Aerovate Therapeutics, Inc. announced topline results from the Phase 2b portion of the Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial (IMPAHCT), a Phase 2b/Phase 3, randomized, double-blind, placebo-controlled, multi-national trial of AV-101, a novel dry powder inhaled formulation of imatinib, in adults with pulmonary arterial hypertension (PAH). The objective of the Phase 2b portion of IMPAHCT was to assess the efficacy, safety and tolerability of three different doses of AV-101 compared to placebo. The primary endpoint for the Phase 2b portion of IMPAHCT is change in PVR compared with placebo.

Results showed that, while AV-101 was well tolerated across all dose groups, the study did not meet its primary endpoint for improvement in PVR compared to placebo for any of the studied doses or show meaningful improvements in the secondary endpoint of change in six minute walk distance (6MWD). The Company has also reviewed data from several additional secondary endpoints of the Phase 2b portion of IMPAHCT, which also failed to show meaningful improvements. Based upon these results, Aerovate, in agreement with the independent study advisory committee, is halting enrollment and shutting down the Phase 3 portion of IMPAHCT as well as the long-term extension study.

Aerovate plans to release full data from the Phase 2b portion of IMPAHCT at a later date, the timing of which is to be determined. As of June 15, 2024, Aerovate has approximately $100 million of cash, cash equivalents and short-term investments. AV-101 is an investigational, proprietary dry powder inhaled formulation of the antiproliferative drug imatinib.

Developed specifically for pulmonary arterial hypertension (PAH), AV-101 targets cellular hyperproliferation and resistance to apoptosis, driven by improper signaling in cells of the distal pulmonary arteries. AV-101 is designed for delivery by an easy-to-use dry powder inhaler, directly into the lungs to maximize potential clinical benefit and limit systemic adverse effects. IMPAHCT (Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial) is a multi-national, placebo-controlled Phase 2b/Phase 3 trial in adults with PAH that continuously enrolled patients from Phase 2b to Phase 3. The Phase 2b portion of the trial evaluated three doses of AV-101 over 24 weeks, compared to placebo, to identify an optimal dose based on the primary endpoint, change in pulmonary vascular resistance (PVR), and safety, tolerability, and other clinical measures.