Drug intelligence / Profile preview

PR5-LL-CM01

Development stage
Preclinical
Lead developer
Indiana University
Modality
Small Molecules
Administration
Intraperitoneal
01

Overview

PR5-LL-CM01 (also known as CM01) is a patented small-molecule inhibitor of Protein Arginine Methyltransferase 5 (PRMT5) being investigated for the treatment of pancreatic ductal adenocarcinoma (PDAC). Developed by researchers at Indiana University and Purdue University, the compound targets the PRMT5-mediated NF-κB signaling pathway, which is a key driver of persistent inflammation and tumorigenesis in PDAC. Due to its poor water solubility, PR5-LL-CM01 has been formulated as an albumin-coated nanocrystal (CM01 NC or Abxtal) to enhance its bioavailability and therapeutic potential. In preclinical models, the nanocrystal formulation demonstrated superior anti-tumor efficacy and reduced toxicity compared to the PRMT5 inhibitor EPZ015666 and showed synergistic effects when combined with gemcitabine.

02

Targets

PRMT5 (Protein arginine N-methyltransferase 5)

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