Avalyn Pharma Presents Data Showing Favorable Tolerability and Potential of AP01 to Preserve Forced Vital Capacity in Adults with Pulmonary Fibrosis

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September 12, 2023 – SEATTLE — Avalyn Pharma Inc., a clinical-stage biopharmaceutical company focused on development of targeted therapies for life-threatening pulmonary diseases, and collaborators made several presentations at the congress of the European Respiratory Society (ERS), demonstrating that treatment with AP01 (inhaled pirfenidone) preserved forced vital capacity in participants with different forms of pulmonary fibrosis in the ongoing AP01-005 open label extension study, which followed the successful Phase 1b ATLAS study in idiopathic pulmonary fibrosis (IPF) patients. Forced vital capacity (FVC) measures the amount of air a person can forcibly exhale and is used by physicians to monitor progression of pulmonary fibrosis.

Dr. Alex West, interstitial lung disease lead at Guy’s and St Thomas’ Hospital, London and an investigator in the ATLAS study made a podium presentation of 48-week results from the AP01-005 study. Participants with idiopathic pulmonary fibrosis (IPF) who were treated with AP01 experienced a less profound reduction in FVC than has been reported with oral pirfenidone. Adults treated with AP01 also reported markedly lower rates of nausea, rash, diarrhea, dyspepsia and vomiting compared to the published side-effect profile of oral pirfenidone. Mild cough was the most commonly reported adverse event (AE) among ATLAS and AP01-005 participants.

Dr. West also presented a poster that compared findings from AP01-005 participants with progressive pulmonary fibrosis (PPF) to published results of PPF patients treated with oral nintedanib, the only medication currently approved for PPF. It was found that PPF patients treated with AP01 reported substantially lower incidence of nausea, diarrhea, vomiting and decreased appetite than did participants in oral nintedanib studies. Further, consistent with the experience of participants with IPF, the AP01-005 participants with PPF also experienced a slower decline in FVC (-31.7mL) over 48-weeks of treatment than has been reported in the literature for PPF patients administered oral nintedanib (-80.8mL).

Dr. Nazia Chaudhuri, a consultant in respiratory medicine at the Ulster University School of Medicine in Ireland and an investigator in the ATLAS and AP01-005 studies, presented a late-breaking poster describing a meta-analysis that was conducted to compare FVC findings from the IPF patients enrolled in ATLAS to pooled placebo results compiled from four published studies of oral pirfenidone and oral nintedanib in adults with IPF. Dr. Chaudhuri’s analysis suggests that 100mg AP01 BID has superior efficacy when compared to placebo. Specifically, when compared to the placebo arm of the ASCEND study of oral pirfenidone, AP01 would have had a statistically significant benefit in FVC (p=0.0007) vs placebo. Similarly, statistical significance would also be observed vs the placebo arms of the INPULSIS-1, INPULSIS-2 and INBUILD studies of oral nintedanib.

“The consistent trend towards stabilization of lung function across the ATLAS and AP01-005 studies indicate to us that AP01 is reaching the lungs with sufficient concentrations and is having the intended effect in participants with both idiopathic and progressive forms of pulmonary fibrosis,” explained Howard M. Lazarus, M.D. FCCP, Avalyn’s chief medical officer. “These efficacy results, combined with the continued strong tolerability profile across patient populations support our continued clinical development of AP01 in adults with pulmonary fibrosis.”

Avalyn expects to announce next steps in the clinical development of AP01 in the coming months.

ERS Presentation Details:

Poster Title: Nebulised Pirfenidone in Non-Idiopathic Pulmonary Fibrosis (IPF) Progressive Pulmonary Fibrosis (PPF): First Look at FVC Data
Location: PA405 in session 58
Day/Time: September 10, 8:00 – 9:30 a.m.

Presentation Title: Nebulised Pirfenidone in Idiopathic Pulmonary Fibrosis (IPF): First Look at FVC Data
Location: OA2581 in session 267
Day/Time: September 11, 11:00 a.m. – 12:15 p.m.

Poster Title: Inhaled Pirfenidone (AP01) Forced Vital Capacity (FVC) Data from the ATLAS Study vs Placebo from 3 Historical Idiopathic Pulmonary Fibrosis (IPF) Randomised Controlled Trials
Location: PA3485 in session 349
Day/Time: September 11, 4:00 – 5:30 p.m.

Copies of all presentations are available on the Avalyn website.

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 (AP01-005). An additional 59 AP01-naïve adults with either IPF or PPF were also recruited to participate. To date, 100 individuals have enrolled in the open-label extension, 8 of whom have been on study medication for over three years.

About Avalyn Pharma

Avalyn is a biopharmaceutical company developing targeted therapeutics for the treatment of rare respiratory diseases including pulmonary fibrosis and other interstitial lung diseases (ILD). Pulmonary fibrosis is characterized by scarring, 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, 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 recent clinical studies of AP01 assessed in 150 individuals with different forms of pulmonary fibrosis – idiopathic (IPF) and progressive pulmonary fibrosis (PPF) — AP01 demonstrated the potential to improve both efficacy and safety over existing therapy. More information can be found at