Pirfenidone

PureTech Health unveiled positive Phase 2b data on Monday for its idiopathic pulmonary fibrosis drug, which showed it can reduce lung function decline in patients. The drug hit the trial’s primary and secondary endpoints, with deupirfenidone showing superiority to placebo in slowing the rate of lung function decline in people with IPF as measured by forced vital capacity — defined as the amount of air a person can exhale after taking a breath in — at 26 weeks. Patients taking the investigational drug at 825 mg three times a day saw an 80.9% treatment effect. In contrast, patients taking 801 mg of pirfenidone, a standard-of-care for IPF, saw a 54.1% effect. In a secondary endpoint, there was a 99.6% probability that the pooled deupirfenidone arms of the trial were superior to placebo in slowing the rate of lung function decline when using a Bayesian analysis. Executives on a Monday investor call said that the results supported moving into a Phase 3 trial. CEO Bharatt Chowrira added on the call that based on the data, only one more trial may be necessary before the company seeks approval. PureTech’s stock $PRTC on the Nasdaq was up nearly 10% on Monday. Deupirfenidone, a deuterated form of pirfenidone, is an oral treatment that works by inhibiting IL-6, TNF-α and transforming growth factor beta, according to the National Cancer Institute. As for safety, the most common adverse events included gastrointestinal issues like nausea and dyspepsia, and the only “key” gastrointestinal adverse event seen was abdominal pain, with 14.1% of patients on the drug experiencing the issue. “We’ve looked into it,” PureTech’s VP of medical affairs Camilla Graham told investors. “There’s no signal, no cause for any concern, and we’re continuing to dig into all of these numbers.” There were discontinuations, with 187 out of 257 patients completing the trial: 42 out of 65 patients in the deupirfenidone 550 mg arm and 50 out of 64 patients in the 825 mg arm. Of the patients who completed the trial, 170 enrolled in the open-label extension portion of the trial. In response to a question about discontinuations from Endpoints News , a representative for the company said they “have just received these data and our primary focus has been assessing efficacy and the key GI adverse events,” adding that “there is nothing in the data to give us cause for concern.”

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