Aerovate Launches Clinical Trial of AV-101 in PAH

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Aerovate Therapeutics is starting a Phase 2b/3 clinical trial that will test AV-101, the company’s experimental inhaled formulation of imatinib, in people with pulmonary arterial hypertension (PAH).

“We are excited and humbled to initiate this Phase 2b/Phase 3 trial of AV-101. Starting enrollment represents an important milestone for Aerovate Therapeutics and advances our goal of improving the lives of patients suffering from rare cardiovascular diseases,” Tim Noyes, Aerovate’s CEO, said in a press release.

The trial, called IMPAHCT (NCT05036135), is currently enrolling participants at the University of Kansas Medical Center in Kansas City, Kansas, with additional sites planned to open later. The study is open to adults with PAH ages 18 to 75.

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In the Phase 2b portion of the trial, participants will be assigned randomly to one of three doses of AV-101, or a placebo.

The main goal of the Phase 2b portion is to determine the effect on pulmonary vascular resistance (PVR) — resistance to blood flow in the lungs’  blood vessels — after 24 weeks, or about six months. Safety, tolerability, and other clinical measures also will be assessed.

Results of the Phase 2b part of the trial are expected midway through 2023, according to Aerovate.

Based on findings from the Phase 2b portion, a single dosage of AV-101 will be selected for the Phase 3 portion, where participants will get that dose of AV-101 or a placebo for 24 weeks. The main goal of the Phase 3 part is to evaluate the effect of treatment on the 6-minute walk distance (6MWD), which measures how far a person can walk in six minutes. It is a common measure of overall physical fitness in people who are able to walk.

AV-101 is a dry-powder formulation of imatinib that has been dosed specifically for PAH. Imatinib works by blocking cell growth; abnormal cell growth is thought to drive the progression of PAH.

An oral formulation of imatinib is approved to treat certain cancers, whereas inhaled formulations of the therapy for PAH are in development. Inhaled formulations that deliver the medication directly to the lungs are expected to limit side effects associated with system-wide exposure to the medication. Those side effects may include gastrointestinal distress (vomiting, diarrhea, etc.), aches/pains, bleeding problems, and unusual fatigue or weakness.

“Imatinib has shown promise as a therapy for PAH and if AV-101 can deliver improvements for patients with fewer of the systemic adverse events associated with oral imatinib in PAH, it could represent a real advancement for patients,” said Nicholas Hill, MD, of Tufts Medical Center. Hill is the chair of the IMPAHCT clinical advisory board.

“There is a real need for new treatment options for PAH patients that work differently from our currently approved therapies,” Hill added.

AV-101 was generally well-tolerated in a recent Phase 1 trial that tested the medication in healthy volunteers, according to Aerovate. The company raised more than $70 million to support the development of AV-101 last year.