Aerovate's PAH drug fails Phase II, shares drop

Aerovate Therapeutics recently announced that its pulmonary arterial hypertension (PAH) treatment candidate, AV-101, did not achieve its primary endpoint in the Phase IIb portion of the IMPAHCT trial. This news caused Aerovate's shares to plummet by more than 90% during early trading on Monday.

The Phase IIb segment of the IMPAHCT trial was designed to evaluate three different doses of AV-101, a novel dry powder inhaled form of the drug imatinib. The primary endpoint of the study was to observe changes in pulmonary vascular resistance (PVR) in comparison to a placebo. Unfortunately, none of the dosages of AV-101 showed a significant improvement in PVR. Additionally, there was no notable enhancement in the secondary endpoint, which measured patients' ability to walk a certain distance within six minutes.

CEO Tim Noyes expressed that the results were "unexpected and disappointing." He emphasized that the company would now prioritize "transparently sharing these findings with investigators, patients, and the PAH community."

AV-101 is a dry powder inhaled formulation of imatinib, an antiproliferative drug specifically designed for the treatment of PAH. The drug aims to mitigate cellular hyperproliferation and resistance to apoptosis, which are driven by improper cell signaling in the distal pulmonary arteries. By delivering the drug directly to the lungs through inhalation, Aerovate intended for more of the drug to reach diseased tissues while reducing systemic exposure.

Due to this setback, Aerovate is halting enrollment and discontinuing the Phase III portion of the IMPAHCT trial as well as the long-term extension study of AV-101. According to the company's website, AV-101 appears to be its sole programme. As of mid-June, Aerovate reported having approximately $100 million in cash, cash equivalents, and short-term investments.