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Bone marrow growth abnormal syndrome (MDS) includes a range of blood system diseases characterized by chronic blood cell reduction (anemia, nemetic granulocyte reduction, and plate reduction) accompanied by abnormal cell maturation.
, MDS patients are at risk of developing symptomatic anemia, infection, bleeding, and developing acute myeloid leukemia (AML, often difficult to treat).
Antegen is a leading biopharmaceutical company dedicated to developing best-in-class therapies for hematology and oncology.
recently, China's State Drug Administration (NMPA) has approved a clinical trial of ATG-016 (eltanexor) for the treatment of high-risk bone marrow growth syndrome (MDS).
this trial is a phase I/II single-arm open-label clinical study designed to assess the pharmacodynamics, safety and ability of ATG-016 (eltanexor).
total survival (OS) in patients with resoicative high-risk MDS was only 4-6 months, and follow-up treatment options were limited.
Preclinical studies have shown that nuclear transport selective inhibitors (SINE) can block the nuclear output of many tumor suppressor proteins (e.g. p53, IkB, p21), resulting in their accumulation and resuspension in the nucleus, thus playing an anti-tumor role.
ATG-016 is a member of the latest generation of SINE compounds.
, the ATG-016 has blood-brain barrier permeability compared to the first generation of SINE, which shows initial anti-cancer activity in patients with high-risk MDS.
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