CASI Pharmaceuticals executed an Assignment Agreement with Cleave Therapeutics, Inc., under which CASI obtained all rights and global intellectual property related to CB-5339, a novel VCP/p97 inhibitor, as well as all remaining CB-5339 drug substance and drug product. Additionally, CASI will assume responsibility of the CB-5339 Investigational New Drug application.

CB-5339 is an oral, second-generation, small molecule valosin-containing protein (VCP)/p97 inhibitor, designed to disrupt protein homeostasis, DNA damage response and other cellular stress pathways. In a Phase 1 clinical trial in patients with acute myeloid leukemia and myelodysplastic syndrome, the drug was well tolerated in 55 patients and demonstrated signs of clinical activity. Two patients remain on CB-5339 under compassionate use protocols at two leading U.S. cancer centers.

Wei-Wu He, Ph.D., CASI’s Chairman and CEO said, “CB-5339 is a first in class small molecule drug candidate that represents a promising opportunity to selectively target VCP/p97 in various malignancies. We are encouraged by the early clinical data in the phase 1 study of CB-5339 in patients with AML and MDS. CB-5339 is a complementary addition to our growing portfolio of approved and investigational therapies in oncology.