The KTX-1001 Program
About the target
Approximately 15-20% of multiple myeloma patients are identified as having a genetic translocation between chromosomes 4 and 14 that generates two abnormal chromosomes which leads to increased expression of the multiple myeloma SET domain (MMSET) gene, and consequently the MMSET protein. Elevated MMSET drives an epigenetic program that converts normal plasma cells into cancerous multiple myeloma cells.
Treatment options for patients with multiple myeloma have improved significantly within the past decade. However, a disparity remains for patients with poor prognostic factors, including a t(4;14) translocation.
About Our Candidate
KTX-1001 is an orally bioavailable small molecule and a selective inhibitor of MMSET, which is overexpressed in multiple myeloma patients who harbor the genetic t(4;14) translocation.
K36 Therapeutics developed KTX-1001 to provide a targeted therapy that specifically addresses the underlying cause of cancer for t(4;14) multiple myeloma patients. KTX-1001 is the first therapeutic candidate to enter the clinic that directly targets overexpression of MMSET.