Avalyn Launches Global Phase 2b MIST Study on AP01 for Pulmonary Fibrosis

Avalyn Pharma Inc., a clinical-stage biopharmaceutical company centered on developing inhaled treatments for severe pulmonary diseases, has commenced the MIST Study, a Phase 2b clinical trial. This study will evaluate the safety and efficacy of AP01, an inhaled form of pirfenidone, in patients with progressive pulmonary fibrosis (PPF). AP01 is specifically formulated to target the lungs and has previously shown promising results in a Phase 1b study involving patients with idiopathic pulmonary fibrosis, achieving near-stabilization of lung function with fewer adverse events compared to oral pirfenidone.

Lyn Baranowski, CEO of Avalyn, expressed enthusiasm about the start of the Phase 2b study, highlighting it as a critical milestone in their mission to advance inhaled treatments for pulmonary fibrosis. Baranowski also acknowledged the support from the pulmonary fibrosis community, including healthcare practitioners, patients, and caregivers, who have been integral to the development of AP01.

The MIST Study is a randomized, double-blinded, placebo-controlled trial designed to assess the efficacy and safety of AP01 in PPF patients. It plans to enroll 300 participants and will compare two dose levels of AP01 to a placebo. The trial will be conducted at centers of excellence for interstitial lung disease (ILD) across North America, South America, Europe, and the Asia-Pacific region. The primary endpoint of the study is the change in forced vital capacity (FVC) from baseline at Week 52. Secondary endpoints will include quality of life assessments, time to disease progression, and changes in lung fibrosis scores using high-resolution computed tomography (HRCT). Additional exploratory outcomes related to lung function and respiratory hospitalization will also be examined.

Dr. Martin Kolb, a professor in the department of medicine at McMaster University and a primary investigator in the MIST Study, expressed his excitement about collaborating with Avalyn on the AP01 clinical program. He noted the promising potential of AP01 to transform the treatment landscape for pulmonary fibrosis, particularly for patients who struggle with the side effects of existing therapies. Kolb emphasized the urgent need for an effective and tolerable treatment option for pulmonary fibrosis patients, and the potential of the Phase 2b study of AP01 to meet this need.

Further information about the MIST Study (NCT06329401) is available on ClinicalTrials.gov. In the U.S., details can also be found at MISTPPFStudy.com.

Avalyn Pharma is dedicated to developing inhaled therapies for rare respiratory diseases such as pulmonary fibrosis and other interstitial lung diseases (ILD). Pulmonary fibrosis is marked by lung tissue scarring, a decline in lung function, reduced exercise capacity, and a lower quality of life, with increased mortality rates. Current treatments can slow the progression of pulmonary fibrosis but are often associated with significant toxicities that limit their use. Avalyn aims to create new inhaled formulations of approved medications that reduce systemic exposure and deliver the drug directly to the lungs. Their leading program, AP01, is an inhaled formulation of pirfenidone optimized for pulmonary fibrosis, currently under investigation in the MIST Phase 2b study. AP01 has shown improved efficacy and safety compared to existing therapies in completed and ongoing studies. Additionally, Avalyn has started a Phase 1b study for its second program, AP02, which involves inhaled nintedanib for idiopathic pulmonary fibrosis (IPF).

Avalyn continues to push forward with innovative inhaled therapeutic options, aiming to improve the lives of individuals suffering from life-threatening pulmonary conditions.