KTX-2001

KTX-2001 is an oral, first-in-class, small molecule inhibitor that selectively targets the nuclear receptor-binding SET domain protein 2 (NSD2), a histone methyltransferase implicated in the oncogenic transcriptional regulation of cancers such as metastatic castration-resistant prostate cancer (mCRPC)[1][2][4][10]. By inhibiting NSD2-mediated methylation of histone H3 at lysine 36 (H3K36me2), KTX-2001 disrupts epigenetic reprogramming, suppresses oncogenic gene expression, and impedes tumor cell proliferation and transcriptional plasticity. These actions aim to normalize chromatin structure and restore sensitivity to treatment in resistant cancer cells. KTX-2001 is under clinical development and currently being assessed in a phase 1 clinical trial (STRIKE-001) both as monotherapy and in combination with darolutamide in patients with mCRPC[1][2][4][10].