Avalyn Showcases AP01 Pulmonary Fibrosis Research at ERS 2024

Avalyn Pharma Inc., a biopharmaceutical firm specializing in inhaled treatments for severe pulmonary diseases, has highlighted the framework of its new Phase 2b MIST trial of AP01, an innovative inhaled pirfenidone formulation, targeting patients with progressive pulmonary fibrosis (PPF). Additionally, the company shared post-hoc analyses from its completed Phase 1b ATLAS trial of AP01 in idiopathic pulmonary fibrosis (IPF) patients. These insights were presented through various posters at the European Respiratory Society (ERS) International Congress 2024, held from September 7-11 in Vienna, Austria.

Dr. Howard M. Lazarus, Avalyn’s Chief Medical Officer, expressed optimism about advancing AP01's development and its potential transformative impact on PPF patients. He emphasized that the post-hoc analyses of the ATLAS Phase 1b trial have provided crucial insights into disease progression and informed the strategy for the ongoing Phase 2b MIST trial. Dr. Lazarus shared his enthusiasm about future updates on their progress.

Dr. Jonathan Goldin of UCLA highlighted the urgent need for new treatments for pulmonary fibrosis that are effective and suitable for long-term use. He noted the promising initial results from the ATLAS study, which indicated that AP01 could meet these needs. The study’s high-resolution CT scan analyses have offered new insights into the disease's pathobiology and progression, essential for developing new treatments like AP01 that could improve patient outcomes.

Avalyn's Phase 2b MIST study is a global, randomized, double-blinded, placebo-controlled trial expected to enroll 300 PPF patients. Participants are randomized to receive either 100 mg or 50 mg of AP01, or a placebo, administered twice daily by inhalation. The primary endpoint is the change from baseline in forced vital capacity (FVC) at Week 52, with secondary endpoints including changes from baseline in a quality-of-life questionnaire, time to disease progression, fibrotic scores via quantitative high-resolution computed tomography (HRCT), and safety outcomes. The study also evaluates cough, both in terms of PPF and AP01 administration, through cough counts and questionnaires.

In another presentation, Avalyn discussed findings from the ATLAS Phase 1b trial on the relationship between functional improvement (FVC) and lung fibrosis based on HRCT imaging. Among patients with evaluable data at Week 24, approximately two-thirds showed functional improvement, which correlated with a decrease in lung fibrosis. This suggests that AP01 may not only improve FVC but also reduce lung fibrosis.

Another analysis focused on cough, a common IPF symptom, and disease progression from the ATLAS Phase 1b trial. Cough was reported in 24% and 31% of patients receiving 50 mg once-daily or 100 mg twice-daily AP01, respectively. Importantly, there was no increase in cough rates over the study duration. There was a statistically significant difference in annual FVC decline between doses, particularly among those without cough adverse events. This indicates that cough may be linked to disease progression, a relationship that Avalyn plans to further investigate in the ongoing Phase 2b MIST trial.

Avalyn also presented a post-hoc analysis on the correlation between early cough and disease activity measured by HRCT from the ATLAS Phase 1b trial. Patients with early cough showed significant reductions in ground glass opacities, an indicator of early fibrosis inflammation, compared to those without cough. These findings suggest that AP01 may facilitate early disease improvement. Avalyn intends to further study the incidence and onset of cough and its impact on imaging-based fibrosis and ground glass in the ongoing Phase 2b MIST trial.

Avalyn Pharma is committed to developing inhaled therapies for rare respiratory diseases. Pulmonary fibrosis, characterized by lung tissue scarring and declining lung function, represents a significant challenge due to the limited and toxic nature of current treatments. Avalyn's lead program, AP01, demonstrates improved efficacy and safety compared to existing therapies and is currently in the Phase 2b MIST study. Avalyn is also developing AP02, an inhaled nintedanib formulation, for treating idiopathic pulmonary fibrosis (IPF).

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