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