Ascentage Pharma presented Phase Ib/II data at the 2025 ASCO Annual Meeting for lisaftoclax, a Bcl-2 inhibitor, combined with azacitidine in patients with treatment-naïve or venetoclax-exposed myeloid malignancies. The study enrolled 103 patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS), highlighting the drug’s antitumor activity and manageable tolerability. Importantly, the study demonstrated responses in venetoclax-refractory patients, differentiating lisaftoclax from other Bcl-2 inhibitors.
This data is particularly relevant for patients with AML and MDS, especially those who have developed resistance to venetoclax, as it offers a potential new treatment option. The positive results in venetoclax-refractory patients suggest lisaftoclax may address a significant unmet need in this patient population, where treatment options are limited. This could potentially change the treatment landscape for these aggressive blood cancers.
The study showed an overall response rate (ORR) of 31.8% in venetoclax-refractory patients with R/R AML/MPAL, with responses observed even in patients with TP53 mutations and complex karyotypes. In newly diagnosed AML/MPAL patients, the ORR was 83.3%. For MDS/CMML, ORR was 80% in treatment-naïve patients and 50% in relapsed/refractory patients. The combination therapy was generally well-tolerated, with primarily hematologic adverse events.
These positive Phase Ib/II results reinforce the potential of lisaftoclax as a promising therapeutic option for myeloid malignancies. The data supports further clinical development, including ongoing Phase III trials, and suggests lisaftoclax could become a key treatment for AML and MDS, particularly in patients who have failed prior therapy with venetoclax. This could significantly improve outcomes for these patients and solidify Ascentage Pharma’s position in the hematology-oncology field.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
