Drug intelligence / Profile preview

VENT-02

Development stage
Phase 2
Lead developer
Ventus Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

VENT-02 is a novel, oral, brain-penetrant small molecule inhibitor of NLRP3 (NOD-, LRR-, and pyrin domain-containing protein 3), a key driver of inflammation and component of the innate immune system. Developed by Ventus Therapeutics, VENT-02 is designed to provide both symptomatic relief and disease modification in neurodegenerative diseases such as Parkinson’s disease. The drug has demonstrated robust pharmacodynamic modulation, full target engagement (100% inhibition of IL-1β in blood), significant central nervous system penetration with sustained levels in cerebrospinal fluid for 24 hours, and reduction of inflammatory biomarkers. Clinical trials have shown it to be well-tolerated with no dose-limiting toxicities or serious adverse events at intended therapeutic doses. VENT-02 is currently being evaluated for its potential to slow disease progression as well as relieve symptoms in Parkinson’s disease and other neuroinflammatory conditions[1][2][3][4][5][6].

02

Targets

NLRP3 (Nod-like receptor family pyrin domain-containing protein 3)

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