Aerovate Therapeutics’ mid-stage treatment for pulmonary arterial hypertension failed to meaningfully improve patients’ blood pressure compared to placebo across three doses, sending the company’s shares into a tailspin.
The Phase 2b data posted Monday cast a pall over the biotech’s ability to rival Merck’s new market entrant, Winrevair. Investors acted swiftly, with Aerovate’s shares $AVTE down more than 90% shortly after the market opened, from $24.62 to $2.26.
Though Aerovate said AV-101 was well tolerated, it did not post a statistically significant improvement in pulmonary vascular resistance across three dose ranges compared to placebo, the trial’s primary endpoint. The drug — an inhaled version of cancer treatment imatinib — also displayed no meaningful improvements in “several” of the study’s secondary endpoints.
Seemingly convinced of the asset’s ineffectiveness, Aerovate said it was halting and shutting down the Phase 3 leg of the study in consultation with the trial’s outside advisers, including a long-term extension portion. The company was enrolling across more than 120 sites in 20 countries. CEO Tim Noyes described the data as “unexpected and disappointing” in Monday’s release.
Timothy Noyes“In the coming weeks, we will engage closely with the IMPAHCT study advisory committee and the PAH community to thoroughly discuss these data and their implications,” Noyes said. The company had about $100 million in cash and short-term investments as of June 15, enough to last into 2026.
Aerovate emerged from RA Capital’s incubator in 2020 with $72.6 million from a Series A led by Sofinnova and went public less then a year later. The company’s share price had risen more than 40% in the last six months in anticipation of the readout and on the heels of Merck’s approval for PAH drug Winrevair, the centerpiece of the pharma’s $11.5 billion acquisition of Acceleron Pharma in September 2021.
In an update posted last month, Aerovate described the Phase 2b patient population as having “significant disease,” with more than half of the patients on triple background therapy.