Aerodone (aerosol pirfenidone) / Avalyn Pharma - LARVOL DELTA

Inhaled Pirfenidone as an innovative therapeutic approach to treat autoimmune ILD and other forms of Progressive Pulmonary Fibrosis: Phase 2b Study Design (EULAR 2025) - "Patients will remain on stable background immunosuppression and up to 30% of patients will remain on background nintedanib therapy. MIST is the first study to analyze the novel concept of inhaled medications in autoimmune ILD. Safety and efficacy of AP01 in patients with PPF will be studied. In addition to the safety and efficacy endpoints, MIST will examine the presence of cough in this population of patients."

P2b data • Cough • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Interstitial Lung Disease • Pulmonary Disease • Respiratory Diseases

Monte Carlo External Control Arm Generation Utilising Real-world Patient Data and Deep Learning-based Quantitative CT Metrics Demonstrates Treatment Effect in the Atlas IPF Trial (ATS 2025) - P1 | "We have shown a significant treatment effect for 100 mg BID of AP01 over 48 weeks, using external control arms generated by MCCV, based on matched clinical and quantitative CT-based variables. This represents the first known application of quantitative CT in creating a matched external control arm utilising these methods, signalling a paradigm shift in the investigation of novel drug candidates for IPF in clinical trials."

Clinical • Late-breaking abstract • Real-world • Real-world evidence • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology

Dose-Dependent Change of Inhaled Pirfenidone Seen in Lung Volume and Fibrosis Quantification in Patients With IPF: A Deep Learning Image-Based Analysis of Data From the ATLAS Phase 1b Trial (ATS 2025) - P1 | "Patients ineligible for, intolerant of, or unwilling to start oral pirfenidone or nintedanib were randomised 1:1 to receive nebulised AP01 50mg QD or 100mg BID for up to 72 weeks... Patients on the higher dose regime of 100mg AP01 showed a smaller lung volume reduction at week 36 and reduced fibrosis progression at week 24 compared to the 50mg dose. There was a trend across both time points to less fibrosis extent progression in the higher dose regime. Deep learning-based imaging biomarkers can be used to quantify dose-dependent effects on disease progression and suggest a dose-dependent effect of AP01 in patients with IPF."

Clinical • P1 data • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology

Long-term Safety and Efficacy Data With Inhaled Pirfenidone (AP01) in the Atlas Open-label Extension Study Up to 240 Weeks (ATS 2025) - "Conclusions Over up to 240 weeks follow up, AP01 appears to have continuing efficacy with a stable safety profile over the long-term of open-label treatment. This data provides support for the ongoing evaluation of AP01 in Phase 2 and 3 studies."

Clinical • Cough • Idiopathic Pulmonary Fibrosis • Immunology • Interstitial Lung Disease

Title: Dose-Dependent Change of Inhaled Pirfenidone Seen in Lung Volume and Fibrosis Quantification in Patients With IPF: A Deep Learning Image-Based Analysis of Data from the ATLAS Phase 1b Trial (GlobeNewswire) - P1b | N=100 | ATLAS (ACTRN12618001838202) | Sponsor: Avalyn Pharma Pty Ltd | "Avalyn showcases clinical data across multiple pulmonary fibrosis programs at ATS 2025....Together with Qureight, Avalyn presented results from deep learning-based analyses of HRCT scans and patient data from the completed ATLAS trial of AP01. The Qureight platform analyzed HRCT scans from patients with IPF from the ATLAS study to quantify the volumes of the lungs, airways, pulmonary vessels, and fibrosis. Across all visits, HRCT-derived total lung volume strongly correlated with the forced vital capacity (FVC). Between the 50 mg QD and 100 mg BID cohorts, patients on the higher dose showed a smaller lung volume reduction at week 36 and reduced fibrosis progression at week 24 compared to the 50 mg QD dose. These data demonstrate dose-dependent effects of AP01 on anatomy through Qureight’s deep learning-based imaging biomarkers and efficacy as measured by FVC."

P1 data • Idiopathic Pulmonary Fibrosis

Title: Long-term Safety and Efficacy Data with Inhaled Pirfenidone (AP01) in the ATLAS Open-label Extension Study Up to 240 Weeks (GlobeNewswire) - P1b | N=100 | ATLAS (ACTRN12618001838202) | Sponsor: Avalyn Pharma Pty Ltd | "Avalyn showcases clinical data across multiple pulmonary fibrosis programs at ATS 2025....Avalyn’s open-label extension (OLE) trial enrolled a total of 100 patients, including 41 patients rolled over from the ATLAS study, 31 AP01-naïve patients with IPF, and 28 AP01-naïve patients with progressive pulmonary fibrosis (PPF)....Data from roll-over patients from the ATLAS study had an FVC change of -69 ml/year; naïve IPF patients had a FVC change of -120 ml/year, while naïve PPF patients had a FVC change of +11 ml/year. AP01 has continued to demonstrate a well-tolerated profile, with no new safety signals seen during weeks 144 to 240. These positive findings, demonstrating durable efficacy and stable safety up to approximately 4.5 years, are unprecedented as the median survival of pulmonary fibrosis is 3-5 years after diagnosis."

