About CB-5339 (VCP/p97 inhibitor)

Investigational Innovative Drug Candidate In-Licensed for Greater China Region

In 2021, CASI acquired CB-5339, a first-in-class VCP/p97 inhibitor from Cleave Therapeutics for the Greater China market. CB-5339 represents a promising new agent for selectively targeting VCP/p97 in cancers and is a complementary addition to CASI’s pipeline of hematology-oncology assets.

VCP/p97 inhibitors exploit molecular features that define cancer cell growth and metabolism. VCP/p97 plays a critical role in protein homeostasis processes such as endoplasmic reticulum associated degradation (ERAD) and chromatin-associated degradation (CAD), as well as the DNA damage response (DDR). These key cellular stress pathways are known to represent sensitivities critical to cancer cell survival. Cellular stress provides an attractive means of targeting non-oncogene addiction as a way to combat tumor heterogeneity and emerging resistance to targeted therapies. Such an approach has the added benefit of broad applicability, not dependent on a single driver mutation and potential efficacy across cancer types.

Clinical Development Status

CASI intends to advance development plans in China. CB-5339 CTA application for the multiple myeloma indication is in preparation after receiving an acceptance letter for the CB-5339 IND package from the China Center of Drug Evaluation.

Cleave is responsible for the ex-China development of CB-5339, and is evaluating the molecule in Phase 1 clinical trial in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).