—Largest Global Clinical Trial Ever Conducted in Bronchiectasis Demonstrates Statistically Significant and Clinically Meaningful Reduction in Frequency of Pulmonary Exacerbations Versus Placebo—
—Brensocatib 25 mg is the First Investigational Therapy for Bronchiectasis to Show a Statistically Significant Reduction in the Rate of Lung Function Decline—
—Brensocatib is Currently Under Priority Review with the U.S. Food and Drug Administration—
BRIDGEWATER, N.J., April 23, 2025 /PRNewswire/ — Insmed Incorporated (Nasdaq: INSM), a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases, today announced that positive results from the pivotal phase 3 ASPEN study of brensocatib in patients with non-cystic fibrosis bronchiectasis were published in the New England Journal of Medicine (NEJM). The landmark ASPEN study is the largest clinical trial ever conducted in bronchiectasis, a serious, chronic, and progressive inflammatory pulmonary disease that today has no approved therapies.
“Bronchiectasis is a debilitating disease characterized by pulmonary exacerbations, which contribute to lung function decline and severely impact quality of life,” said lead author James Chalmers, MBChB, PhD, Professor and Consultant Respiratory Physician at the School of Medicine, University of Dundee, UK. “With limited treatment options and no approved therapies, the burden of exacerbations remains high, with many patients experiencing multiple episodes each year. For the first time, the ASPEN data published in NEJM demonstrates that a treatment which targets inflammation can reduce exacerbations and slow the rate of lung function decline. This is an exciting development and represents a potentially transformative breakthrough for people living with bronchiectasis, offering new hope for patients with this challenging condition if brensocatib is approved.”
As previously reported, the ASPEN study met its primary endpoint, with both brensocatib doses achieving statistical and clinical significance for the reduction in the annualized rate of pulmonary exacerbations versus placebo over the 52-week treatment period. The annualized rate of exacerbations was 1.02 for brensocatib 10 mg, 1.04 for brensocatib 25 mg, and 1.29 for placebo. These rates were significantly lower in the brensocatib 10 mg and 25 mg groups versus placebo with rate ratios of 0.79 (adjusted P=0.004) and 0.81 (adjusted P=0.005), respectively.
Both dosage strengths of brensocatib also met several exacerbation-related secondary endpoints, including significantly prolonging the time to first exacerbation and significantly increasing the proportion of patients remaining exacerbation-free over the treatment period. Patients treated with brensocatib 25 mg also showed significantly lower lung function decline at week 52 as measured by post-bronchodilator forced expiratory volume over one second (FEV1).
Brensocatib was well-tolerated in the study. Treatment-emergent adverse events (TEAEs) occurring in at least 5.0% of patients treated with either dose of brensocatib and more frequently than in placebo were COVID-19 (15.8%, 20.9%, 15.8%), nasopharyngitis (7.7%, 6.3%, 7.6%), cough (7.0%, 6.1%, 6.4%), and headache (6.7%, 8.5%, and 6.9%) for brensocatib 10 mg, brensocatib 25 mg, and placebo, respectively.
“Currently, people with bronchiectasis have no approved treatments to address the frequent, damaging exacerbations that are the hallmark of this disease,” said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. “Brensocatib has the potential to be the first approved therapy to fill this critical unmet need in the care of patients with bronchiectasis, as well as the first approved dipeptidyl peptidase 1 (DPP1) inhibitor—a new mechanism of action with the potential to address a range of neutrophil-mediated inflammatory diseases. The ASPEN trial represents a transformative step forward for the millions of people globally diagnosed with bronchiectasis.”
Brensocatib is currently under Priority Review with the U.S. Food and Drug Administration, with a target action date of August 12, 2025, under the Prescription Drug User Fee Act (PDUFA).
About ASPEN
The total number of active sites in ASPEN was 391 sites in 35 countries. Adult patients (ages 18 to 85 years) were randomized 1:1:1 and adolescent patients (ages 12 to