OX401 is the second candidate utilizing Valerio Therapeutics' proprietary platform of decoy agonists, platON™.

OX401 was designed by capitalizing on Valerio Therapeutics' expertise of oligonucleotides acting as decoy agonists and exhibits very original properties.

  • During optimization, OX401 has demonstrated that it inhibited the DNA Damage Response by acting on PARP proteins.
  • In parallel, OX401 activated the STING pathway, a recent and promising field of research in immuno-oncology, which makes it amenable to combinations with immuno- oncology agents such as checkpoint inhibitors.

While the clinical relevance of PARP inhibitors is now well-established, this class still has a number of limiting factors, particularly the relatively rapid onset of resistance. Its decoy agonist mechanism of action positions OX401 as a next-generation PARP inhibitor that should not present these limitations and instead offer a lack of acquired resistance and more specificity to cancer cells.

OX401 was also developed to induce a strong immune response through the activation of the STING pathway, an area of significant interest in immuno-oncology. However, current molecules have experienced challenges, notably in terms of toxicity. OX401 is based on the same decoy agonist mechanism as AsiDNA™, Valerio Therapeutics' first-in-class DNA repair inhibitor, which showed good tolerance in the DRIIV-1 Phase 1 study, and should trigger a rapid and significant inducing effect of innate immunity against tumor cells.

OX401 is currently undergoing proof-of-concept preclinical studies, alone and in combination with cancer immunotherapies.

Patents has been filed to protect Valerio Therapeutics' intellectual property rights on OX401 , alone and in combination with cancer immunotherapies.

PARP & DDR Inhibitors Summit 2020

Download presentation: Introducing OX401, a Next Generation PARP Inhibitor Able to Exploit Metabolic Vulnerabilities of Cancer Cells and Inducing a Potent STING Response

AACR Virtual meeting  2021

Download abstract : A new generation of PARP interfering drug candidates for cancer treatment