Drug intelligence / Profile preview

PT001

Development stage
Unknown
Lead developer
PulmoSIM Therapeutics
Modality
Small Molecules
Administration
Inhalation
01

Overview

PT001 is an investigational, inhaled formulation of pirfenidone being developed by PulmoSIM Therapeutics, a subsidiary of VeriSIM Life, for the treatment of pulmonary arterial hypertension (PAH). Developed using the BIOiSIM artificial intelligence platform, PT001 is designed as a potentially disease-modifying therapy that targets multiple pathways involved in the pathological remodeling of pulmonary arteries. By delivering the drug directly to the lungs via inhalation, PT001 aims to achieve high local concentrations to stop and reverse disease progression while minimizing the systemic side effects typically associated with oral pirfenidone. The drug received Orphan Drug Designation from the U.S. FDA in June 2021 and is being advanced through partnerships with National Jewish Health and Brown University.

Other names
Inhaled pirfenidone
02

Targets

TGFB (GARP–latent transforming growth factor beta 1 complex)TNFA (Tumor necrosis factor alpha (soluble))

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