InnoCare Announces FDA Approval of Clinical Trial for BCL2 Inhibitor Mesutoclax in AML and MDS

July 14, 2025 ，InnoCare Pharma (HKEX: 09969; SSE: 688428), a leading biopharmaceutical company focusing on the treatment of cancer and autoimmune diseases, announced that the U.S. Food & Drug Administration has approved  the Investigational New Drug (IND) application to conduct the clinical trial of B-cell lymphoma-2 (BCL2) inhibitor mesutoclax (ICP-248) in combination with azacitidine for the treatment of myeloid malignancies, such as acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

Mesutoclax is a novel, orally bioavailable BCL2 selective inhibitor. BCL2 is an important regulatory protein in the apoptosis pathway, and its abnormal expression is associated with the development of various hematologic malignancies. Mesutoclax exerts anti-tumor activity by selectively inhibiting BCL2 and restoring the normal apoptosis process in cancer cells.

As an important global pipeline asset, InnoCare is advancing multi-center, multi-indication clinical trials of mesutoclax globally, including a registrational Phase III clinical trial in combination with orelabrutinib as a first-line treatment for chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), a registrational clinical trial for the treatment of relapsed/refractory mantle cell lymphoma (MCL), and a clinical trial in combination with azacitidine for the treatment of myeloid malignancies such as AML and MDS.

Dr. Jasmine Cui, Co-founder, Chairwoman, and CEO of InnoCare, said, “Mesutoclax is a key global asset for InnoCare in the field of hematology. We believe mesutoclax will become a new option for the treatment of myeloid malignancies such as AML and MDS. We will accelerate clinical development both in China and globally, and look forward to bringing greater benefits to patients as early as possible.”

AML is a malignant hematological disease originating from hematopoietic stem/progenitor cells. The risk of developing AML increases with age and is more common in older adults.

MDS is a group of heterogeneous clonal myeloid diseases characterized by the abnormal proliferation of hematopoietic stem cells, recurrent genetic abnormalities, myelodysplasia, ineffective hematopoiesis, peripheral-blood cytopenia, and a high risk of progression to AML. 

About InnoCare

InnoCare is a commercial stage biopharmaceutical company committed to discovering, developing, and commercializing first-in-class and/or best-in-class drugs for the treatment of cancers and autoimmune diseases with unmet medical needs in China and worldwide. InnoCare has branches in Beijing, Nanjing, Shanghai, Guangzhou, Hong Kong, and the United States.