Drug intelligence / Profile preview

AS1411 + cytarabine

Development stage
Discontinued
Lead developer
Sarossa Capital
Modality
RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules
Administration
Intravenous
01

Overview

AS1411 + cytarabine is a combination of two drugs, AS1411 and cytarabine, investigated primarily for the treatment of acute myeloid leukemia (AML), especially in patients with relapsed or refractory disease. AS1411 is a guanosine-rich oligodeoxynucleotide aptamer that binds specifically to nucleolin, a protein overexpressed on the surface of many cancer cells. Binding to nucleolin results in internalization of AS1411 and inhibits key cellular processes essential for cancer cell survival and proliferation. Cytarabine is a well-established antimetabolite chemotherapy agent that inhibits DNA synthesis by acting as a cytosine nucleoside analog, primarily targeting rapidly dividing cells. The combination has progressed to phase II clinical trials and has demonstrated superior efficacy compared to cytarabine alone in AML, with AS1411 offering a novel, targeted mechanism that synergizes with cytarabine’s cytotoxic action[1][2][4][5][8].

02

Targets

NCL (Nucleolin)

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