1521O A phase II biomarker-driven study evaluating the clinical efficacy of an MDM2 inhibitor, milademetan, in patients with intimal sarcoma, an ultra-rare cancer with highly life-threatening unmet medical needs (NCCH1806/MK004)

Abstract

Intimal sarcoma is an ultra-rare, high-grade malignant neoplasm arising in the intima of large blood vessels, most frequently the pulmonary arteries and aorta. As murine double minute 2 (MDM2) amplification is found in over 70% of intimal sarcomas, inhibition of MDM2 could provide clinical benefit in this patient population. This study was conducted to evaluate the activity of milademetan, a novel specific small-molecule inhibitor of MDM2 in intimal sarcoma patients as a sub-study under the nationwide large registry for rare cancers in Japan (MASTERKEY Project).