Drug intelligence / Profile preview

pidnarulex

Development stage
Phase 2
Lead developer
Senhwa Biosciences
Modality
Small Molecules
Administration
Intravenous
01

Overview

Pidnarulex (CX-5461) is a first-in-class, small molecule inhibitor of RNA polymerase I (Pol I) transcription and a G-quadruplex stabilizer. Developed by Senhwa Biosciences, it selectively inhibits the synthesis of ribosomal RNA (rRNA), leading to nucleolar stress and activation of p53-dependent and independent apoptotic pathways. Beyond Pol I inhibition, pidnarulex stabilizes G-quadruplex DNA structures, inducing DNA double-strand breaks. This mechanism is particularly effective in tumors with homologous recombination (HR) deficiencies, such as those with BRCA1/2 mutations, through a synthetic lethality approach. It is being investigated for the treatment of various advanced solid tumors, including breast and ovarian cancers, as well as hematologic malignancies.

Brand names
Pidnarulex
Other names
CX-5461CX5461CX 5461
02

Targets

Pol I (RNA polymerase I)telomeric G4 (Telomeric DNA G-quadruplex)

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