Aerovate Therapeutics has announced results of their phase 1 trial of AV-101, a new dry powder inhaled imatinib formulation for the treatment of patients with pulmonary arterial hypertension (PAH). The results were presented at the American Thoracic Society (ATS) International Conference in San Francisco, California on May 16, 2022.

“We are pleased these Phase 1 data showed that AV-101 was generally well-tolerated in this trial of healthy adult volunteers across a dose range we believe may overlap or exceed lung exposures from 400 mg of oral imatinib, the dose used in the Novartis global Phase 3 IMPRES trial, while simultaneously reducing systemic exposure,” Timothy Noyes, chief executive officer of Aerovate, said.

“These findings represent an important step forward in our clinical development of AV-101, and support the selection of doses being evaluated in our ongoing Phase 2b/Phase 3 IMPAHCT trial.”

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Imatinib has already shown clinically meaningful benefit for patients with PAH in the phase 3 IMPRES trial performed by Novartis. However, the oral tablet formulation was not well tolerated by the patients due to adverse events and was therefore discontinued.

AV-101 is a novel dry powder inhaled formulation of imatinib, and it has shown lower systemic exposure than the oral form and no significant adverse effects in the participating patients. The phase 1 trial assessed safety, tolerability and pharmacokinetics of AV-101 in single and multiple ascending doses in healthy volunteers.

Aerovate Therapeutics is currently enrolling patients in the global phase 2b/phase 3 Inhaled Imatinib Pulmonary Arterial Hypertension Clinical Trial to assess the safety and efficacy of various doses of AV-101 in adult patients with PAH.


Aerovate Therapeutics presents phase 1 data for AV-101, a novel dry powder inhaled formulation of imatinib being developed for the treatment of patients with pulmonary arterial hypertension (PAH). News release. Aerovate Therapeutics; May 16, 2022.