Ascentage Pharma Gets FDA and EMA Approval for Phase 3 Lisaftoclax Study in High-Risk MDS

Ascentage’s Phase 3 Study of Lisaftoclax

Announcement

Ascentage Pharma, a leading marketizing level merger biopharmaceutical company involved in the development, exploration, and marketing of numerous different therapies to identify required medical needs in cancer, has received approval from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to start with the GLORA-4 study (NCT06641414). A worldwide registrational Phase 3 study of lisaftoclax (APG-2575), an exclusive Bcl-2 inhibitor, in combination with azacitidine (AZA) for newly diagnosed high-risk myelodysplastic syndrome (HR-MDS) patients.

This represents all about the second registrational phase 3 study of lisaftoclax, a cleared study by both the EMA and FDA. The GLORA-4 study balanced enrollment of patients at participating centers in various countries at one time to leverage the drug’s intention to achieve market authorization on time. Currently, lisaftoclax is the only BCL-2 inhibitor being studied in a registrational phase 3 trial in high-risk MDS worldwide. On successful completion of the study, it may turn out to be a long-term potential treatment in high-risk MDS, forever printing another crucial milestone in the clinical development of lisaftoclax globally.

Past Clinical Record

Lastly, ascentage officially announced the clinical data of lisaftoclax in the merger with AZA in treatment-naïve (TN) MDS at the 2024 American Society of Hematology (ASH) annual meeting and the American Society of Clinical Oncology (ASCO) annual meeting 2025. The data showcased an ORR of 75% which is higher than HMAs alone. This indicates the benefit to the clinical development of the merger regimen. The merger also showcased a positive safety profile with fewer consequences of extreme hematologic toxicities and infections related to neutropenia. Additionally, the number of patients requesting dose adjustments was lower, and there was no such treatment regarding mortalities in the 60 days.

Views and Statements from Company Leaders

Chief medical officer of Ascentage Pharma, Dr. Yifan Zhai, said, “We lack targeted therapies for the risky MDS patients that demonstrate a vast unmet clinical need globally. For now, hypomethylating agents (HMA) and allogeneic hematopoietic stem cell transplantation are still the treatment alternative for MDS in the first place. The clearance to the glora-4 study has uncovered potential ways for lisaftoclax.”

MD, and academician of the Chinese Academy of Engineering, director of the Institute of Hematology at Peking University, and the director of the department of hematology at Peking University People’s Hospital, and a global co-leading principal investigator of the study, Prof. Xiaojun Huang said, “Despite of the prominent advancement the treatment of hematologic malignancies risky MDS is still a crucial clinical challenge because of various factors.”