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VRN13 is an inhaled small molecule kinase inhibitor developed by Voronoi for the treatment of pulmonary arterial hypertension (PAH). It selectively targets the platelet-derived growth factor receptor (PDGFR), a key mediator of the pathological vascular remodeling and smooth muscle cell proliferation that characterize PAH. By utilizing an inhalation route of administration, VRN13 is designed to deliver the drug directly to the pulmonary vasculature, achieving high local concentrations while minimizing systemic exposure. This approach aims to improve the safety profile compared to systemic PDGFR inhibitors, such as imatinib, which have been limited by systemic toxicities. VRN13 also demonstrates high selectivity for PDGFR over VEGFR-2 to further reduce the risk of vascular side effects.
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