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Pipeline

platON™ is Valerio Therapeutics’ proprietary chemistry platform of oligonucleotides acting as decoy agonists, which generates new innovative compounds that broaden the Company’s product pipeline.

AsiDNA™, the first compound sourced from platON™, is a first-in-class, highly differentiated DNA Damage Response (DDR) inhibitor based on a decoy mechanism with an agonist effect, acting upstream of multiple DDR pathways. AsiDNA™ has the ability to abrogate tumor resistance to PARP inhibitors regardless of the genetic mutation status. AsiDNA™ has also shown a strong synergy with other tumor DNA-damaging agents such as chemotherapy, radiotherapy and PARP inhibitors. Thanks to a good safety profile, AsiDNA™ is particularly amenable to combination treatment, a standard of care in aggressive or resistant cancers. AsiDNA™ was evaluated in the DRIIV-1b phase 1b study in combination with chemotherapy and is currently evaluated in the REVOCAN phase 1b/2 study in combination with PARP inhibitors in relapsed ovarian cancer.

OX401 is the second drug candidate from platON™ acting on both the DNA Damage Response and the activation of immune response, without inducing resistance. In vivo, OX401 has shown a potent antitumoral activity and a strong immune response activation. Optimization of this new molecule is in progress.

OX425, the second compound from platON, is a novel DDR Decoy Agonist with high antitumor activity. It also mediates multiple immunostimulatory effects by activating the STING pathway. OX425 is currently in IND-enabling preclinical development.