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.