AbbVie's Bcl-2 Inhibitor Fails in Phase III Trial for High-Risk MDS

▎Armstrong

On June 16, 2025, AbbVie announced that the Phase III clinical trial VERONA of Bcl-2 inhibitor Venetoclax + Azacitidine in the first-line treatment of high-risk myelodysplastic syndrome (HR-MDS) did not meet the primary endpoint of OS, with an HR of 0.908 and a p-value of 0.3772.

Venclexta is the world's first Bcl-2 inhibitor, approved for use in CLL/SLL and AML indications.

HR-MDS is a core indication for which Venclexta is expanding, but this Phase III clinical trial has ended in failure.

Summary

HR-MDS still has serious unmet clinical needs, with the FIC CD47 antibody and Bcl-2 inhibitor both having failed. Nevertheless, next-generation products still hold promise for success in HR-MDS, such as Akeso's new-generation CD47 antibody and Ascentage Pharma's new-generation Bcl-2 inhibitor.