On May 28, 2025 InnoCare Pharma (HKEX: 09969; SSE: 688428), a leading biopharmaceutical company focusing on the treatment of cancer and autoimmune diseases, reported the approval of the Investigational New Drug (IND) by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) 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, including but not limited to myelodysplastic syndromes (MDS) (Press release, InnoCare Pharma, MAY 28, 2025, View Source [SID1234653471]).
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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.
Myelodysplastic syndrome (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 acute myeloid leukemia (AML). The annual incidence of myelodysplastic syndromes is about 4 cases/100,000 people/year (reaching 40–50/100,000 in patients aged ≥ 70 years).
Mesutoclax has been granted Breakthrough Therapy Designation (BTD) by the CDE for the treatment of BTKi-treated relapsed or refractory mantle cell lymphoma (R/R MCL). This marks the first BCL2 inhibitor to receive BTD recognition in China. The Company is accelerating patient enrollment of a Phase III registrational trial of mesutoclax in combination with orelabrutinib as a first line therapy for the treatment of chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), as well as a clinical trial of mesutoclax for the treatment of AML.
Dr. Jasmine Cui, the Co-founder, Chairwoman, and CEO of InnoCare, said, "Mesutoclax is an important global asset of our company in the field of hematology. We are delighted to receive approval to initiate the clinical trial for the fourth indication of our BCL2 inhibitor. We will accelerate the clinical development of mesutoclax across multiple indications in China and globally to bring benefits to patients as early as possible."