Insmed Announces Positive Topline Results from Landmark ASPEN Study of Brensocatib in Patients with Bronchiectasis

—Phase 3 Study Achieves Primary Endpoint for Both Dosage Strengths of Brensocatib with Statistically Significant and Clinically Meaningful Reduction in Frequency of Pulmonary Exacerbations Versus Placebo—

—Treatment with Brensocatib Also Achieves Statistical Significance on Multiple Secondary Endpoints for Both Dosage Strengths Versus Placebo—

—Brensocatib Well-Tolerated at Both Dosage Strengths—

—Results from ASPEN Validate DPP1 Inhibition as New Mechanism of Action with Potential to Address Range of Neutrophil-Mediated Diseases—

—Insmed Plans to Advance Quickly Toward U.S. Regulatory Filing, with Anticipated U.S. Launch in Mid-2025, Pending Approval—

—Insmed to Host Investor Call at 8:00 am ET on Tuesday, May 28, 2024—

BRIDGEWATER, N.J., May 28, 2024 /PRNewswire/ -- Insmed Incorporated (Nasdaq: INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today announced positive topline results from the ASPEN study, a global, randomized, double-blind, placebo-controlled Phase 3 study to assess the efficacy, safety, and tolerability of brensocatib in patients with non-cystic fibrosis bronchiectasis. The study met its primary endpoint, with both dosage strengths of brensocatib demonstrating statistically significant reductions in the annualized rate of pulmonary exacerbations (PEs) versus placebo. The study also met several of its prespecified secondary endpoints with statistical significance.

Based on these results, Insmed plans to file a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for brensocatib in patients with bronchiectasis in the fourth quarter of 2024. Pending regulatory approvals, Insmed anticipates a U.S. launch for brensocatib in mid-2025 followed by launches in Europe and Japan in the first half of 2026. If approved, brensocatib would be the first approved treatment for 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 diseases.

Topline efficacy results from the ASPEN study are as follows:

"I am thrilled that the ASPEN study has demonstrated a statistically significant and clinically meaningful treatment effect for brensocatib compared with placebo, underscoring the impact this investigational therapy may have on patients with bronchiectasis," said lead study investigator James Chalmers, MBChB, Ph.D., Professor and Consultant Respiratory Physician at the School of Medicine, University of Dundee, UK. "Today, there is no approved treatment for bronchiectasis and there remains an urgent need for a therapy that can reduce exacerbations. As a DPP1 inhibitor, brensocatib would be the first treatment in its class and could offer a completely new approach to managing this difficult-to-treat patient population, heralding a new era in clinical management of bronchiectasis."

As part of the ASPEN study's conduct, more than 460 trial sites were engaged in nearly 40 countries. After excluding sites that did not enroll any patients and all sites in Ukraine, 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