SEATTLE–(BUSINESS WIRE)–Avalyn Pharma Inc., a biopharmaceutical company focused on development of improved therapies for life threatening pulmonary diseases, today announced completion of last patient in the initial 24 week evaluation of a Phase I/II clinical study of two dose regimens of AP01 (a formulation of pirfenidone optimized for delivery via inhalation) in patients with idiopathic pulmonary fibrosis (IPF). Ninety-one patients with IPF were randomized to 50 mg once daily (n=46) or 100 mg twice daily (n=45), administered by a PARI investigational eFlow® nebulizer. During the first 24 weeks, the high dose group had a trend toward stabilization of lung function as measured by forced vital capacity (FVC). The low dose group had a progressive loss of lung function. Based on these results the data safety monitoring board recommended conversion of all low dose patients to the high dose during the optional 12 month extension which is ongoing. AP01 was safe and well tolerated at both doses. Flu-like symptoms and GI adverse events most commonly attributed to oral pirfenidone were seen in less than 10% of patients treated with AP01. Adverse events with a frequency of greater that 10% were rash 18%, upper respiratory tract infection 18%, cough 24%.
Most subjects have opted to continue in the 12 month extension. Twenty-four week efficacy and safety data will be presented at an upcoming scientific meeting.
“We are pleased by the efficacy and safety profile we have seen to date with aerosolized pirfenidone in 91 patients over 24 weeks. The six month FVC data of the high dose is very promising as well as the overall safety profile to date,” said Dr. A. Bruce Montgomery, CEO of Avalyn Pharma. “Despite the 2014 approval of two oral antifibrotic therapies, IPF and other fibrotic lung diseases remain fatal diseases with substantial unmet need. We hope to establish improved tolerability and long term efficacy with aerosolized pirfenidone in Phase 3 trials to be initiated in 2021.”
IPF and other fibrotic lung diseases are characterized by progressive scarring, reduced exercise capacity and ultimate death from respiratory failure and/or co-morbidities. IPF treatments are relatively new, with the first and only approvals coming in 2014: oral pirfenidone (Esbriet®) and oral nintedanib (Ofev®). Both medicines, while effective in slowing disease progression are associated with significant adverse effects that limit dosing and their full potential for efficacy. While these medicines are an important first step to treat IPF, a substantial unmet need remains for therapies with improved safety profiles and better efficacy as either stand-alone or add-on combination therapies in both IPF and other fibrotic lung diseases.
The Inhaled Advantage
Although oral pirfenidone has shown to slow IPF disease progression, it is a low potency drug requiring a very large oral dose to achieve efficacious lung levels. Unfortunately, oral delivery results in blood levels which cause substantial adverse effects and limit the delivered lung dose. In a Phase I single dose study of AP01, we have demonstrated the potential of aerosolized pirfenidone to improve both efficacy and safety. Inhalation of a dose of AP01 equivalent to 1/16 of the licensed oral dose delivered 35-fold higher peak lung levels of pirfenidone.
About Avalyn Pharma
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