CAMBRIDGE, Mass., Sept. 03, 2024 (GLOBE NEWSWIRE) -- Avalyn Pharma Inc., a clinical-stage biopharmaceutical company focused on development of inhaled therapies for treatment of life-threatening pulmonary diseases, today announced the initiation of the MIST Study, a Phase 2b clinical trial evaluating the safety and efficacy of AP01 (inhaled pirfenidone) in patients with progressive pulmonary fibrosis (PPF).

AP01 is a novel formulation of inhaled pirfenidone optimized to target the lungs. In the completed Phase 1b ATLAS Study of patients with idiopathic pulmonary fibrosis, AP01 achieved near-stabilization of lung function in a dose-responsive manner as assessed by forced vital capacity (FVC) and was well tolerated with fewer adverse events compared to historical data with oral pirfenidone.

“We are thrilled to start this Phase 2b study of AP01, which marks another significant milestone for Avalyn and our commitment to advancing inhaled treatments for patients with pulmonary fibrosis,” said Lyn Baranowski, CEO of Avalyn. “We are encouraged by AP01’s robust clinical data generated to date, which demonstrated strong tolerability and efficacy with inhaled delivery of pirfenidone. We would like to extend our sincere gratitude to the pulmonary fibrosis community of health care practitioners, patients, and their caregivers for their ongoing support of our efforts to develop an innovative therapeutic option for people living this life-threatening disease.”

The randomized, double-blinded, placebo-controlled MIST Study will investigate the efficacy and safety of AP01 in patients with PPF. The study is expected to enroll 300 patients with PPF and will evaluate two dose levels of AP01 compared to placebo. The study will be conducted at interstitial lung disease (ILD) centers of excellence in North America, South America, Europe and Asia-Pacific. The primary endpoint of the study is change from baseline in FVC at Week 52. Secondary endpoints include quality of life measurements, time to disease progression, and change in lung fibrosis score based on high-resolution computed tomography (HRCT). Additional exploratory outcomes, including those related to lung function and respiratory hospitalization, will also be evaluated.

“My colleagues and I are excited to collaborate with Avalyn on the next phase of the AP01 clinical program as the results from earlier studies showed its promising potential to change the treatment paradigm for pulmonary fibrosis,” said Martin Kolb, M.D., Ph.D., professor, department of medicine at McMaster University, and a primary investigator in the MIST Study. “We are driven by a need to bring forward an effective and tolerable treatment for people suffering with pulmonary fibrosis, many of whom have difficulty tolerating and even need to discontinue existing therapies due to side effects, despite the fatal nature of this disease. This Phase 2b study of AP01 has the potential to bring us closer to providing the option of an inhaled therapeutic that could change patients’ lives.”

More information about the MIST Study (NCT06329401) can be found by visiting ClinicalTrials.gov or emailing MISTPPFStudy@avalynpharma.com. In the U.S., information can also be found at MISTPPFStudy.com.

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 of lung tissue, 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 directly to the site of disease. Avalyn’s lead program, AP01, is an optimized inhaled formulation of pirfenidone, currently being studied in the ongoing MIST Phase 2b study in progressive pulmonary fibrosis (PPF). AP01has been assessed in over 150 individuals with different forms of pulmonary fibrosis and demonstrated clinical proof-of-concept with improved efficacy and safety compared to historical data with existing therapies in the completed Phase 1b study and its ongoing open-label extension study. Avalyn has initiated a Phase 1b study for its second program, AP02, inhaled nintedanib, that is being developed for the treatment of idiopathic pulmonary fibrosis (IPF). For more information, please visit avalynpharma.com and follow us on LinkedIn.

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