Myelodysplastic Syndrome

Ofirnoflastum (Ofirnoflast) is an orally active first-in-class allosteric NEK7 inhibitor with an IC₅₀ of 46 nM. Ofirnoflastum binds an allosteric site adjacent to NEK7’s ATP-binding pocket, induces conformational shifts, disrupts NEK7-NLRP3 binding, blocks NLRP3 inflammasome assembly, spares NEK7’s physiological functions, and suppresses caspase-1, caspase-8, NF-κB, and TNF activity. Ofirnoflastum reduces pro-inflammatory cytokine production, suppresses ASC specks, IL-1β release, pyroptotic cell death, and leukemic burden, induces apoptosis and erythroid differentiation, restores hematopoiesis, and improves outcomes in colitis models. Ofirnoflastum can be used for the research of myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia.

Cytarabine-daunorubicin (CPX 351) is a Liposomal formulation prepared from Cytarabine (HY-13605) and Daunorubicin (HY-13062A) at a fixed synergistic molar ratio of 5:1. Cytarabine-daunorubicin improves secondary acute myeloid leukemia. Cytarabine-daunorubicin can be used in research related to secondary acute myeloid leukemia, high-risk myelodysplastic syndrome, and chronic myelomonocytic leukemia.

SIK2/3-IN-2 (compound 12, compound I-200) is a potent SIK2/3 inhibitor (SIK2 IC₅₀ = 65 nM, SIK3 IC₅₀ = 14 nM). SIK2/3-IN-2 is also a p21-activated protein kinase (PAK) 1 inhibitor with a K_i of 20.7 nM. SIK2/3-IN-2 can be used for hyperproliferative diseases and cancer research, such as Paclitaxel (HY-B0015)-resistant ovarian cancer.

JAK2-IN-18 (Compound example1) is a selective JAK2 inhibitor. JAK2-IN-18 can inhibit JAK-STAT signaling and shows an IC₅₀ of <100 nM for pSTAT5 in HEL9217 cells. JAK2-IN-18 can inhibit the proliferation of abnormally proliferating myeloid cells and can be used for the research of myeloproliferative disorders, such as essential thrombocythemia.

CVS-1123 is an orally active direct thrombin inhibitor. CVS-1123 inhibits in vitro platelet aggregation of γ-thrombin and prolongs activated partial thromboplastin time. CVS-1123 alters the thrombotic response to deep vessel wall damage in arterial and venous circulation. CVS-1123 can be used in antithrombotic studies

BI-836858 is a fully human anti-CD33 monoclonal antibody. BI-836858 reduces CD33+ cells via antibody-dependent cellular cytotoxicity (ADCC), blocks downstream signaling of S100A9/CD33, decreases the secretion of immunosuppressive cytokines and reactive oxygen species-induced genomic instability, and restores bone marrow hematopoietic function. BI-836858 is applicable to the research of myelodysplastic syndrome (MDS) and AML.