Trial Design Showcased for the MIST Study, a Recently Initiated Phase 2b Clinical Trial, Evaluating the Efficacy and Safety of AP01 (inhaled pirfenidone) Compared to Placebo in Patients with Progressive Pulmonary Fibrosis (PPF) Additional Poster Presentations on Post-hoc Analyses of Safety, Imaging Data, and Disease Progression from the ATLAS Phase 1b Study CAMBRIDGE, Mass., Sept. 08, 2024 (GLOBE NEWSWIRE) — Avalyn Pharma Inc., a clinical-stage biopharmaceutical company focused on development of inhaled therapies for the treatment of life-threatening pulmonary diseases, today showcased the design of its recently initiated MIST Phase 2b trial of AP01, a novel formulation of inhaled pirfenidone, in patients with progressive pulmonary fibrosis (PPF). Avalyn also presented post-hoc analyses from its completed Phase 1b ATLAS trial of AP01 in patients with idiopathic pulmonary fibrosis (IPF). The Phase 2b trial design and Phase 1b data were outlined in multiple poster presentations during the European Respiratory Society (ERS) International Congress 2024 held September 7-11, 2024, in Vienna, Austria. “We are excited to advance the development of AP01 and showcase the design of the ongoing MIST Phase 2b study, which has the potential to be a transformative treatment for patients living with PPF,” said Howard M. Lazarus, M.D., FCCP, Chief Medical Officer at Avalyn. “We also highlight post-hoc analyses of the ATLAS Phase 1b trial, which illuminated key insights into disease progression through its impact on cough and imaging data. These analyses have further informed the strategy and design of our recently initiated MIST Phase 2b trial. We look forward to sharing updates on our progress.” “Patients living with pulmonary fibrosis are in urgent need of new treatments that slow disease progression and are tolerable for long-term use. We are encouraged by the initial results of AP01 from the ATLAS study that demonstrated these important qualities,” said Jonathan Goldin, MD, PhD, Professor of Radiological Sciences and Medicine at the University of California Los Angeles. “Analyses of the high-resolution CT scans in the completed ATLAS trial provide new insights into disease pathobiology and disease progression. These understandings are key to improving our knowledge so that we can contribute to the development of new treatments like AP01 that have the potential to improve patient outcomes.” Title: Inhalation Innovation: Phase 2b Study Design on Inhaled Pirfenidone in the Treatment of Progressive Pulmonary Fibrosis (Session: PS-28, Poster #: PA690) Avalyn presented the design of its ongoing Phase 2b MIST study, a randomized, double-blinded, placebo-controlled, global study, expected to enroll 300 patients with progressive pulmonary fibrosis (PPF). Patients are randomized 2:1:2 to receive AP01 (100 mg or 50 mg) or placebo twice daily by inhalation. Patients can be on background immunosuppressants or oral nintedanib. The primary endpoint is the change from baseline in forced vital capacity (FVC) at Week 52. In addition, secondary endpoints include change from baseline in a quality-of-life questionnaire, time to disease progression, change in fibrotic scores via quantitative high-resolution computed tomography (HRCT), and safety outcomes. Cough, be it related to PPF or the administration of AP01, is analyzed through cough counts and cough questionnaires. Title: Association between Functional Improvement and Structural Changes in Quantitative High-resolution CT in ATLAS: a Phase 1b Study of Inhaled Pirfenidone in Idiopathic Pulmonary Fibrosis (Session: PS-28, Poster #: PA692) Avalyn presented findings on the association between changes in functional improvement (FVC) and quantitative lung fibrosis based on HRCT imaging from the ATLAS Phase 1b trial of AP01 in patients with idiopathic pulmonary fibrosis (IPF). Among patients who had evaluable data at Week 24, approximately two-thirds of patients had functional improvement, as defined as positive changes in FVC. Patients with functional improvement had a correlated decrease in the level of fibrosis, as measured by QLF, based on HRCT imaging. This data suggests that AP01 may have the unique ability to reduce lung fibrosis in addition to providing beneficial changes in FVC. Title: Cough adverse events and FVC decline in the ATLAS study of inhaled pirfenidone (AP01) in Idiopathic Pulmonary Fibrosis (Session: PS-27, Poster #: PA685) Avalyn presented a post-hoc analysis of cough, a common symptom of IPF, and disease progression from the ATLAS Phase 1b trial. Cough occurred in 24% and 31% of patients receiving 50 mg once-daily or 100 mg twice-daily AP01. Notably, there was no increase or progression of cough rates over the duration of the study. There was a statistically significant difference in annual FVC decline between doses in the overall study population, and notably, among a cohort without cough AE. As cough may be associated with disease progression, Avalyn will further examine the presence of cough and its relationship with other outcome measures in its ongoing Phase 2b MIST trial. Title: Correlation of Early Cough with Disease Activity measured by High-Resolution CT in ATLAS: a Phase 1b Study of Inhaled Pirfenidone in Idiopathic Pulmonary Fibrosis (Session: PS-28, Poster #: PA691) Avalyn also presented a post-hoc analysis of the correlation between early cough and disease activity measured by high-resolution computed tomography (HRCT) from the ATLAS Phase 1b trial of AP01 in patients with IPF. Of the patients in the ATLAS study with evaluable HRCT at Week 24, participants with early cough (n=10) showed significant reductions in ground glass (QGG), which can represent an inflammatory process related to early fibrosis, compared to those without cough. There were no statistically significant differences between the various onsets of cough and lung fibrosis (QLF) or cough and honeycombing (QHC). As AP01 (inhaled pirfenidone) is suspected to have both anti-inflammatory and anti-fibrotic properties, these findings suggest that AP01 may be associated with early improvement of disease. Avalyn will further examine the incidence and the onset of cough and impact on imaging-based fibrosis and ground glass in its ongoing Phase 2b MIST trial. About Avalyn PharmaAvalyn is a biopharmaceutical company developing inhaled therapies for the treatment of rare respiratory diseases including pulmonary fibrosis and other interstitial lung diseases (ILD). Pulmonary fibrosis is characterized by scarring of lung tissue, decline in lung function, reduced exercise capacity and quality of life, and is associated with increased mortality. Currently approved therapeutic options slow pulmonary fibrosis progression but are associated with significant toxicities that restrict their use and dosing. Avalyn is developing a pipeline of new inhaled formulations of approved medicines designed to reduce systemic exposure and deliver medication directly to the site of disease. Avalyn’s lead program, AP01, is an optimized inhaled formulation of pirfenidone, currently being studied in the ongoing MIST Phase 2b Study in progressive pulmonary fibrosis (PPF). AP01has been assessed in over 150 individuals with different forms of pulmonary fibrosis and demonstrated clinical proof-of-concept with improved efficacy and safety compared to historical data with existing therapies. Avalyn has initiated a Phase 1b study for its second program, AP02, inhaled nintedanib, that is being developed for the treatment of idiopathic pulmonary fibrosis (IPF). For more information, please visit avalynpharma.com and follow us on LinkedIn. Investor Contact:Alex StrausTHRUST Strategic Communicationsalex@thrustsc.com