Karyopharm doses patients in two KPT-330 Phase 1 trials
Karyopharm Therapeutics has dosed patients with oral KPT-330 in its two Phase 1 first-in-human trials.
KPT-330, a selective inhibitor of nuclear export (SINE) chromosome region maintenance protein 1(CRM1) antagonist, is a mediator of nuclear export of p53, p73, pRb, FOXO, p21, p27, BRACA1, the endogenous inhibitor of Nuclear Factor kB (NF-kB), and other tumor suppressor and growth regulatory proteins.
The first study includes patients with solid tumors whose disease has progressed after at least one prior therapy for metastatic disease (NCT01607905), while the second study includes patients with chronic lymphocytic leukemia, non-Hodgkin's lymphoma, multiple myeloma, and Waldenstrom's macroglobulinemia whose disease has relapsed after standard therapies (NCT01607892).
The primary endpoints of both studies are to determine the safety and tolerability profile and the maximum tolerated dose of KPT-330 given orally 2-3 times per week.
Karyopharm founder and chief scientific officer and research and development head Sharon Shacham said the initiation of the two Phase 1 trials with KPT-330 is a key milestone since the closing of the company's Series A funding and the initiation of its operations in late 2010.
"This is the first oral SINE to ever enter human studies, and we are eager to assess the potential of our drug candidate across a wide variety of cancers," Shacham added.