P1 data • Idiopathic Pulmonary Fibrosis

Title: Monte Carlo External Control Arm Generation Utilizing Real-world Patient Data and Deep Learning-based Quantitative CT Metrics Demonstrates Treatment Effect in the ATLAS IPF Trial (GlobeNewswire) - "Avalyn showcases clinical data across multiple pulmonary fibrosis programs at ATS 2025....Avalyn, in collaboration with Qureight, presented a late-breaking poster presentation on findings from a Monte Carlo Cross Validation (MCCV) approach to generate external placebo control arms using real-world data of treatment-naive IPF patients. The approach produced well-matched external placebo control arms for both treatment groups. The results, comparing the change in FVC between these external placebo control arms and the treatment arms, demonstrated a significant treatment effect for 100 mg BID over 48 weeks. This represents the first known application of quantitative HRCT in creating a matched external placebo control arm, signaling a potential paradigm shift in the investigation of novel drug candidates for IPF in clinical trials."

Real-world • Idiopathic Pulmonary Fibrosis

Avalyn Announces Multiple Presentations on AP01 and AP02 for Pulmonary Fibrosis at the American Thoracic Society 2025 International Conference (GlobeNewswire) - "Avalyn Pharma Inc...announced multiple presentations at the American Thoracic Society (ATS) 2025 International Conference being held May 16-21, 2025, at the Moscone Center in San Francisco, CA...Avalyn’s presentations will include topline data from the single- and multiple-ascending dose Phase 1 clinical trials of AP02 (inhaled nintedanib) in healthy adult volunteers and patients with idiopathic pulmonary fibrosis (IPF) and new long-term safety and efficacy data from the ATLAS open-label extension trial of AP01 (inhaled pirfenidone) in patients with IPF and progressive pulmonary fibrosis (PPF). Furthermore, the company, in collaboration with Qureight, will present results from two deep learning-based analyses of high-resolution computed tomography (HRCT) scans and patient data from the Phase 1b ATLAS trial of AP01, exploring the utility of novel imaging biomarkers and synthetic study arms in validating treatment efficacy in patients with IPF."

Clinical data • Idiopathic Pulmonary Fibrosis

An Extension Study of Subjects Who Received an Avalyn Inhaled Antifibrotic Agent (SAIL) (clinicaltrials.gov) - P2 | N=340 | Not yet recruiting | Sponsor: Avalyn Pharma Inc.

New P2 trial • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

Qureight and Avalyn launch strategic partnership in pulmonary fibrosis (PharmaTimes) - "Qureight Ltd and Avalyn Pharma Inc have announced a strategic partnership in progressive pulmonary fibrosis (PPF). The partnership begins with the MIST study, a phase 2b clinical trial evaluating the safety and efficacy of AP01 (inhaled pirfenidone) in PPF patients....This partnership marks the first time deep-learning imaging technologies have been used in a PPF clinical study. Qureight’s technology will assess a MIST study clinical outcome measure. Qureight’s Core Imaging Platform technology will also be extended across multiple Avalyn studies."

Commercial • Idiopathic Pulmonary Fibrosis

Avalyn Announces Upcoming Presentations on its Lead Program AP01, Inhaled Pirfenidone, for the Treatment of Pulmonary Fibrosis at the 22nd International Colloquium on Lung and Airway Fibrosis (GlobeNewswire) - "Avalyn Pharma Inc...announced multiple upcoming presentations at the 22nd International Colloquium on Lung and Airway Fibrosis (ICLAF). The congress will be held October 12-16, 2024, in Athens, Greece. Avalyn’s poster presentations at the ICLAF 2024 will include post-hoc analyses on Brainomix’s e-Lung biomarkers as well as association between functional improvement and anatomical changes from the completed Phase 1b ATLAS trial evaluating AP01 in patients with idiopathic pulmonary fibrosis (IPF)."

P1 data • Fibrosis • Idiopathic Pulmonary Fibrosis • Respiratory Diseases

Avalyn Announces Initiation of its Global Phase 2b MIST Study of AP01 Inhaled Pirfenidone for Progressive Pulmonary Fibrosis (GlobeNewswire) - "Avalyn Pharma Inc...announced the initiation of the MIST Study, a Phase 2b clinical trial evaluating the safety and efficacy of AP01 (inhaled pirfenidone) in patients with progressive pulmonary fibrosis (PPF)."

