Avalyn Pharma Made Corporate and Clinical Presentations at ATS 2023 Including Results of Long-Term Treatment of Pulmonary Fibrosis with AP01

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May 23, 2023 – SEATTLE — Avalyn Pharma Inc., a clinical-stage biopharmaceutical company developing inhaled therapies to treat life-threatening pulmonary diseases, today presented safety and tolerability data from the ongoing AP01-005 open-label extension (OLE) trial of AP01 (inhaled pirfenidone), an experimental inhaled drug candidate in development for the treatment of pulmonary fibrosis. The oral presentation reviewed safety and tolerability findings from AP01-naïve participants in the OLE trial as well as data from participants who continued into the OLE study from the Phase 1b ATLAS study, some of whom have been on therapy for at least 3 years. The long-term efficacy and tolerability profile of the AP01-005 roll-over participants indicates a durable and consistent response to AP01 therapy.

The OLE enrolled individuals with idiopathic pulmonary fibrosis (IPF) who had completed the ATLAS study of AP01 plus two new cohorts of IPF and progressive pulmonary fibrosis (PPF) patients not previously treated with AP01, who were intolerant of or ineligible for oral antifibrotic medication with no other treatment options. Findings reported at ATS 2023 show that AP01 was associated with fewer reported adverse events in all three cohorts (roll-over, naïve IPF and naïve PPF) compared to historical reports of oral pirfenidone, with few clinically meaningful differences seen in tolerability between the PPF & IPF participants. Specifically, the number of adverse events (AEs) most commonly reported in the published pooled Phase 3 oral pirfenidone studies and that are typically associated with the product (nausea, rash, and diarrhea) occurred at a much lower frequency (<10%) among AP01-005 patients. This long-term safety profile was notable considering that the ATLAS participants were older than the IPF population enrolled in the oral pirfenidone studies.

“The unique design of this OLE study allowed us to offer AP01 to PPF patients who would not otherwise qualify for anti-fibrotic treatment,” explained Felix Woodhead, MD PhD, Senior Medical Director at Avalyn who presented the results. “While PPF progresses much like IPF, it is common for PPF patients to have underlying rheumatological disorders or other conditions that require chronic medication. Multiple treatments may be used, making the patient more prone to adverse reactions to multiple systemic drugs. Inhaled therapy has lower systemic exposure, in turn, minimizing the potential interactions between the products while treating multiple different conditions, improving tolerability. The PPF patients who participated in our OLE have been able to tolerate the targeted inhaled delivery of AP01 even with a variety of background therapies. Given the favorable safety and efficacy findings among the PPF patients as well as the two cohorts of IPF participants, we intend to continue the OLE study indefinitely to enable these patients to maintain their treatment regimen.”

Earlier at ATS 2023 Lyn Baranowski, CEO of Avalyn participated in a company showcase during the Respiratory Innovation Summit (RIS) where she highlighted the company’s approach to addressing life-threatening pulmonary diseases.

“Our goal in developing AP01 as an inhaled therapeutic is to provide pulmonary fibrosis patients with a formulation of pirfenidone with a long-term tolerability profile that allows patients to stay healthier for longer and also to offer this potentially disease-modifying medication to a broader population of patients,” Ms. Baranowski added. “Tolerability of oral pirfenidone remains suboptimal, with some studies citing up to 50% discontinuation within one year. Anecdotal reports speak to significant challenges managing patient nausea, diarrhea, and vomiting on oral pirfenidone. We continue to be encouraged by the long-term safety and efficacy data generated to date with some patients on AP01 for beyond three years. This, suggests that AP01 may offer a better and more durable safety and efficacy profile compared to oral medication.”

The company’s presentation took place on May 22 in the American Thoracic Society (ATS) Mini Symposium titled “Emerging Data on Disease and Symptom Based Therapeutics for Patients with IPF.” In addition to the oral presentation, an e-poster titled “Long-term Safety Data of Inhaled Pirfenidone (AP01) in Idiopathic Pulmonary Fibrosis (IPF) and Non-IPF Interstitial Lung Disease (ILD) Patients” is available on the ATS 2023 virtual meeting platform and will be made available on the Avalyn website following the conclusion of the ATS conference.

About AP01 (Pirfenidone Solution for Inhalation)
Pirfenidone is a small molecule shown to inhibit fibroblast differentiation and extracellular matrix (ECM) production. AP01 is an inhaled aerosol formulation of pirfenidone that is delivered using an optimized eFlow® Technology Nebulizer, a high efficiency vibrating membrane nebulizer similar in design and operation to PARI’s 510(k) cleared/FDA-approved eFlow-based nebulizers marketed with other products for other disease indications. This administration method allows a smaller pirfenidone dose to be delivered as a soft mist directly to the lung, maximizing pirfenidone’s effect on diseased lung tissue while sparing tissue outside of the lungs from the debilitating toxicities associated with oral delivery. Avalyn is planning to advance AP01 (100 mg BID) into the next phase of the clinical program in 2023.

About Avalyn Pharma
Avalyn is a biopharmaceutical company developing targeted therapeutics for the treatment of rare respiratory diseases including idiopathic pulmonary fibrosis (IPF) and other interstitial lung diseases (ILD). ILDs are characterized by scarring, decline in lung function, reduced exercise capacity and quality of life, and are associated with increased mortality. Currently approved therapeutic options slow ILD progression but are associated with significant toxicities, which restrict their use and dosing. Avalyn is developing a pipeline of inhaled therapeutics designed to reduce systemic exposure and deliver medication to the site of disease. AP01, Avalyn’s lead candidate, is an inhaled formulation of pirfenidone optimized for delivery via inhalation. In a recent clinical study of two doses assessed in 91 individuals with IPF, AP01 demonstrated the potential to improve both efficacy and safety over existing therapy. More information can be found at