AP01 (Inhaled Pirfenidone) Treatment Demonstrated Substantially Improved Efficacy and Tolerability Out to 3.5 Years Compared to Historical Data with Oral Pirfenidone

Additional Poster Presentations Highlighted Post-hoc Analyses of the ATLAS Phase 1b Study that Support the Advancement of AP01 into a Phase 2b Trial

CAMBRIDGE, Mass., May 20, 2024 (GLOBE NEWSWIRE) -- Avalyn Pharma Inc. (Avalyn), a clinical-stage biopharmaceutical company focused on development of inhaled therapies for treatment of life-threatening pulmonary diseases, today presented clinical data for AP01, its novel formulation of inhaled pirfenidone, for the treatment of pulmonary fibrosis, supporting the Company’s planned Phase 2b trial in patients with progressive pulmonary fibrosis (PPF). Analysis from Avalyn’s ongoing ATLAS open-label extension study demonstrated that treatment with AP01 stabilized forced vital capacity (FVC) in patients with both idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF), with enhanced safety and tolerability compared to oral pirfenidone. Furthermore, findings from post-hoc analyses examining disease progression and Brainomix’s e-Lung biomarker scores support the Company’s selected dosing of 100 mg twice-daily and 50 mg twice-daily in the planned Phase 2b study.

The data were presented in multiple poster presentations and featured events during the 2024 American Thoracic Society (ATS) International Conference held May 17-22, 2024, in San Diego, CA.

“Having dedicated decades to treating patients with respiratory diseases, I’ve seen firsthand the pressing need for new treatments. There is a significant need for therapies that not only slow disease progression but are also tolerable for long-term use,” said Howard M. Lazarus, M.D., FCCP, Chief Medical Officer at Avalyn. “Pulmonary fibrosis is a devastating, and often fatal, disease despite currently available oral therapies. Our excitement stems from the long-term data with AP01 that shows treatment of patients out to nearly four years, surpassing the average survival rates for patients with progressive disease. We’ve generated a robust dataset with AP01 that shows substantially better efficacy and safety compared to today’s orally available therapies, which enables sustained treatment and ultimately, the potential for improved outcomes.”

Poster Title: Safety and Efficacy of Inhaled Pirfenidone (AP01) in the ATLAS Open-label Extension Study (Thematic Poster Session: B48, Poster #: 1242)

Avalyn’s open-label extension study enrolled a total of 100 patients across three cohorts: 41 patients with IPF who rolled over from the company’s ATLAS Phase 1b clinical trial and had been previously treated with AP01; 31 AP01-naïve patients with IPF; and 28 AP01-naïve patients with progressive pulmonary fibrosis (PPF). All patients received 100 mg AP01 twice-daily in the open-label extension. Data presented show that both IPF and PPF patients treated with AP01 experienced a clinically meaningfully lower annualized change in FVC than with oral pirfenidone as reported in prior pivotal studies.

Data from the cohort of roll-over IPF patients who were previously treated with AP01 were out to 180 weeks and showed an annualized change in FVC of -107 ml/year. These findings were substantially lower than the -235 ml/year previously reported with oral pirfenidone across three pivotal trials. Further, data from the cohorts of AP01- naïve IPF and PPF patients were out to 84 weeks and showed an annualized change in FVC of -134 ml/year and -13 ml/year, respectively.

A total of 47 patients across all three cohorts remain in the study and continue to receive AP01 treatment as of today, with some patients on therapy out to 252 weeks.

AP01 has continued to be generally well-tolerated, enabling long-term treatment. Patients treated with AP01 have reported markedly lower rates of nausea, rash, diarrhea, fatigue, dyspepsia and vomiting compared to the rate of side effects reported with oral pirfenidone.

Poster Title: Inhaled Pirfenidone Reduces the Risk of Disease Progression: A Post-hoc Analysis of the ATLAS Study (Thematic Poster Session: B48, Poster #: 1244)

Avalyn conducted a post-hoc analysis of disease progression in the ATLAS Phase 1b study in patients with IPF, which demonstrated that AP01 reduced the risk of disease progression, as defined by decline of greater than 10% FVC in patients with IPF. Fewer IPF patients in the AP01 100 mg twice-daily dosing arm (21%) experienced greater than 10% FVC decline by 48 weeks compared to the 50 mg once-daily dosing arm (35%). A similar, favorable trend of the AP01 100 mg twice-daily cohort was observed leading up to 48 weeks, at 12- and 24- weeks. This was consistent with the numerical advantages that were observed when comparing AP01 100 mg twice-daily to published results for placebo from pivotal studies. These data support Avalyn’s selection of AP01 100 mg twice-daily and 50 mg twice-daily for the Company’s planned Phase 2b clinical trial.

Poster Title: Differences in e-Lung Biomarker Scores Between Treatment Groups in Post-hoc Analysis of the ATLAS Inhaled Pirfenidone Solution (AP01) for IPF Clinical Trial (Thematic Poster Session: B48, Poster #: 1251)

Avalyn, together with Brainomix, presented promising data on e-Lung imaging biomarkers in a post-hoc analysis of the ATLAS Phase 1b study in patients with IPF. The imaging analysis utilized Brainomix’s proprietary weighted reticulovascular score (WRVS), an AI-enabled computed tomography (CT) processing software. Findings showed that WRVS strongly and substantially correlated with the risk of future IPF progression. AP01’s 100 mg twice-daily dosing arm was associated with stabilization of IPF with trends to a mean relative decrease in the WRVS and mean relative increase in FVC. These data suggest that e-Lung WRVS may be able to identify the risk of disease progression and identify treatment effects on CT, even when FVC is stable.

About Avalyn Pharma
Avalyn is a biopharmaceutical company developing inhaled therapies 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 that restrict their use and dosing. Avalyn is developing a pipeline of new inhaled formulations of approved medicines designed to reduce systemic exposure and deliver medication to the site of disease. Avalyn’s lead pipeline program, AP01, an optimized inhaled formulation of pirfenidone, which has been assessed in 150 individuals with different forms of pulmonary fibrosis and demonstrated clinical proof-of-concept with improved efficacy and safety over existing therapies. For more information, please visit avalynpharma.com, and follow us on social media: LinkedIn.

Investor Contact:
Monique Allaire
THRUST Strategic Communications
monique@thrustsc.com