combined with carboplatin[2], a neoadjuvant chemotherapy used in TNBC.

AsiDNA™ via IV administration is therefore an ideal candidate for monotherapy, as well as for combination therapy with genotoxic oncology treatments, such as radio or chemotherapy, or with other DNA repair inhibitors that target a single repair pathway, such as PARP inhibitors[3].

Additionally, pharmacodynamics data generated supports AsiDNA™ unique mechanism of action whereby it acts as a decoy that attracts repair enzymes, breaks the cycle of tumor DNA repair activities and interferes with multiple repair pathways, whilst sparing healthy cells.

The activity of AsiDNA™ via systemic injection was demonstrated to be related to its ability to sequester and hyperactivate two key DNA repair proteins, DNA-PK and PARP, thus preventing their recruitment at damage sites in tumor cells.

“These positive results, combined with robust manufacturing data, provide a solid foundation from which to advance AsiDNA™ towards clinical development via a systemic route of administration,” commented Françoise Bono, Chief Scientific Officer of Onxeo. “Initial data in combination with carboplatin are quite promising and we are beginning subsequent in-vivo testing of AsiDNA™ combined with PARP inhibitors.”

Based on its unique attributes and the preclinical data obtained to date, AsiDNA™ has the potential to address large unmet medical needs in treating aggressive and resistant cancers, such as TNBC and ovary cancer. GlobalData estimates that the TNBC market alone will grow from €0.8 billion in 2016 to €2.1 billion by 2025.

“Achieving the in-vivo preclinical proof of concept of AsiDNA™ activity via systemic administration significantly enhances its potential compared to local administration only and represents a key milestone in the development of this promising product candidate,” concluded Judith Greciet, Chief Executive Officer of Onxeo. “Together with the optimization of the manufacturing process that the team has successfully achieved, we are on track to advance towards the clinical phase and we intend to file the phase I trial submission dossier to regulatory authorities by the end of 2017.”


[1] Le Tourneau C et al. Br J Cancer. 2016 May 24;114(11):1199-205

[2] GeparSixto: von Minckwitz et al. Lancet Oncol 2014; CALGB 40603: Sikov et al. J Clin Oncol 2015.

[3] Jdey W et al. Clin Cancer Res. 2017 Feb 15;23(4):1001-101