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Advancements in Myelodysplastic Syndrome Therapy: Lisaftoclax by Ascentage Pharma

At the 66th American Society of Hematology (ASH) Annual Meeting held in San Diego, California, Ascentage Pharma, a prominent biopharmaceutical entity, presented updated clinical data for its investigational drug, lisaftoclax (APG-2575). This novel Bcl-2 inhibitor aims to address significant medical needs primarily for malignancies, specifically in the treatment of myelodysplastic syndrome (MDS).

The ASH Annual Meeting serves as a critical platform for hematology professionals worldwide, facilitating the exchange of groundbreaking research and advancements in clinical therapies. Ascentage Pharma’s latest study was conducted alongside azacitidine (AZA), a standard treatment for MDS, and demonstrated considerable clinical efficacy and manageable safety, reaffirming the drug's therapeutic potential in treating higher-risk MDS.

Key Highlights of the Study

The data unveiled exciting outcomes, indicating an overall response rate (ORR) of an impressive 75% for patients with treatment naïve (TN) or relapsed/refractory (R/R) MDS. This is a significant finding, given the limited advancement in treatment options for higher-risk MDS in recent years. Notably, no patients withdrew from the study due to intolerable side effects, underscoring the favorable safety profile of the combination therapy.

Professor Jie Jin, the principal investigator from the First Affiliated Hospital, Zhejiang University School of Medicine, emphasized the historical challenges faced in treating higher-risk MDS, stating, “The current standard treatment outcomes have been disappointing. Our results with lisaftoclax in conjunction with AZA suggest a potential new standard for treating MDS.”

In an intriguing comment, Professor Huafeng Wang, who presented the study results, highlighted that many patients suffering from higher-risk MDS struggle with inadequate marrow function, making them less tolerant to traditional therapies. The combination of lisaftoclax and AZA has showcased manageable safety, effectively balancing the need for potent treatment while minimizing impacts on bone marrow function.

The reported data indicated a low rate of treatment-related infections, primarily mild in nature, and minimal early treatment mortality, illustrating progress in overcoming some of the most considerable challenges facing this patient population.

Ongoing and Future Commitment

Dr. Yifan Zhai, Chief Medical Officer at Ascentage Pharma, expressed optimism about the updated efficacy and safety data of lisaftoclax, reaffirming the drug's promising role in treating MDS. He stated that a global registrational Phase III study is currently ongoing, and an NDA for lisaftoclax in chronic lymphocytic leukemia/small lymphocytic lymphoma has also received Priority Review designation in China.

Ascentage Pharma's commitment to addressing unmet clinical needs extends beyond MDS. The company is focused on expediting clinical development programs for its key drug candidates, aspiring to deliver safe and effective therapies globally.

Data Presentation Details

The findings were presented in a Poster Presentation format (#3202) during the Myelodysplastic Syndromes Clinical and Epidemiological Poster II session. The study enrolled patients classified with higher-risk MDS, employing a regimen that combined specific doses of lisaftoclax (ranging from 400 to 800 mg) with azacitidine for a determined duration. The early data revealed a promising profile for the combined therapy, illustrating potential new avenues for treatment in this challenging area.

In conclusion, the findings presented at ASH 2024 highlight the emerging role of the investigational drug, lisaftoclax, as a potential cornerstone in the treatment regimen for higher-risk MDS patients, advocating for its further development and clinical application.

Lisaftoclax remains an investigational medication and has not yet received approval in any region.