A Multinational, Long-Term, Safety and Tolerability, Open-Label Extension Study of Subjects Who Have Participated in Avalyn Pharma Studies of Inhaled Antifibrotic Agents (AP-LTE-008 [SAIL])

This is an open-label extension study for participants who were previously enrolled in and completed an Avalyn Pharma Sponsored study with an inhaled antifibrotic, such as AP01. Eligible participants will have their final dose of drug at the end of study visit from the lead-in study and first AP-LTE-008 study visit on the same day.
All participants will receive 100 mg inhaled pirfenidone inhalation solution (AP01) taken twice daily using the eFlow Nebulizer System for until such a time that the drug is approved, the participant withdraws from the study, or the study is terminated.
New patients will be trained to use the nebuliser at the first treatment visit. All patients will use the eFlow nebuliser to administer AP01. Hands on training for use and cleaning of the eFlow nebuliser will be performed by site personnel with patients using the study specific instructions for use and quick reference guide provided. The first treatment for new patients will be overseen by clinic personnel.
A paper dosing diary will be used to monitor adherence along with returns of any unused investigational product.

Start Date30 Apr 2025

Sponsor / Collaborator

Avalyn Pharma, Inc.

ACTRN12624000825550

/ CompletedPhase 1

A Phase 1 randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of AP02 (nintedanib solution for inhalation) delivered via the eFlow® Nebulizer System in healthy volunteers

Start Date24 Jul 2024

Sponsor / Collaborator

Avalyn Pharma, Inc.

NCT06329401

/ RecruitingPhase 2

A Randomized, Double-Blind, Placebo-Controlled, Phase 2b Study Evaluating the Safety and Efficacy of Pirfenidone Solution for Inhalation (AP01) in Participants With PPF

A randomized, double-blind, placebo-controlled clinical study to evaluate the safety and efficacy of 2 doses of inhaled pirfenidone (AP01) versus placebo on top of standard of care in participants with PPF over 52 weeks.

Start Date03 Apr 2024

Sponsor / Collaborator

Avalyn Pharma, Inc.

[+1]

100 Clinical Results associated with Avalyn Pharma, Inc.

0 Patents (Medical) associated with Avalyn Pharma, Inc.

Literatures (Medical) associated with Avalyn Pharma, Inc.

01 May 2024·B48. NEW TREATMENTS IN DIFFUSE PARENCHYMAL LUNG DISEASE

Safety and Efficacy of Inhaled Pirfenidone (AP01) in the Atlas Open-label Extension Study

Author: Nair, D. ; Corte, T.J. ; Woodhead, F.A. ; Bao, H. ; Lazarus, H.M. ; Wilsher, M.L.

01 May 2024·B48. NEW TREATMENTS IN DIFFUSE PARENCHYMAL LUNG DISEASE

Differences in E-lung Biomarker Scores Between Treatment Groups in Post-hoc Analysis of the ATLAS Inhaled Pirfenidone Solution (AP01) for IPF Clinical Trial

Author: Abou-Zeid, W. ; Gerry, S. ; Sifostratoudaki, A. ; Ottink, F. ; Rennison-Jones, C. ; Lazarus, H.M. ; George, P.M. ; Stewart, R. ; Devaraj, A. ; Woodhead, F.A. ; Harston, G. ; Fernandez, C. ; Nair, D. ; Joly, O.

01 Sep 2023·Thorax

Inhaled pirfenidone solution (AP01) for IPF: a randomised, open-label, dose–response trial

Article

Author: Glaspole, Ian ; Veale, Antony ; Hopkins, Peter ; Wilsher, Margaret L ; Piotrowski, Wojciech ; Raghu, Ganesh ; Shaffer, Michele L ; Wijsenbeek, Marlies S ; Barczyk, Adam ; Chaudhuri, Nazia ; Jassem, Ewa ; Šterclová, Martina ; Grainge, Christopher ; Montgomery, A Bruce ; Freeman, Lisa ; West, Alex ; Corte, Tamera Jo ; Nair, Deepthi ; Otto, Kelly

