近日,美国FDA传来喜讯,授予 PCLX-001孤儿药称号(ODD),用于治疗急性髓细胞白血病患者。 PCLX-001是一种 N-肉豆蔻酰转移酶 (NMT) 抑制剂,靶向蛋白质的脂质修饰,这些蛋白质可启动癌细胞增殖和存活的信号通路,尤其是在白血病和淋巴瘤中。 在来自淋巴瘤细胞系、肺癌细胞系、乳腺癌细胞系的皮下肿瘤异种移植模型中,以...
PCLX-001 also demonstrated anticancer activity in multiple solid tumor xenograft models. Based on pre-clinical data, we hypothesized that PCLX-001 would be safe, tolerable, and active as a novel oral cancer therapeutic. Methods: Patients with relapsed/refractory B-cell lymphomas and advanced solid...