Highlights 

  • Lisaftoclax shows a 31.8% ORR in venetoclax-exposed R/R AML/MPAL patients. 
  • Newly diagnosed high-risk MDS/CMML patients achieved an 80% ORR. 
  • No dose-limiting toxicities were reported across all 103 patients. 
  • Combination with azacitidine demonstrates encouraging anti-tumor activity. 
  • Median OS reached 7.6 months in R/R AML/MPAL patients. 

Ascentage Pharma Group International (NASDAQ:AAPG) presented new clinical results from its Phase Ib/II trial evaluating Lisaftoclax (APG-2575) in combination with azacitidine during the 67th American Society of Hematology Annual Meeting. The trial focused on patients with newly diagnosed or prior venetoclax-exposed myeloid malignancies, including acute myeloid leukemia and higher-risk myelodysplastic syndromes (HR MDS). 

The study involved 103 enrolled patients as of July 1, 2025. This included 63 individuals diagnosed with AML or mixed-phenotype acute leukemia, and 40 with HR MDS or chronic myelomonocytic leukemia. The trial’s aim was to explore how Lisaftoclax, an oral Bcl-2 selective inhibitor, performs when paired with azacitidine, especially in cases where resistance to venetoclax poses a major treatment challenge. 

Efficacy Signals: Meaningful Responses Across Key Patient Groups 

The data presented at ASH 2025 highlighted meaningful clinical responses. Among 44 evaluable patients with relapsed or refractory AML/MPAL, the overall response rate reached 43.2%, with complete responses achieved in 31.8% of cases. Notably, in the more difficult subset of venetoclax-exposed individuals, the regimen still delivered a 31.8% ORR. 

For newly diagnosed HR MDS/CMML patients, results were also encouraging. In the 15 evaluable patients, the ORR climbed to 80%, with complete responses and marrow CRs evenly split. Median overall survival reached 7.6 months in the relapsed/refractory AML/MPAL cohort and 11.3 months among patients with relapsed/refractory HR MDS/CMML. The OS for newly diagnosed HR MDS/CMML patients had not yet been reached. 

Safety and Clinical Outlook 

The safety profile observed in this study suggests Lisaftoclax has the potential to serve as a viable therapeutic candidate for difficult-to-treat myeloid malignancies. Across all cohorts, no dose-limiting toxicities were identified. The most frequent grade ≥3 treatment-emergent adverse events included neutropenia, febrile neutropenia, thrombocytopenia, and anemia—consistent with expectations in this class of therapies. 

Ascentage Pharma continues advancing four global Phase III registrational programs for Lisaftoclax across multiple indications. With additional ongoing studies, the company is positioning Lisaftoclax as a next-generation therapy that may help close treatment gaps for patients who are resistant or intolerant to existing options. 

Conclusion 

Ascentage Pharma’s latest ASH 2025 data demonstrate encouraging progress for Lisaftoclax as a combination treatment for AML, MDS, and related malignancies. The reported response rates and safety outcomes signal meaningful potential for patients who have limited options, including those with prior venetoclax exposure. Ongoing late-stage trials will further clarify the therapy’s future role in hematologic oncology. 

Ascentage Pharmas’ shares closed at USD 32.88, marking a 7.10% increase from the prior session.