Drug intelligence / Profile preview

angiozyme

Development stage
Phase 2
Lead developer
Alnylam Pharmaceuticals
Modality
RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous, Intravenous
01

Overview

Angiozyme (RPI.4610) is a chemically stabilized, synthetic hammerhead ribozyme designed to inhibit angiogenesis by targeting the mRNA of Vascular Endothelial Growth Factor Receptor 1 (VEGFR-1, also known as FLT-1). As a catalytic RNA molecule, Angiozyme binds to and cleaves the VEGFR-1 mRNA in a sequence-specific manner, leading to a reduction in the expression of the receptor on the cell surface and the subsequent abrogation of VEGF-mediated signaling pathways. Developed by Ribozyme Pharmaceuticals (later Sirna Therapeutics), the drug was investigated in Phase I and Phase II clinical trials for various solid tumors, including metastatic renal cell carcinoma and metastatic breast cancer. Although the drug demonstrated a favorable safety profile and was well-tolerated when administered alone or in combination with chemotherapy, it ultimately failed to show significant clinical efficacy, leading to the discontinuation of its development.

Brand names
Angiozyme
Other names
anti-FLT-1 ribozymeanti-FLT1 ribozymeanti-FLT 1 ribozymeanti-VEGFR-1 ribozymeanti-VEGFR1 ribozymeanti-VEGFR 1 ribozyme
02

Targets

Vascular endothelial growth factor receptor 1 (FLT1) mRNA

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