Pirfenidone

CAMBRIDGE, Mass., May 22, 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 management will participate in the Jefferies Global Healthcare Conference on June 3, 2025. Format:1x1 Meetings Date:Tuesday, June 3, 2025 Location:New York Marriott Marquis, New York, NY Participants:Douglas Carlson, CFO & CBO; Craig Conoscenti, SVP, Clinical Development About Avalyn PharmaAvalyn is reimagining the future of pulmonary fibrosis treatment with a pipeline of new inhaled formulations of approved medicines. 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’s inhaled approach tackles the underlying pathophysiology of pulmonary fibrosis at its source by safely delivering treatments directly into the lungs, thereby improving tolerability by decreasing systemic exposure and improving efficacy by increasing lung exposure. Avalyn’s 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 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 StrausTHRUST Strategic Communicationsalex@thrustsc.comir@avalynpharma.com Media Contact:media@avalynpharma.com

PureTech hopes to launch a phase 3 trial of its deuterated version of Roche’s idiopathic pulmonary fibrosis drug Esbriet by the end of this year. The company showed promising results from a phase 2b trial at the American Thoracic Society International Conference Tuesday in San Francisco. \n One day after Boehringer Ingelheim presented so-so data from a successful trial of its highly touted idiopathic pulmonary fibrosis (IPF) candidate nerandomilast, PureTech has answered with results from a study of its IPF hopeful deupirfenidone.The new data from PureTech come after the company reported successful results from the phase 2b Elevate study in December after 26 weeks of therapy. The new round of preliminary figures highlight the durability of deupirfenidone’s effect through at least 52 weeks of treatment.In the trial, as of May 9, there were 101 patients who had received deupirfenidone for at least 52 weeks. Their lung function decline—as measured by forced vital capacity (FVC)—was at -32.8 mL over the treatment period, which is within the expected range (-30 mL to -50 mL) of natural decline in lung function in older, healthy people.Forced vital capacity is a measure of the amount of air exhaled during a lung capacity test.By comparison, in the first round of results, in patients who had received deupirfenidone for 26 weeks, the FVC decline came in at -21.5 mL. This result was also within the expected natural decline in lung function through the same time period in older, healthy adults, which is between -15 mL and -25 mL, according to PureTech.PureTech says it hopes to meet with the FDA by the end of the third quarter and to initiate a phase 3 trial of deupirfenidone by the end of the year. “The ability for a monotherapy to reduce lung function decline close to a level seen in healthy older adults and to sustain that effect over time without compromising tolerability, is not something we have seen with currently available therapies,” Toby Maher, M.D., Ph.D., the trial’s lead investigator and a professor of medicine at USC, said in a release. “Deupirfenidone has the potential to raise the bar for what patients and physicians can expect from IPF treatment.”The company presented the results Tuesday at the American Thoracic Society International Conference in San Francisco. Deupirfenidone is a deuterated version of Roche’s IPF drug Esbriet (pirfenidone), which achieved blockbuster status each year from 2018 to 2022 before it lost patent protection. Deuteration is a process in which hydrogen atoms in a compound are replaced with a heavier isotope of hydrogen. It can improve the pharmacokinetic properties of existing drugs, increasing their efficacy and safety.The Elevate trial enrolled four treatment arms receiving three daily doses. In addition to the dose of 825 mg of deupirfenidone producing a change in FVC of -21.5 mL at 26 weeks, a 550-mg dose of deupirfenidone was much less effective at -80.7 mL. An 801-mg dose of pirfenidone yielded a 51.6 mL decline in lung function, while the placebo arm saw a -112.5 mL FVC result.Preliminary pharmacokinetic data from the trial, comparing to pirfenidone 801 mg and deupirfenidone 825 mg, resulted in an approximately 50% increase in drug exposure, PureTech said. The increased drug exposure did not result in an increase in tolerability challenges, “suggesting that the deuterated structure of deupirfenidone may overcome the dose-limiting adverse events associated with pirfenidone,” the company added. Boehringer Ingelheim’s long-awaited results presentation Monday came after the company reported the top-line success of its trial in September. In the phase 3 study, patients were on background antifibrotic therapy, with 535 on Boehringer’s Ofev and 380 using Esbriet. Across the entire population, mean changes on FVC at 52 weeks were -114.7 mL on a high dose of nerandomilast and -183.5 mL on placebo.In response to the data, Leerink Partners analysts called the PDE4B inhibitor “incrementally better” than blockbuster Ofev but said it\'s still “disease-slowing rather than disease-halting.” The analysts added the “modest effect size” on lung function “is not likely to encourage an immediate change in IPF treatment.”