Apta-16

Apta-16 (also known as SJP1604) is a first-in-class, synthetic aptamer-drug conjugate developed for the treatment of relapsed or refractory acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). It consists of a nucleotide aptamer that specifically targets surface nucleolin, which is highly expressed on cancer cells, conjugated to a small molecule inhibitor. The drug acts primarily as a nucleolin inhibitor and also exhibits histone methyltransferase inhibitory activity. Preclinical studies have shown that Apta-16 selectively binds to nucleolin on AML cells, inhibits their growth—including drug-resistant subtypes—and demonstrates anti-leukemic efficacy in vivo with reduced toxicity compared to standard therapies. Mechanistically, it downregulates nucleolin expression and inactivates NFκB signaling, leading to decreased DNMT1 expression and increased p15 expression; it also increases p53 levels while reducing bcl-2 expression. The drug has received orphan drug designation for AML[1][2][6].

Other names

APTA 16APTA16APTA-16