Karyopharm Initiates Phase 1 Trial Of Selinexor In Pediatric Patients

(RTTNews) - Karyopharm Therapeutics Inc. (KPTI) announced the initiation of its first pediatric clinical study, a Phase 1 trial of its novel, oral Selective Inhibitor of Nuclear Export or SINE compound Selinexor (KPT-330) in pediatric patients.

The trial will enroll up to 28 children with relapsed or refractory acute lymphoblastic leukemia or (ALL) or acute myeloblastic leukemia or AML.

Selinexor (KPT-330) is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. Selinexor functions by binding with the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus, which subsequently reinitiates and amplifies their tumor suppressor function. This is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells.

To date, over 290 patients have been treated with Selinexor in Phase 1 trials in advanced hematologic malignancies and solid tumors. Additional Phase 1 and Phase 2 studies are ongoing or currently planned and three registration-directed clinical trials in hematological indications are expected to begin enrollment during 2014.