Pirfenidone

Topline Results from the AP02 (Inhaled Nintedanib) Phase 1 Clinical Trials Among the Company’s Five Poster Presentations 4.5 Year Long-Term Safety and Efficacy Data Supporting AP01 (Inhaled Pirfenidone) from the ATLAS Open-Label Extension Trial to be Presented Avalyn to be featured in ATS Industry Theater Program CAMBRIDGE, Mass., May 06, 2025 (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 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. “We’re proud to showcase a strong portfolio of presentations at the upcoming ATS conference, which reflects the dedication of our team and collaboration with physicians and medical professionals to meet the urgent therapeutic needs in the pulmonary fibrosis community,” said Lyn Baranowski, CEO of Avalyn Pharma. “We look forward to reporting topline data from two Phase 1 clinical trials of AP02, which support its advancement into a Phase 2 trial, and up to 240 weeks of long-term clinical efficacy and safety data on AP01. Our presence at ATS will showcase our belief that we are well-positioned to transform how pulmonary fibrosis will be treated in the future through the advent of optimized inhaled formulations that offer improved safety, efficacy and long-term adherence – a critical must-have given the progressive, chronic nature of this disease.” 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. Avalyn will also be featured in the ATS Industry Theater program, where Joyce Lee, M.D., Professor of Medicine in Pulmonary Sciences and Critical Care Medicine, Director of the Interstitial Lung Disease Program, University of Colorado School of Medicine, an investigator in the Phase 2b clinical trial of AP01, and a steering committee member for AP02, will provide data highlights from the Phase 1 clinical trials of AP02. Avalyn’s abstracts for the conference are available on the ATS 2025 online program and the Company’s event schedule is outlined as follows: ATS 2025 Poster Presentations: Thematic Poster Session A57: Late Breaking Abstracts in Clinical Problems, Sunday, May 18, 2025, 11:30 a.m.-1:15 p.m. PT: Title: A Double-blind, Placebo-controlled, Randomized Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics After Single or Repeat Twice-daily Doses of AP02, a Nebulized Formulation of NintedanibAuthors: Michelle Palacios, Rebecca Boone, Stephen Pham, Howard M. Lazarus, Craig S. Conoscenti, Melissa RhodesPoster #: P1007 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 TrialAuthors: Kiril R. Kirov, Elliott Bussell, Muhunthan Thillai, Felix A. Woodhead, Howard M. Lazarus, Craig S. Conoscenti, Simon L.F. WalshPoster #: P1009 Thematic Poster Session A101: New Approaches to the Monitoring and Treatment of ILD, Sunday, May 18, 2025, 2:15 p.m.-4:15 p.m. PT: Title: Long-term Safety and Efficacy Data with Inhaled Pirfenidone (AP01) in the ATLAS Open-label Extension Study Up to 240 WeeksAuthors: Tamera J. Corte, Sebastien Tilleux, Deepthi K. Nair, Felix A. Woodhead, Hao Bao, Craig S. Conoscenti, Howard M. Lazarus, Margaret L. Wilsher Poster #: 103 Title: Inhaled Nintedanib (AP02) for the Treatment of IPF: Safety, Tolerability, and Pharmacokinetics After Single Ascending Doses to Healthy Subjects and IPF PatientsAuthors: Michelle Palacios, Stephen Pham, Mark Surber, Deepthi K. Nair, Felix A. WoodheadPoster #: 112 Thematic Poster Session C23: On the Horizon: Imaging and Molecular Biomarkers in Fibrotic ILD, Tuesday, May 20, 2025, 9:15 a.m.-11:15 a.m. PT: 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 TrialAuthors: Elliott Bussell, Miguel Monteiro, Fahdi Kanavati, Muhunthan Thillai, Simon L.F. Walsh, Felix A. Woodhead, Howard M. Lazarus, Craig S. ConoscentiPoster #: 601 ATS 2025 Industry Theater Conference Event, Tuesday, May 20, 2025, 12:00 p.m.-12:30 p.m. PT: Title: Breathing New Life: Inhaled Nintedanib and the Future of Pulmonary Fibrosis TreatmentPresenters: Joyce Lee, M.D., Professor of Medicine in Pulmonary Sciences and Critical Care Medicine, Director of the Interstitial Lung Disease Program, University of Colorado School of Medicine; Howard M. Lazarus, MD, FCCP, Chief Medical Officer, Avalyn Pharma About Avalyn PharmaAvalyn 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