IntroductionOral pirfenidone reduces lung function decline and mortality in patients with idiopathic pulmonary fibrosis (IPF). Systemic exposure can have significant side effects, including nausea, rash, photosensitivity, weight loss and fatigue. Reduced doses may be suboptimal in slowing disease progression.MethodsThis phase 1b, randomised, open-label, dose–response trial at 25 sites in six countries (Australian New Zealand Clinical Trials Registry (ANZCTR) registration number ACTRN12618001838202) assessed safety, tolerability and efficacy of inhaled pirfenidone (AP01) in IPF. Patients diagnosed within 5 years, with forced vital capacity (FVC) 40%–90% predicted, and intolerant, unwilling or ineligible for oral pirfenidone or nintedanib were randomly assigned 1:1 to nebulised AP01 50 mg once per day or 100 mg two times per day for up to 72 weeks.ResultsWe present results for week 24, the primary endpoint and week 48 for comparability with published trials of antifibrotics. Week 72 data will be reported as a separate analysis pooled with the ongoing open-label extension study. Ninety-one patients (50 mg once per day: n=46, 100 mg two times per day: n=45) were enrolled from May 2019 to April 2020. The most common treatment-related adverse events (frequency, % of patients) were all mild or moderate and included cough (14, 15.4%), rash (11, 12.1%), nausea (8, 8.8%), throat irritation (5, 5.5%), fatigue (4, 4.4%) and taste disorder, dizziness and dyspnoea (three each, 3.3%). Changes in FVC % predicted over 24 and 48 weeks, respectively, were −2.5 (95% CI −5.3 to 0.4, −88 mL) and −4.9 (−7.5 to −2.3,–188 mL) in the 50 mg once per day and 0.6 (−2.2 to 3.4, 10 mL) and −0.4 (−3.2 to 2.3, −34 mL) in the 100 mg two times per day group.DiscussionSide effects commonly associated with oral pirfenidone in other clinical trials were less frequent with AP01. Mean FVC % predicted remained stable in the 100 mg two times per day group. Further study of AP01 is warranted.Trial registration numberACTRN12618001838202 Australian New Zealand Clinical Trials Registry.

News (Medical) associated with Avalyn Pharma, Inc.

01 Apr 2025

·www.globenewswire.com

Avalyn Completes Successful Phase 1 Clinical Trials of AP02, its Novel Inhaled Formulation of Nintedanib, Preparing for Phase 2 Development in Idiopathic Pulmonary Fibrosis

Topline data from the single- and multiple-ascending dose studies confirmed safety and favorable tolerability profile of AP02 at all dose levels Full safety, tolerability, and pharmacokinetics data to be presented at the American Thoracic Society 2025 International Conference in May 2025 Company plans to advance AP02 into a Phase 2 clinical trial in patients with idiopathic pulmonary fibrosis (IPF) CAMBRIDGE, Mass., April 01, 2025 (GLOBE NEWSWIRE) -- Avalyn Pharma Inc., a clinical-stage biopharmaceutical company developing inhaled therapies for the treatment of life-threatening pulmonary diseases, today announced that AP02 (inhaled nintedanib) was safe and well tolerated in multiple single-ascending dose (SAD) and multiple-ascending dose (MAD) Phase 1 clinical trials in healthy adult volunteers and patients with idiopathic pulmonary fibrosis (IPF). IPF is largely untreatable and characterized by scarring of lung tissue, decline in lung function, and reduced exercise capacity and quality of life. There is a clear need to bring treatment options to patients that maximize therapeutic impact while minimizing systemic exposure and transform the disease from a fatal diagnosis into a manageable condition. The most recently completed Phase 1 trial enrolled a total of 60 healthy adult volunteers and consisted of a single-ascending dose (SAD) portion, which assessed AP02 at 2.0 mg, 4.0 mg, and 8.0 mg once-daily (QD); a multi-ascending dose (MAD) portion that evaluated twice-daily (BID) dosing of AP02 at the same dose levels over a seven-day treatment period; and a bronchoalveolar lavage (BAL) portion, which assessed lung exposure following a single 4.0 mg AP02 dose across three timepoints. “We are excited to complete this innovative set of clinical trials for AP02, where we assessed lung exposure in both studies of the inhaled compared to oral nintedanib,” said Lyn Baranowski, Chief Executive Officer of Avalyn. “Through its enhanced drug delivery directly to the lungs, our approach allows us to address the underlying pathophysiology of the disease and positions AP02 as a potential future standard-of-care for patients with IPF.” The company expects to present full topline data from two Phase 1 studies, including safety, tolerability, and pharmacokinetics at the American Thoracic Society 2025 International Conference in May 2025. Based on these results, Avalyn plans to advance AP02 into a Phase 2 clinical program for patients with idiopathic pulmonary fibrosis (IPF). About Avalyn Pharma Avalyn is reimagining the future of pulmonary fibrosis treatment with a pipeline of new inhaled formulations of approved medicines designed to reduce systemic exposure and deliver medication directly to the site of disease. Pulmonary fibrosis is characterized by scarring of lung tissue, decline in lung function, and 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’s inhaled approach tackles the underlying pathophysiology of pulmonary fibrosis at its source and is 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). AP01 has 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. The company completed two Phase 1 studies for its second program, AP02, inhaled nintedanib, for the treatment of idiopathic pulmonary fibrosis (IPF). For more information, please visit avalynpharma.com and follow us on LinkedIn. Investor Contact:Alex Straus, THRUST alex@thrustsc.comir@avalynpharma.com Media Contact:media@avalynpharma.com

