Drug intelligence / Profile preview

PKT100

Development stage
Preclinical
Lead developer
The University of Sydney
Modality
Small Molecules
Administration
Unknown
01

Overview

PKT100 is a **novel small molecule antagonist of the P2X7 receptor**, developed for potential use in cardiovascular diseases, specifically targeting the right ventricle in pulmonary hypertension. Unlike other agents that primarily lower pulmonary vascular resistance, PKT100 appears to directly attenuate right ventricular (RV) hypertrophy and improve RV contractile function and survival in animal models, without significantly altering pulmonary vascular remodeling or inflammation. It has demonstrated benefit in a mouse model of pulmonary fibrosis-induced pulmonary hypertension, suggesting therapeutic potential for right ventricular failure associated with pulmonary hypertension. The drug was investigated in preclinical studies by researchers affiliated with The University of Sydney, but there is no evidence of commercial development or availability in humans[1].

02

Targets

P2RX7 (P2X purinoceptor 7)

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