HOUSTON--(BUSINESS WIRE)--Tvardi Therapeutics, Inc. (“Tvardi”) (NASDAQ: TVRD), a clinical-stage biopharmaceutical company focused on the development of novel, oral, small molecule therapies targeting STAT3 to treat fibrosis-driven diseases, today announced that an abstract will be presented at the American Thoracic Society 2025 Annual Conference, which is being held May 16-21 in San Francisco. The abstract highlights the role of STAT3 as a master regulator of idiopathic pulmonary fibrosis (IPF). In preclinical studies, TTI-101, Tvardi’s STAT3 inhibitor, suppressed fibrotic markers which are not addressed with currently approved therapies (nintedanib and pirfenidone). Using single-cell RNA sequencing on lung samples from untreated IPF patients and those treated with current approved therapies, McKenna et al. mapped the transcriptional landscape across 40 pulmonary cell types. They found ~60% of IPF-associated dysregulated genes are not addressed by either approved drug, which they termed the “IPF therapeutic gap.” They further identified STAT3 as a dominant regulatory transcription factor both in untreated IPF samples and in the “IPF therapeutic gap.” In addition, ex vivo analysis of human lung slices treated with TTI-101, nintedanib or pirfenidone resulted in greater repression of genes within alveolar fibroblast (key effector cells in fibrosis) with TTI-101 vs current approved therapies. Notably, TTI-101 downregulated genes involved in extracellular matrix production, including collagen genes, which were largely unaffected by nintedanib or pirfenidone. Details of the presentation are as follows: Title: Single Cell Transcriptomics in A Treatment Status Segregated Cohort Exposes a STAT-3-Regulated Therapeutic Gap in Idiopathic Pulmonary Fibrosis Abstract # 11766 Session: C18 – Spatial and Single-Cell Analysis of Lung Disease: Bridging Early Mechanisms to Therapeutic Gaps Date/time: Tuesday, May 20, 2025, 10:03am-10:15am PT (1:03pm-1:15pm ET) Tvardi team members will also be hosting a booth: Booth: CT-11 Date/time: Sunday, May 18, 2025, through Tuesday, May 20, 2025, 10:30 am-3:30 pm PT For additional information, or to register for the conference: https://ats2025.d365.events/ About Tvardi Therapeutics Tvardi is a clinical-stage biopharmaceutical company focused on the development of novel, oral small molecule therapies targeting STAT3 to treat fibrosis-driven diseases with significant unmet need. STAT3 is a central mediator across critical fibrotic signaling pathways that drive uncontrolled deposition, proliferation, survival and immune suppression. STAT3 is also positioned at the intersection of many signaling pathways integral to the survival and immune evasion of cancer cells. The company is conducting Phase 2 clinical trials in fibrosis-driven diseases with high unmet need: idiopathic pulmonary fibrosis (NCT05671835) and hepatocellular carcinoma (NCT05440708). To learn more, please visit tvarditherapeutics.com or follow us on LinkedIn and X (Twitter). Cautionary Statement Regarding Forward-looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Examples of these forward-looking statements include statements concerning the anticipated benefits of Tvardi’s product candidates; its ongoing clinical trials; and other statements regarding management’s intentions, plans, beliefs, expectations or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are subject to a number of risks, including, among other things: the uncertainties associated with Tvardi’s product candidates, as well as risks associated with the clinical development and regulatory approval of product candidates, including potential delays in the completion of clinical trials; the significant net losses Tvardi has incurred since inception; Tvardi’s ability to initiate and complete ongoing and planned preclinical studies and clinical trials and advance its product candidates through clinical development; the timing of the availability of data from Tvardi’s clinical trials; the outcome of preclinical testing and clinical trials of the Tvardi’s product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; Tvardi’s plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of Tvardi’s product candidates; the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all; Tvardi’s ability to attract, hire, and retain skilled executive officers and employees; Tvardi’s ability to protect its intellectual property and proprietary technologies; Tvardi’s reliance on third parties, contract manufacturers, and contract research organizations; the possibility that Tvardi may be adversely affected by other economic, business, or competitive factors; risks associated with changes in applicable laws or regulations; those factors discussed in Tvardi’s filings with the Securities and Exchange Commission, including the “Risk Factors” section of the Registration Statement on Form S-4, as amended (File No. 333-283900) initially filed with the Securities and Exchange Commission (the “SEC”) on December 18, 2024 and declared effective by the SEC on February 14, 2025, and Tvardi’s other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. The combined company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.