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Pirfenidone is a small molecule inhibitor that attenuates profibrotic pathways, including TGF-β1, which is implicated in fibroblast differentiation and ECM production. In its oral formulation, however, pirfenidone is a low-potency drug that requires a very large dose to deliver sufficient levels to the lung. These doses are associated with adverse events, including GI pain, diarrhea, dyspepsia, photosensitivity, rash, fatigue, and elevated liver enzymes, which can lead to liver damage. Due to these systemic toxicities, prescribing is limited, and compliance is often poor, reducing the product’s potential benefit.

AP01 Medical device photo

Avalyn has reformulated pirfenidone for inhaled aerosol lung delivery via an approved handheld eFlow® vibrating membrane nebulizer. This aerosolized formulation allows small inhaled doses to be delivered as a soft mist directly to the lung, maximizing pirfenidone’s lung dose and effect on diseased lung tissue while sparing tissue outside the lungs from oral-associated drug levels.

Phase 1b ATLAS Study (n=91) complete: 91 participants with Idiopathic Pulmonary Fibrosis were administered one of two doses of AP01 (50 mg once daily and 100 mg twice daily). Results in brief: AP01 was dose-responsive, wherein the 100 mg BID dose achieved near-stabilization of lung function, on average, over 48 weeks of treatment. AP01 was well tolerated with fewer systemic adverse events vs. historic oral pirfenidone. Adverse events were generally mild, with mild cough being the most common. A fully-enrolled open label study is on-going wherein the first enrolled participants have been on therapy for greater than four years.