Trial status • Fibrosis • Idiopathic Pulmonary Fibrosis • Respiratory Diseases

Avalyn Announces Upcoming Presentations on its Lead Program AP01, Inhaled Pirfenidone, for the Treatment of Pulmonary Fibrosis at the European Respiratory Society International Congress 2024 (GlobeNewswire) - "Avalyn Pharma Inc...announced multiple upcoming presentations at the European Respiratory Society (ERS) International Congress 2024. The congress will be held September 7-11, 2024, in Vienna, Austria. Avalyn’s poster presentations at the ERS International Congress 2024 will include the trial design of a Phase 2b MIST study for AP01 (inhaled pirfenidone) for the treatment of progressive pulmonary fibrosis (PPF), and post-hoc analyses from the ATLAS trial evaluating AP01 in patients with idiopathic pulmonary fibrosis (IPF)."

Clinical protocol • Retrospective data • Fibrosis • Idiopathic Pulmonary Fibrosis • Respiratory Diseases

Differences in E-lung Biomarker Scores Between Treatment Groups in Post-hoc Analysis of the ATLAS Inhaled Pirfenidone Solution (AP01) for IPF Clinical Trial (ATS 2024) - "When using imaging biomarkers to assess the efficacy of the intervention, treatment with nebulised pirfenidone at 100mg twice daily was associated with a numerically smaller change of WRVS on serial CT compared to the lower dose group mirroring the same trends in FVC change. These data and ongoing research will assist in identifying the optimum role for AI imaging tools to enrich clinical trials for progressive patients, to facilitate matching treatment arms and to explore novel trial end points."

Biomarker • Clinical • Retrospective data • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology

Safety and Efficacy of Inhaled Pirfenidone (AP01) in the Atlas Open-label Extension Study (ATS 2024) - "Conclusions. At 180 weeks follow up, AP01 appears to have continuing efficacy with a safety profile superior to oral pirfenidone."

Clinical • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Interstitial Lung Disease • Pulmonary Disease • Respiratory Diseases

Avalyn Presents Clinical Data on AP01 for Pulmonary Fibrosis, Including Long-Term Efficacy and Imaging Biomarker Data, at ATS 2024 (GlobeNewswire) - P1b | N=91 | ATLAS (ACTRN12618001838202) | Sponsor: Avalyn Pharma Pty Ltd | "Poster #: 1244....Avalyn conducted a post-hoc analysis of disease progression in the ATLAS Phase 1b study in patients with IPF, which demonstrated that AP01 reduced the risk of disease progression, as defined by decline of greater than 10% FVC in patients with IPF. Fewer IPF patients in the AP01 100 mg twice-daily dosing arm (21%) experienced greater than 10% FVC decline by 48 weeks compared to the 50 mg once-daily dosing arm (35%). A similar, favorable trend of the AP01 100 mg twice-daily cohort was observed leading up to 48 weeks, at 12- and 24- weeks....These data support Avalyn’s selection of AP01 100 mg twice-daily and 50 mg twice-daily for the Company’s planned Phase 2b clinical trial."

P1 data • Idiopathic Pulmonary Fibrosis

Avalyn Presents Clinical Data on AP01 for Pulmonary Fibrosis, Including Long-Term Efficacy and Imaging Biomarker Data, at ATS 2024 (GlobeNewswire) - P1b | N=91 | ATLAS (ACTRN12618001838202) | Sponsor: Avalyn Pharma Pty Ltd | "Poster #: 1251....Avalyn, together with Brainomix, presented promising data on e-Lung imaging biomarkers in a post-hoc analysis of the ATLAS Phase 1b study in patients with IPF. The imaging analysis utilized Brainomix’s proprietary weighted reticulovascular score (WRVS), an AI-enabled computed tomography (CT) processing software. Findings showed that WRVS strongly and substantially correlated with the risk of future IPF progression. AP01’s 100 mg twice-daily dosing arm was associated with stabilization of IPF with trends to a mean relative decrease in the WRVS and mean relative increase in FVC. These data suggest that e-Lung WRVS may be able to identify the risk of disease progression and identify treatment effects on CT, even when FVC is stable."

