Shots:
The US FDA has granted ODD for eltanexor, a novel oral, SINE compound that inhibits XPO1 leading to the accumulation of tumor suppressor proteins in the cell nucleus to treat MDS
The therapy is currently being evaluated in an ongoing P-I/II study as monothx. or in combination with approved & investigational agents in 119 patients with multiple types of hematologic & solid tumor cancers
In the P-I study, the therapy showed 53% ORR, m-OS (9.9mos.) in patients with HMA-refractory MDS. In preclinical models, the therapy has a broad therapeutic window with minimal penetration of the blood-brain barrier & has the potential for another SINE compound to treat cancer
Ref: PR Newswire | Image: Karyopharm
