On December 11, 2021 Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that the Phase II pivotal study (the APG2575CC201 study) of the company’s novel Bcl-2 selective inhibitor, lisaftoclax (APG-2575), for the treatment of relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (R/R CLL/SLL) has been approved by the Center for Drug Evaluation (CDE) in China (Press release, Ascentage Pharma, DEC 11, 2021, View Source [SID1234596878]).

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APG2575CC201 is a single-arm, open-label, Phase II pivotal study designed to evaluate the efficacy and safety of lisaftoclax, with objective response rate (ORR) as the primary endpoint. Based on existing safety and efficacy data of lisaftoclax, the CDE has agreed that results from the APG2575CC201 study can be used to support the future New Drug Application for the indication of R/R CLL/SLL.

Lisaftoclax is a novel, orally administered Bcl-2 selective inhibitor being developed by Ascentage Pharma. Lisaftoclax is designed to treat a variety of malignancies by selectively blocking Bcl-2 to restore the normal apoptosis process in cancer cells. It is the first China-developed Bcl-2 inhibitor entering clinical development in China. Lisaftoclax is being studied in multiple clinical studies in countries and regions including the U.S., China, Australia, and the European Union, for a range of hematologic malignancies and solid tumors such as CLL/SLL. At the 2021 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, data of China studies of lisaftoclax in the treatment of hematologic malignancies were released for the first time. These data demonstrated lisaftoclax’s favorable tolerability and enormous therapeutic potential, without evidence of any tumor lysis syndrome (TLS). The six patients with CLL who received lisaftoclax at 200 mg or higher doses achieved an ORR of 100% and one case of complete response (CR).

Globally, there are significant unmet medical needs in the treatment of CLL/SLL. Patients with R/R CLL/SLL, especially those who are refractory or resistant to immunotherapies, chemotherapies, and Bruton Tyrosine Kinase (BTK) inhibitors, commonly experience rapid disease progression and currently lack any effective treatment, thus represents an urgent need for an effective novel therapy.

"Lisaftoclax is a key candidate in our apoptosis-targeted pipeline. In earlier studies, it has demonstrated promising efficacy and safety implicating great best-in-class potential. The approval for this Phase II pivotal study makes us even more confident in the developmental and regulatory pathway of lisaftoclax," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "There are urgent unmet medical needs in patients with R/R CLL/SLL who currently lack effective salvage therapies. We will actively advance this Phase II pivotal study of lisaftoclax to hopefully offer a new treatment option to patients around the world, and let more patients with CLL/SLL who now lack any effective treatment benefit from this novel therapeutic as soon as possible."

About Lisaftoclax (APG-2575)
Lisaftoclax is a novel, orally administered small-molecule Bcl-2‒selective inhibitor being developed by Ascentage Pharma to treat hematologic malignancies and solid tumors by selectively blocking antiapoptotic protein Bcl-2 and hence restoring the normal apoptosis process in cancer cells. Lisaftoclax is the first China-developed Bcl-2 inhibitor entering clinical development in China.

Lisaftoclax is being studied in multiple clinical studies in countries and regions including the U.S., China, Australia, and the European Union, for a range of hematologic malignancies and solid tumors such as chronic lymphocytic leukemia, acute myeloid leukemia, and breast cancer. Lisaftoclax has been granted Orphan Drug Designations for five indications including Waldenström macroglobulinemia, chronic lymphocytic leukemia, multiple myeloma, acute myeloid leukemia, and follicular lymphoma.