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P707 project

Treatment with PARP inhibitors affects DNA repair and is clinically effective in patients with defects in the BRCA gene, due to the simultaneous impairment of two complementary DNA repair pathways. NMS-293 is a second generation PARP inhibitor that differentiates from other approved or advanced molecules in its unique selectivity for PARP1 vs PARP2 enzymes and low DNA trapping activity, both features potentially linked to lower haematological toxicity and higher potential for combination with other drugs. It also has a superior ability to penetrate the blood brain barrier, a very important feature supporting its utilization in CNS tumors and brain metastases. Clinical development of the drug has recently been initiated in breast, ovarian, prostate and pancreatic tumors, enriched for tumors harboring a BRCA mutation. The high brain penetration observed for NMS-293 in preclinical studies provides a strong rationale for investigating the potential efficacy of this agent against CNS tumors. For this reason, a second study in combination with standard of care in glioblastoma is being planned.