China NMPA Approves Ascentage Pharma’s Lisaftoclax, A First-In-Class Bcl-2 Inhibitor, For Treating CLL/SLL

Ascentage Pharma, a global biopharmaceutical company focused on addressing critical unmet needs in cancer treatment, has announced that its innovative Bcl-2 selective inhibitor, lisaftoclax (APG-2575), has been granted conditional approval by the National Medical Products Administration (NMPA) of China. The approval allows the use of lisaftoclax for the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least one prior systemic therapy, including treatment with Bruton’s tyrosine kinase (BTK) inhibitors.

This approval marks a major milestone for Ascentage Pharma, as lisaftoclax is now the first Bcl-2 inhibitor approved for the treatment of CLL/SLL in China and only the second Bcl-2 inhibitor to be approved globally. It also represents the company’s second approved novel therapy following the earlier approval of olverembatinib. Lisaftoclax is an orally administered, small-molecule inhibitor that selectively targets the Bcl-2 protein. Bcl-2 is an anti-apoptotic protein that prevents cancer cells from undergoing programmed cell death. By blocking Bcl-2, lisaftoclax helps to restore the normal process of apoptosis, thereby promoting the destruction of cancer cells. In clinical studies, lisaftoclax has shown strong potential in treating a range of blood cancers and solid tumors, especially CLL/SLL. It has been evaluated both as a standalone treatment and in combination with other therapies.

The conditional approval was based on findings from a pivotal Phase II registrational study (APG2575CC201), which evaluated lisaftoclax monotherapy in patients with relapsed or refractory CLL/SLL. The study’s primary endpoint was overall response rate (ORR), and lisaftoclax successfully met this endpoint, demonstrating encouraging efficacy in patients previously treated with BTK inhibitors and/or immunochemotherapy. The treatment also showed a favorable safety profile, with no cases of tumor lysis syndrome (TLS), a low and manageable incidence of blood-related toxicities, and mostly mild non-blood-related side effects.

Prof. Jianyong Li, Principal Investigator of the study from the Lymphoma Center at Jiangsu Province Hospital, stated, “CLL/SLL is a common hematologic malignancy in aging societies, and its prevalence has been steadily rising in China. Despite advancements in treatment, patients with CLL/SLL still face a range of challenges such as drug resistance, long-term medical treatment that is difficult to manage, and disappointing response rates. In recent years, drugs targeting pro-apoptotic proteins have received widespread interest in the oncology field while Bcl-2 inhibitors have emerged as a new treatment strategy for patients with CLL/SLL. Globally, Bcl-2 inhibitors have already become a key part of the treatment of CLL/SLL, but no Bcl-2 inhibitor has been approved in China up till now. This approval for the next-generation Bcl-2 inhibitor lisaftoclax represents a timely response to the urgent unmet medical need of this patient population, effectively fulfilling the void for Bcl-2 inhibitors in CLL/SLL in China. 

He also added, “As the first China-developed Bcl-2 inhibitor, lisaftoclax has demonstrated favorable efficacy and a unique safety profile, thus providing clinicians a new treatment option that can meaningfully improve the survival of patients with CLL/SLL. In April 2025, supported by its groundbreaking efficacy and safety data in Chinese patients with CLL/SLL, lisaftoclax was included in the 2025 Chinese Society of Clinical Oncology (CSCO) Guidelines for the Diagnosis and Treatment of Lymphoid Malignancies. The approval and guideline recommendations for lisaftoclax, the only Bcl-2 inhibitor approved in China for the treatment of CLL/SLL, validated the drug as a safe and efficacious new treatment option, underscoring a major advancement in precision therapy for hematologic malignancies in China.”

Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma, mentioned, “We are encouraged by the Chinese drug regulators’ recognition of lisaftoclax’s efficacy and safety, and would like to thank our investigators and their teams, and the patients for participating in the clinical study, for their efforts and trust. This approval for lisaftoclax is a milestone achieved through over a decade of committed research and clinical development by Ascentage Pharma. Lisaftoclax addresses an urgent unmet clinical need and brings a safe and effective new treatment option to patients with CLL/SLL. This represents another big step forward in our patient-centric global innovation. Lisaftoclax has broad therapeutic potential in multiple hematologic malignancies and solid tumors. We will accelerate the global development of this drug in various indications and bring it to more patients as soon as possible.”

Dr. Dajun Yang, Chairman & CEO of Ascentage Pharma, said in a statement, “Our founding team has over 20 years of research experience in the field of apoptosis and accumulated deep expertise on the Bcl-2 target. This approval for lisaftoclax is a culmination of their dedicated research and a major milestone in our never-ending journey of innovation, further solidifying our leadership in the hematology field. As a proprietary novel drug developed through global innovation, lisaftoclax is a testament to our strength in drug development, bringing much needed change to the global landscape for Bcl-2 inhibitors. Moving forward, Ascentage Pharma will remain steadfastly committed to its mission of addressing unmet clinical needs in China and around the world and aspire to bring more innovative therapeutics to more patients globally.”

CLL/SLL is a type of blood cancer arising from mature B-cell neoplasms and typically affects older adults. Each year, more than 100,000 people are diagnosed with the condition worldwide. Although CLL/SLL is less common in China compared to Western countries, its incidence in China is rising quickly, with younger patients and more aggressive disease patterns being observed. While BTK inhibitors have become a standard first-line treatment for CLL/SLL and have significantly improved patient outcomes, they are often associated with limitations such as an inability to achieve deep responses, risk of relapse with long-term use, and tolerability issues. This has created a growing need for new and more effective therapies.

Bcl-2 inhibitors have emerged as a promising treatment option for CLL/SLL, targeting a key mechanism that allows cancer cells to evade death. However, developing a Bcl-2 inhibitor has been especially challenging due to the protein's complex structure and its location within the mitochondria. Lisaftoclax has overcome these obstacles, offering a new avenue of treatment that was previously unavailable in China.

In addition to this approval, Ascentage Pharma is conducting four global Phase III clinical trials involving lisaftoclax. These include the GLORA study, which evaluates lisaftoclax in combination with BTK inhibitors for CLL/SLL patients with a suboptimal response to prior BTK therapy; the GLORA-2 study for newly diagnosed CLL/SLL patients; the GLORA-3 study for elderly and unfit patients with newly diagnosed acute myeloid leukemia (AML); and the GLORA-4 study for patients with newly diagnosed higher-risk myelodysplastic syndrome (MDS). This approval of lisaftoclax not only fills a significant treatment gap for CLL/SLL patients in China but also reinforces Ascentage Pharma’s position as a leading innovator in the field of oncology drug development.