Avalyn Pharma Reports Data After One Year of Treatment in Ongoing AP01 Open-label Extension Trial in Adults with Pulmonary Fibrosis

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April 11, 2023 – SEATTLE — Avalyn Pharma Inc., a clinical-stage biopharmaceutical company developing inhaled therapies to treat life-threatening pulmonary diseases, today reported top-line results from the company’s ongoing open-label extension (OLE) trial for investigational drug AP01 (inhaled pirfenidone). The study (AP01-005) enrolled a total of 41 individuals with idiopathic pulmonary fibrosis (IPF) who had completed the AP01-002 Phase 1b ATLAS study. In addition, Avalyn enrolled two new cohorts of patients who had diagnoses of either IPF or progressive pulmonary fibrosis (PPF), a poorly served segment of the ILD market with high mortality rates. Top-line results for all cohorts after 48 weeks of open-label treatment are consistent with the tolerability and efficacy findings reported in the ATLAS study, data from which were recently published in the journal, Thorax.

Lyn Baranowski, Avalyn’s CEO, explained, “Oral pirfenidone is not approved for patients with PPF; yet the pathology and progression of the condition is similar to that of IPF, leading us to expect AP01 to show a treatment effect in both patient groups. We are encouraged to see a consistency in AP01’s tolerability and efficacy profile in participants with IPF and PPF and look forward to opening future clinical studies for participants with both diagnoses.”

All participants in the AP01-005 OLE were administered with 100 mg BID AP01 using an optimized eFlow® Technology Nebulizer (PARI GmbH, Germany). Spirometry data are available at Week 48 for 36 of the 59 AP01-naïve participants, 18 of whom were diagnosed with IPF and 18 with PPF. These findings showed a trend towards disease stabilization consistent with what was observed in the ATLAS study at the same time point. Over the 48 weeks, mean change in forced vital capacity (FVC) % predicted, a measure of lung capacity, compared to OLE Day 0 was -0.9% for the PPF cohort and -2.2% for the newly enrolled IPF cohort. Generally, AP01 was well tolerated with the most common treatment-related adverse events being cough, fatigue and oropharyngeal pain.

From the literature, it is evident that pirfenidone can extend the lives of patients with pulmonary fibrosis; but published real-world evidence suggests that up to 50% of patients are intolerant of the most effective oral dose and almost 30% are intolerant of oral pirfenidone at any dose level. Thus, the AP01-005 study, which was initiated in 2021, provides a much-needed opportunity to evaluate the long-term effects of inhaled pirfenidone in patients, some of whom have now been on AP01 for more than three years, showing a potential for long-term tolerability and efficacy.

Ms. Baranowski concluded, “Our ultimate goal in developing a pipeline of inhaled medications is to enable patients with life-threatening lung diseases to remain healthy longer, which can only be accomplished with long-term treatment. The tolerability profile of AP01 may allow patients with IPF and PPF to remain on medication longer than they can tolerate the oral systemic compound.”

Avalyn has been selected to present findings from the AP01-005 open-label extension study in an oral presentation at the American Thoracic Society (ATS) annual meeting. Avalyn plans to present additional details from this and other studies during medical congresses in 2023.

About the ATLAS Study

The open-label ATLAS study enrolled 91 adults with IPF who were intolerant of or ineligible for oral pirfenidone or nintedanib. The ATLAS study assessed the safety, tolerability and efficacy of two AP01 doses (50 mg QD or 100 mg BID) in patients with IPF. Of the 91 participants who enrolled, 77 (85%) completed 24 weeks of treatment. Following review of week 24 data, the DSMB recommended all participants transition to the higher dose of AP01. Dosing continued in the ATLAS study through week 72, after which all participants were eligible to participate in the AP01-005 open-label extension study.

Of the 55 who reached week 72, 41 (75%) continued into the open-label extension. An additional 59 adults with either IPF or PPF were recruited to participate in AP01-005. To date, 100 individuals have enrolled in the open-label extension, eight of whom have been on study medication for over three years.

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 Idiopathic and Progressive Pulmonary Fibrosis

Idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) are both associated with the unregulated production of fibrotic tissue in the scaffolding of the lungs, reducing the lung’s flexibility, making breathing difficult and reducing the absorption of inhaled oxygen into the blood. In some cases, the cause of pulmonary fibrosis is known and may be due to toxins, medications, radiation treatment, or connective tissue diseases. When the cause is unknown, the disease is referred to as IPF.

IPF is a chronic, irreversible, progressive interstitial lung disease (ILD) that affects approximately 100,000 people in the U.S. Untreated, IPF is associated with a median survival of 2-5 years after diagnosis. Almost twice as many people in the US are diagnosed with PPF, which has a similar median survival to that of IPF.

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