Results from Phase 2b part of inhaled imatinib trial due in June

IMPAHCT is testing multiple doses of AV-101 against placebo in adults

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The Phase 2b portion of the IMPAHCT clinical trial, which is testing multiple doses of the inhaled imatinib formulation AV-101 against a placebo in adults with pulmonary arterial hypertension (PAH), has finished enrollment.

Top-line results are expected in June 2024, according to AV-101’s developer, Aerovate Therapeutics.

“The completion of enrollment in the Phase 2b portion of the trial marks an exciting milestone for Aerovate and for people living with PAH,” Tim Noyes, Aerovate’s CEO, said in a company press release.

The Phase 2b portion of the IMPAHCT study (NCT05036135) enrolled 202 adults with PAH. The participants are being randomly assigned to one of three doses of AV-101, or a placebo, for about six months.

The main goal of this part of the trial is to test how the treatment affects pulmonary vascular resistance (PVR), a measure of the resistance to blood flow. PAH is marked by elevated pressure in the lung’s blood vessels.

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Phase 3 portion of IMPAHCT

IMPAHCT also includes a Phase 3 portion that has already begun to enroll patients.

“Because of our innovative adaptive Phase 2b/Phase 3 trial design for IMPAHCT, we were also able to rapidly enroll our first patient in the Phase 3 portion of the study,” Noyes said.

The Phase 3 part of the trial will be testing a single dose of AV-101 against a placebo for about six months, with the goal of assessing how it affects the distance that patients can walk in six minutes, a common measure of exercise capacity.

Results from the Phase 2b part of IMPAHCT will determine the optimal dose for Phase 3 testing. Once Phase 2b results are in, all subsequently enrolled patients will be given either the optimal dose of AV-101 or a placebo. The study is recruiting adults with PAH ages 18-75 at sites worldwide.

“I am extremely grateful for the commitment of our clinical investigators and their patients, and proud of the Aerovate team’s dedication and ongoing execution of the trial, as this furthers our goal of developing meaningful therapeutic options for PAH patients as quickly as possible,” Noyes said.

AV-101 is a dry powder formulation of imatinib, a compound that’s thought to prevent abnormal cell growth in blood vessels that contributes to high blood pressure in PAH. An oral formulation of imatinib, developed by Novartis, showed beneficial effects on exercise capacity in people with PAH in clinical trials, but also caused substantial side effects and its development was discontinued.

Aerovate expects AV-101 may provide similar exercise capacity benefits, but with a more manageable safety profile because it delivers the therapy straight into the lungs. The inhaled formulation has shown promise in preclinical studies.

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