Drug intelligence / Profile preview

ezn-2968

Development stage
Phase 1
Lead developer
Enzon Pharmaceuticals
Modality
Small Molecules, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

EZN-2968 is a third-generation antisense oligonucleotide designed to inhibit the expression of hypoxia-inducible factor 1 alpha (HIF-1α), a transcription factor involved in tumor adaptation to hypoxia and upregulation of genes necessary for cancer cell survival. By specifically binding to HIF-1α mRNA, EZN-2968 blocks its translation and reduces HIF-1α protein levels, leading to inhibition of angiogenesis, tumor cell proliferation, and induction of apoptosis. Preclinical studies demonstrated potent down-regulation of HIF-1α and its downstream targets in various cancer models. Clinically, EZN-2968 has been evaluated in phase I trials for advanced solid tumors including liver cancer and lymphoma[2][4][6][7]. The drug was originally developed by Santaris Pharma and further developed by Enzon Pharmaceuticals.

Other names
anti-HIF-1alpha LNA antisense oligonucleotideanti-HIF1alpha LNA antisense oligonucleotideanti-HIF 1alpha LNA antisense oligonucleotide
02

Targets

HIF1A (Hypoxia-inducible factor 1-alpha)

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