FDA, EMA Clear Global Phase III Trial of Lisaftoclax for Higher-Risk MDS

Ascentage Pharma has received clearance from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to conduct a global registrational Phase III study, GLORA-4, evaluating lisaftoclax (APG-2575), a novel BCL-2 inhibitor, in combination with azacitidine for first-line treatment of newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS). This trial represents a significant advancement as lisaftoclax is currently the only BCL-2 inhibitor in a registrational Phase III trial for higher-risk MDS globally and could fill a critical unmet medical need by potentially becoming the first targeted therapy approved for this indication since hypomethylating agents' introduction. HR-MDS primarily affects older adults and has a poor prognosis with limited treatment options, mainly hypomethylating agents and allogeneic hematopoietic stem cell transplantation. Early studies of lisaftoclax have shown promising clinical benefits and tolerability, and the trial is enrolling patients internationally to expedite potential market authorization. This development marks a major milestone in global clinical efforts to improve outcomes for patients with higher-risk MDS, offering hope for a new therapeutic option that could fundamentally reshape the treatment landscape. Ascentage Pharma is a commercial-stage biopharmaceutical company focused on novel therapies addressing unmet needs in cancer and related diseases.