InnoCare's mesutoclax approved for Phase III clinical trial in head-to-head comparison with venetoclax for treating newly diagnosed AML

InnoCare (SSE: 688428; HKEX: 09969) announced today that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) has approved the initiation of a registrational Phase III clinical trial for its self-developed novel BCL2 inhibitor, mesutoclax (ICP-248), in combination with azacitidine. The trial will conduct a head-to-head comparison against venetoclax combined with azacitidine for the treatment of elderly or unfit patients with newly diagnosed acute myeloid leukemia (AML).

This is a randomized, open-label, multicenter, registrational Phase III clinical trial for elderly or unfit patients with newly diagnosed AML, with overall survival (OS) as the primary endpoint.

Mesutoclax is a novel oral highly selective BCL2 inhibitor. BCL2 is a key regulatory protein in the apoptosis pathway, and its abnormal expression is associated with the development of various malignant hematologic tumors. Mesutoclax exerts anti-tumor efficacy by selectively inhibiting BCL2, thereby restoring the programmed cell death mechanism in tumor cells. Mesutoclax has received two Breakthrough Therapy Designations (BTD) from the CDE in China.

Data released by the American Society of Clinical Oncology (ASCO) this May showed that mesutoclax combined with azacitidine demonstrated favorable efficacy and safety in treating AML patients. As of April 13, 2026, among evaluable patients with newly diagnosed AML, the composite complete response rate (cCR, including CR and CRi) was 81.8%. Among patients who achieved an overall response, 86.5% reached minimal residual disease (MRD) negativity. 83% of cCR patients achieved cCR in the first cycle, indicating that the mesutoclax regimen can help patients achieve faster and deeper responses. In terms of safety, no dose-limiting toxicities (DLT) occurred, and the maximum tolerated dose (MTD) was not reached. Among patients with newly diagnosed AML, early mortality (at 30 and 60 days) was 0%. The 6-month overall survival rate at the recommended dose was 90.5%.

Dr. Cui Jisong, Co-founder, Chairman, and CEO of InnoCare Pharma, stated: “This marks a significant milestone in the clinical development of mesutoclax and underscores our strong confidence in the drug. Mesutoclax is a key asset in our global pipeline; the clinical trial evaluating its combination with azacitidine for the treatment of AML has successfully completed patient enrollment in the United States and Australia and is now accelerating. We are fully committed to advancing clinical development to bring this innovative therapy to more patients worldwide as soon as possible.”

As a leading player in the field of hematologic oncology, InnoCare Pharma has successfully launched two innovative drugs, orelabrutinib and tislelizumab, establishing robust commercial capabilities in the lymphoma sector. With this entry into acute myeloid leukemia (AML), the company further refines its hematologic oncology pipeline, covering two major therapeutic areas: B-cell lymphomas and myeloid malignancies.

AML is a hematologic malignancy originating from hematopoietic stem/progenitor cells. The risk of incidence rises continuously with age, predominantly affecting middle-aged and elderly individuals [1].[1] Technical Guidelines for Clinical Development of New Drugs for Acute Myeloid Leukemia

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