Core Viewpoint - Ascentage Pharma has received FDA and EMA clearance for the GLORA-4 study, a Phase III trial of lisaftoclax in combination with azacitidine for treating higher-risk myelodysplastic syndrome (HR-MDS), marking a significant step towards addressing unmet medical needs in this area [1][2][3] Company Overview - Ascentage Pharma is a global, commercial stage biopharmaceutical company focused on developing novel therapies for cancer [1][11] - The company has a robust pipeline that includes innovative drug candidates targeting key proteins in the apoptotic pathway [12][14] Study Details - The GLORA-4 study is a global, multi-center, randomized, double-blind Phase III trial designed to evaluate the efficacy and safety of lisaftoclax combined with azacitidine compared to placebo plus azacitidine in newly diagnosed adult patients with HR-MDS [3][4] - This study is notable as it is the second registrational Phase III study of lisaftoclax to receive clearance from both the FDA and EMA [2][3] Clinical Need - There is a significant unmet clinical need for targeted therapies in first-line treatment of higher-risk MDS, as current options like hypomethylating agents (HMA) and allogeneic hematopoietic stem cell transplantation (allo-HSCT) have limitations [3][5][9] - The overall response rate (ORR) for HMAs is only 30-40%, with a complete response (CR) rate of 10-17%, highlighting the need for more effective treatments [5][9] Lisaftoclax Profile - Lisaftoclax is a proprietary, orally administered Bcl-2 selective inhibitor that has shown promising clinical benefits and tolerability in earlier studies [6][8] - The drug is already approved in China for chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) patients who have received prior systemic therapy [7][14] Clinical Data - Clinical data presented at major conferences indicated an ORR of 75% for the combination of lisaftoclax and azacitidine in treatment-naïve MDS, significantly higher than HMAs alone [8] - The combination therapy demonstrated a favorable safety profile with low incidence of severe hematologic toxicities [8][9] Global Collaboration - The GLORA-4 trial is being co-led by prominent researchers from leading institutions, including The University of Texas MD Anderson Cancer Center and Peking University [3][4]
Ascentage Pharma Announces Global Registrational Phase III Study of Lisaftoclax for First-line Treatment of Patients with Higher-Risk Myelodysplastic Syndrome Cleared by US FDA and EMA