• AV-101, a novel dry powder inhaled imatinib formulation for pulmonary arterial hypertension (PAH), targets abnormal cell growth in lung arteries. Designed for easy inhalation, AV-101 aims to improve patient outcomes by enhancing clinical benefits and reducing systemic side effects compared to existing therapies.
• IMPAHCT, a global Phase IIb/III trial for PAH, evaluates AV-101’s efficacy and safety. Phase IIb assesses three doses over 24 weeks, focusing on pulmonary vascular resistance. 
• The Phase III portion of the trial will evaluate patients receiving the optimal dose of AV-101, determined from the Phase IIb results, against a placebo. The primary endpoint for this phase will be the change in 6-min walk distance (6MWD) over 24 weeks compared to the placebo group.

According to DelveInsight’s “Pulmonary Arterial Hypertension (PAH) – Market Insight, Epidemiology and Market Forecast – 2034,” an estimated 88,000 individuals across the 7MM (United States, Germany, France, Italy, Spain, the United Kingdom, and Japan) are affected by PAH as of 2023, posing a significant respiratory challenge. There is a substantial unmet need for more effective treatments in PAH, particularly disease-modifying agents, which are crucial for improving survival rates and enhancing overall disease management. Addressing this unmet need requires continued research and development of innovative therapies that can modify the disease course and improve patient outcomes.

At the American Thoracic Society International Conference 2024, Aerovate Therapeutics, Inc., a clinical-stage biopharmaceutical company dedicated to developing treatments for rare cardiopulmonary diseases, will present a poster detailing the baseline characteristics of patients participating in the dose-ranging Phase IIb segment of the Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial (IMPAHCT) (NCT05036135). The therapeutic potential of imatinib, a tyrosine kinase inhibitor, for treating adult patients with PAH has been demonstrated in randomized controlled trials and case studies. In previous studies, imatinib was administered orally at a dose of 400 mg. Although the treatment showed clinical efficacy, concerns about tolerability arose due to systemic side effects. AV-101 is being developed as a dry powder formulation of imatinib, administered via an inhalation device in two capsules twice daily. This approach aims to achieve equal or greater lung exposure with a lower total dose, thereby minimizing systemic exposure and enhancing tolerability.

In the IMPAHCT trial, the baseline characteristics align closely with recently published Phase III studies on PAH, encompassing parameters such as hemodynamics, 6MWD, functional class, age, and gender. Cardiovascular comorbidities are relatively rare. Despite the majority of participants receiving treatment with three PAH medications, functional impairment and advanced hemodynamics persist. This typical profile of the study population underscores the limitations of current therapies and emphasizes the ongoing unmet need for PAH treatment. As the IMPAHCT trial progresses into Phase III, it is anticipated to yield comprehensive dose-response data, offering valuable clinical insights into the safety and efficacy of AV-101 (imatinib) for PAH patients.

According to the findings from DelveInsight’s “Pulmonary Arterial Hypertension (PAH) – Market Insight, Epidemiology and Market Forecast – 2034,” the PAH market size in the 7MM was approximately USD 5 billion in 2023. Projections suggest a significant surge at a considerable compound annual growth rate (CAGR) throughout the forecast period.