AV-101 (imatinib)

AV-101 is a proprietary dry powder inhalation formulation of the tyrosine kinase inhibitor imatinib, developed by Aerovate Therapeutics for the treatment of pulmonary arterial hypertension (PAH). While oral imatinib (Gleevec) demonstrated clinical efficacy in PAH patients in the IMPRES trial, its use was severely limited by systemic adverse events. AV-101 is designed to be delivered directly to the lungs via a dry powder inhaler, achieving high local concentrations in the pulmonary vasculature to inhibit pathological remodeling while maintaining low systemic exposure to improve the safety and tolerability profile. The drug targets several kinases involved in PAH pathogenesis, including the platelet-derived growth factor receptors (PDGFR-α/β), colony-stimulating factor 1 receptor (CSF1R), and c-KIT. In June 2024, Aerovate announced that the Phase 2b portion of the IMPAHCT trial did not meet its primary endpoint of change in pulmonary vascular resistance (PVR), leading to the decision to halt the Phase 3 portion of the study.

Other names

inhaled imatinibimatinib dry powder for inhalation