KTX-1001 is an oral, first-in-class, selective MMSET catalytic inhibitor that suppresses H3k36me2 in patients with relapsed and refractory multiple myeloma (RRMM) with a focus on patients with the t(4;14 ) genetic translocation. This study will evaluate the safety, tolerability, and preliminary efficacy of KTX-1001.

We developed KTX-1001 to provide a targeted therapy that specifically addresses the underlying cause of cancer for these multiple myeloma patients. KTX-1001 is the first therapeutic agent to enter the clinic that directly targets overexpression of MMSET.