• Aerami Therapeutics showcased their “uniPHied” Phase II trial design, enabling efficient evaluation of AER-901 across multiple pulmonary hypertension types in a single study. This trial will assess AER-901’s safety and efficacy in PH-ILD and PAH, potentially expediting its development.
• AER-901 achieved over twofold higher lung deposition and three-fold greater peripheral lung deposition compared to DPI-imatinib, suggesting effective treatment at lower doses.

Aerami Therapeutics presented findings related to AER-901, a proprietary liquid formulation of imatinib for inhalation administered via a high-performance, handheld, smart nebulizer, at the American Thoracic Society (ATS) 2024 International Conference, held on May 19–20, 2024. The presentations highlighted new lung deposition modeling data comparing AER-901 to a hypothetical dry powder inhaled (DPI)-imatinib in subjects with pulmonary hypertension associated with interstitial lung disease (PH-ILD) and pulmonary arterial hypertension (PAH), as well as healthy volunteers. These data were generated by FLUIDDA, Inc., using their Functional Respiratory Imaging (FRI) and Rapid Deposition Analysis (RDA) platforms. Additionally, Aerami showcased the innovative design of the “uniPHied” platform Phase II trial, aimed at evaluating AER-901 across multiple pulmonary hypertension cohorts.

AER-901 is designed to efficiently deliver imatinib therapy deep into the diseased tissues of the lung, using a proprietary liquid formulation of imatinib administered by a breath-activated, high-performance, handheld, smart nebulizer. This nebulizer controls the flow rate and provides real-time feedback to optimize lung deposition. AER-901 is currently in clinical development for PH-ILD and PAH, two rare and serious forms of pulmonary hypertension with a high unmet medical need for improved treatment options.

Key findings from the lung deposition study revealed that AER-901 resulted in more than a twofold increase in overall lung (intrathoracic) deposition compared to DPI-imatinib, with over threefold greater deposition in the peripheral lung regions. These results were consistent across all populations evaluated, suggesting enhanced potential for AER-901 to achieve efficacy in pulmonary hypertension at lower, more tolerable doses.

The “uniPHied” platform study design allows for the efficient evaluation of AER-901 in multiple forms of pulmonary hypertension within a single trial. This Phase II, placebo-controlled, randomized, parallel-group, proof-of-concept trial will assess the safety and efficacy of AER-901 in PH-ILD and PAH, with the potential to expand to other forms of pulmonary hypertension. Participants will be randomized to receive either AER-901 or a placebo twice daily, with a primary endpoint of a percentage reduction in pulmonary vascular resistance after 24 weeks of treatment. Secondary endpoints include changes in mean pulmonary arterial pressure, N-terminal pro-B-type natriuretic peptide levels, 6-min walk distance, multicomponent improvement, and quality of life.

In conclusion, the emergence of AER-901 represents a promising advancement in the treatment of multiple pulmonary hypertension cohorts (PH-ILD and PAH). Its novel mechanism of action and efficient lung deposition capabilities offer the potential for significant clinical benefits at lower doses, addressing the high unmet needs of patients with PH-ILD and PAH. The innovative “uniPHied” trial design further underscores Aerami’s commitment to expeditiously and efficiently bringing AER-901 to patients in need, with the potential to transform the treatment landscape for pulmonary hypertension.