BEIJING, China (July 20, 2023) CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, today announced the execution of the Assignment Agreement (the “Agreement”) with Cleave Therapeutics, Inc. (“Cleave”), pursuant to which CASI obtained all rights and global intellectual property rights 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 United States (“US”) CB-5339 Investigational New Drug (“IND”) 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 US cancer centers.  

Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, “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.”

About Cleave Therapeutics

Cleave was founded in 2011 with scientific co-founders Raymond Deshaies, PhD, Seth Cohen, PhD, and Francesco Parlati, PhD. The company has focused on novel biological mechanisms and pathways in the AAA (ATPases Associated with diverse cellular Activities) family of ATPases to address unmet needs in oncology and neurodegeneration. Cleave investors include 5AM Ventures, Bristol-Myers Squibb, Orbimed, U.S. Venture Partners, Arcus Ventures, Astellas Venture Management, and Osage University Partners.