Ascentage Pharma Announces CDE's Approval for the Phase II Pivotal Study in China of the Bcl-2 Inhibitor Lisaftoclax (APG-2575) for the Treatment of Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

SUZHOU, China and ROCKVILLE, Md., Dec. 12, 2021 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced 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.

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) 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.

About Ascentage Pharma
Ascentage Pharma (6855.HK) is a globally focused biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B, and age-related diseases. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code 6855.HK.

Ascentage Pharma focuses on developing therapeutics that inhibit protein-protein interactions to restore apoptosis, or programmed cell death. The company has built a pipeline of eight clinical drug candidates, including novel, highly potent Bcl-2, and dual Bcl-2/Bcl-xL inhibitors, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation tyrosine kinase inhibitors (TKIs). Ascentage Pharma is also the only company in the world with active clinical programs targeting all three known classes of key apoptosis regulators. The company is conducting more than 40 Phase I/II clinical trials in the US, Australia, Europe, and China. Olverembatinib, the company's core drug candidate developed for the treatment of drug-resistant chronic myeloid leukemia (CML), was granted Priority Review status and a Breakthrough Therapy Designation (BTD) by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA), and is already approved for the indication. In addition, the olverembatinib was also granted an Orphan Drug Designation (ODD) and a Fast Track Designation (FTD) by the US FDA, and an Orphan Designation by the EU. To date, Ascentage Pharma has obtained a total of 12 ODDs from the US FDA and 1 ODD from the EU for four of the company's investigational drug candidates. Ascentage Pharma has been designated for multiple Major National R&D Projects, including five National Major New Drug Discovery and Manufacturing projects, one New Drug Incubator status, four Innovative Drug Programs, and one Major Project for the Prevention and Treatment of Infectious Diseases.

Leveraging its robust R&D capabilities, Ascentage Pharma has built a portfolio of global intellectual property rights and entered into global partnerships with numerous renowned biotechnology and pharmaceutical companies and research institutes such as UNITY Biotechnology, MD Anderson Cancer Center, Mayo Clinic, Dana-Farber Cancer Institute, MSD, AstraZeneca, and Pfizer. The company has built a talented team with global experience in the discovery and development of innovative drugs and is setting up its world-class commercial manufacturing and Sales & Marketing teams. One pivotal aim of Ascentage Pharma is to continuously strengthen its R&D capabilities and accelerate its clinical development programs, in order to fulfil its mission of addressing unmet clinical needs in China and around the world for the benefit of more patients.

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SOURCE Ascentage Pharma