Biomarker • P1 data • Idiopathic Pulmonary Fibrosis

Avalyn Presents Clinical Data on AP01 for Pulmonary Fibrosis, Including Long-Term Efficacy and Imaging Biomarker Data, at ATS 2024 (GlobeNewswire) - P=NA | N=100 | "Avalyn Pharma...presented clinical data for AP01, its novel formulation of inhaled pirfenidone, for the treatment of pulmonary fibrosis, supporting the Company’s planned Phase 2b trial in patients with progressive pulmonary fibrosis (PPF)....Poster #: 1242:...Avalyn’s open-label extension study enrolled a total of 100 patients across three cohorts: 41 patients with IPF who rolled over from the company’s ATLAS Phase 1b clinical trial and had been previously treated with AP01....Data from the cohort of roll-over IPF patients who were previously treated with AP01 were out to 180 weeks and showed an annualized change in FVC of -107 ml/year. These findings were substantially lower than the -235 ml/year previously reported with oral pirfenidone across three pivotal trials. Further, data from the cohorts of AP01- naïve IPF and PPF patients were out to 84 weeks and showed an annualized change in FVC of -134 ml/year and -13 ml/year, respectively."

Clinical data • Idiopathic Pulmonary Fibrosis

Avalyn Announces Multiple Presentations on its Lead Program, AP01, for Pulmonary Fibrosis and its Approach to Developing Novel Inhaled Therapies for Lung Diseases at the 2024 American Thoracic Society International Conference (GlobeNewswire) - "Avalyn Pharma Inc...announced multiple presentations at the 2024 American Thoracic Society (ATS) International Conference being held May 17-22, 2024 in San Diego, CA. Avalyn will also be featured in the ATS Industry Theater program, where the Company will share information about its Phase 2b trial evaluating AP01, a novel formulation of inhaled pirfenidone, for the treatment of progressive pulmonary fibrosis."

Clinical protocol • P1 data • Fibrosis • Idiopathic Pulmonary Fibrosis • Respiratory Diseases

Breathing Innovation: Inhaled Pirfenidone for the Treatment of Progressive Pulmonary Fibrosis (ATS 2024) - "This event will provide an overview of Avalyn Pharma, spotlighting its commitment to optimizing drug delivery in pulmonary fibrosis treatment through its pipeline of inhaled antifibrotics. Positive outcomes from the Phase 1b study with inhaled pirfenidone will be highlighted, and insights into the upcoming Phase 2b MIST study, focusing on safety and efficacy in Progressive Pulmonary Fibrosis patients, will be shared."

Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

A Study Evaluating the Safety and Efficacy of AP01 in Participants With Progressive Pulmonary Fibrosis (PPF) (clinicaltrials.gov) - P2 | N=300 | Recruiting | Sponsor: Avalyn Pharma Inc. | Not yet recruiting ➔ Recruiting | N=230 ➔ 300

Enrollment change • Enrollment open • Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

A Study Evaluating the Safety and Efficacy of AP01 in Participants With Progressive Pulmonary Fibrosis (PPF) (clinicaltrials.gov) - P2 | N=230 | Not yet recruiting | Sponsor: Avalyn Pharma Inc.

New P2 trial • Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

Avalyn Presents Data from its AP01 and AP02 Inhaled Therapeutics Programs for Pulmonary Fibrosis (GlobeNewswire) - P1b | N=91 | "Avalyn will also review the results from the Phase 1b trial of AP01 (ATLAS) in participants with idiopathic pulmonary fibrosis (IPF)....An additional Avalyn poster will highlight the safety, tolerability, and efficacy findings from the open-label extension (OLE) following the ATLAS trial. The OLE enrolled ATLAS participants and AP01-naïve pulmonary fibrosis participants, and the data showed a durable and consistent response to AP01, including fewer reported adverse events compared to historical reports of oral pirfenidone."

P1 data • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases

Avalyn Raises $175 Million in Oversubscribed Series C Financing to Advance Inhaled Pulmonary Fibrosis Programs into Later Stage Clinical Studies (GlobeNewswire) - "Avalyn Pharma Inc...announced the closing of an oversubscribed $175 million Series C financing. Avalyn plans to use the funds to continue development of its portfolio of inhalation therapies for interstitial lung disease that improve upon currently approved medications, and advance lead clinical assets, AP01 (inhaled pirfenidone) and AP02 (inhaled nintedanib), into mid-stage clinical trials."

Commercial • Fibrosis • Idiopathic Pulmonary Fibrosis • Respiratory Diseases

Avalyn Pharma Presents Data Showing Favorable Tolerability and Potential of AP01 to Preserve Forced Vital Capacity in Adults with Pulmonary Fibrosis (GlobeNewswire) - P1b | N=91 | "Avalyn Pharma Inc...demonstrating that treatment with AP01 (inhaled pirfenidone) preserved forced vital capacity in participants with different forms of pulmonary fibrosis in the ongoing AP01-005 open label extension study, which followed the successful Phase 1b ATLAS study in idiopathic pulmonary fibrosis (IPF) patients. Forced vital capacity (FVC) measures the amount of air a person can forcibly exhale and is used by physicians to monitor progression of pulmonary fibrosis."

P1 data • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Respiratory Diseases