Phase 1Phase 2

17 Dec 2024

·www.globenewswire.com

Avalyn to Present at the 43rd Annual J.P. Morgan Healthcare Conference

CAMBRIDGE, Mass., Dec. 17, 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 announced that Lyn Baranowski, chief executive officer of Avalyn, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2025, at 3:00 p.m. PT in San Francisco. Ms. Baranowski, CEO; Douglas Carlson, CFO, CBO; Howard Lazarus, M.D., FCCP, CMO; and Melissa Rhodes, Ph.D., COO, will also participate in 1x1 meetings with investors. 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). AP01 has 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.comir@avalynpharma.com Media Contact:Marites CoulterDeerfield Groupmarites.coulter@deerfieldgroup.commedia@avalynpharma.com

Phase 2Phase 1

25 Nov 2024

·www.globenewswire.com

Avalyn to Participate in Multiple Upcoming Investor Conferences

CAMBRIDGE, Mass., Nov. 25, 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 announced that management will participate in the following upcoming investor conferences in December. 7th Annual Evercore HealthCONx Conference Format: Fireside Chat and 1x1 Meetings Date: Tuesday, December 3, 2024, at 3:50 p.m. ET Location: The Loews Coral Gables Hotel, Coral Gables, FL Participants: Lyn Baranowski, CEO; Douglas Carlson, CFO, CBO; Howard M. Lazarus, CMO Citi’s 2024 Global Healthcare Conference Format: Fireside Chat and 1x1 Meetings Date: Wednesday, December 4, 2024, at 11:00 a.m. ET Location: InterContinental Hotel, Miami, FL Participants: Lyn Baranowski, CEO; Douglas Carlson, CFO, CBO; Howard M. Lazarus, CMO 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). AP01 has 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 ir@avalynpharma.com Media Contact:Marites CoulterDeerfield Groupmarites.coulter@deerfieldgroup.com media@avalynpharma.com

Phase 2Clinical ResultPhase 1

100 Deals associated with Avalyn Pharma, Inc.

100 Translational Medicine associated with Avalyn Pharma, Inc.

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The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

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Drug(Targets)	Indications	Global Highest Phase

Pirfenidone	Progressive fibrotic interstitial lung disease More	Phase 2
Nintedanib esylate ( CSF-1R x FGFRs x FLT3 x PDGFRs x VEGFR )	Idiopathic Pulmonary Fibrosis More	Phase 1
Pirfenidone/Nintedanib	Lung Diseases, Interstitial More	Preclinical
Imatinib mesylate ( ABL x Bcr-Abl x PDGFRs x c-Kit )	Pulmonary Arterial Hypertension More	Discontinued

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