ASLAN003 是一种具有口服活性的二氢乳清酸脱氢酶 (DHODH) 抑制剂，对人 DHODH 酶的IC50为 35 nM。它通过激活 AP-1 转录因子来抑制蛋白质合成，可以诱导凋亡，并在急性髓样白血病异种移植小鼠中大大延长其生存期。

ASLAN003 is an orally active and potent inhibitor of DHODH (Human Dihydroorotate Dehydrogenase) with antitumor activity. It has the potential to be a first-in-class candidate in AML.

ASLAN003 is a highly potent dihydroorotate dehydrogenase inhibitor that induces differentiation, as well as reduces cell proliferation and viability, of acute myeloid leukemia cell lines and primary acute myeloid leukemia blasts including in chemo-resistant cells.?Apoptotic pathways are triggered by ASLAN003, and it also significantly inhibits protein synthesis and activates AP-1 transcription, contributing to its differentiation promoting capacity[2].

ASLAN003 substantially reduces leukemic burden and prolongs survival in acute myeloid leukemia xenograft mice and acute myeloid leukemia patient-derived xenograft models. Notably, the drug has no evident effect on normal hematopoietic cells and exhibits excellent safety profiles in mice, even after a prolonged period of administration. ASLAN003 is an agent targeting dihydroorotate dehydrogenase with potential in the treatment of acute myeloid leukemia. ASLAN003 is currently being evaluated in phase 2a clinical trial in acute myeloid leukemia patients[2].

1035688-66-4

C19H14F2N2O3

356.32

ASLAN003

DMSO:120 mg/mL (336.78 mM),Need ultrasonic
Powder: -20°C for 3 years
In solvent: -80°C for 2 years