Drug intelligence / Profile preview

pegtomarginase

Development stage
Phase 1
Lead developer
Athenex
Modality
Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Biodegradable Polymers → Polymer-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

Pegtomarginase is a PEGylated, genetically modified human arginase enzyme developed as an anticancer agent. Its mechanism of action involves metabolizing the amino acid arginine to ornithine and urea, thereby depleting circulating arginine levels. Many cancer types are unable to synthesize arginine due to deficiencies in key urea cycle enzymes such as argininosuccinate synthetase 1 (ASS1), argininosuccinate lyase (ASL), or ornithine transcarbamylase (OTC). By removing extracellular arginine, pegtomarginase selectively starves these cancers of an essential nutrient required for their growth and survival while sparing healthy cells that can produce their own arginine. The drug is a recombinant human enzyme with site-specific PEGylation for improved pharmacokinetics and reduced immunogenicity compared to non-human or randomly PEGylated alternatives. It was initially developed by researchers at The Hong Kong Polytechnic University and further advanced by Avalon Biomedical and Athenex for the treatment of advanced malignancies[1][2][5][7].

Other names
Pegylated arginasePegylated Modified Human Arginase
02

Targets

ARG1 (Arginase 1)

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