Phase 3 ASPEN Trial Testing Brensocatib Now Recruiting; First Patient Dosed

Phase 3 ASPEN Trial Testing Brensocatib Now Recruiting; First Patient Dosed

The first participant has been dosed in ASPEN, a Phase 3 trial — now recruiting patients — that is evaluating the investigational medication brensocatib in people with non-cystic fibrosis (CF) bronchiectasis.

The trial (NCT04594369), sponsored by brensocatib’s developer Insmed, is currently recruiting participants in California, and is expected to enroll 1,620 individuals at 480 sites in 40 countries, according to the company. More information on trial sites will be available here.

In bronchiectasis, neutrophils, a type of immune cell, become activated and cause damage to the lungs. Brensocatib acts by blocking the protein dipeptidyl peptidase 1 (DPP1), which prevents this neutrophil activation.

As a potential brochiectasis treatment, brensocatib has received breakthrough therapy designation from the U.S. Food and Drug Administration and priority medicines (PRIME) designation from the European Medicines Agency. The investigational oral medication was originally developed by AstraZeneca before being licensed to Insmed in 2016.

In a previous Phase 2 trial, called WILLOW (NCT03218917), brensocatib reduced the risk and rate of exacerbations in adults with non-CF bronchiectasis, irrespective of age or clinical history.

Insmed announced the design for the ASPEN trial earlier this year. The study’s main goal is to determine the effect of brensocatib treatment on the rate of exacerbations, or times when symptoms suddenly get worse.

“There is an urgent need for an approved therapy for the treatment of bronchiectasis that has the potential to reduce exacerbations — the major driver of morbidity and mortality in these patients,” Martina Flammer, MD, chief medical officer of Insmed, said in a press release.

“Building on the strength of data from our Phase 2 WILLOW study, which showed that treatment with brensocatib both prolonged the time to first exacerbation and reduced the risk of exacerbation over the 24-week treatment period, we are very pleased to initiate the registrational ASPEN trial,” Flammer said.

Participants in ASPEN will be evenly divided among three groups, with all patients dosed daily for 52 weeks (one year). One group will be given a placebo, while the other two will get brensocatib — one group at a low dose of 10 mg and the other at a high dose of 25 mg.

In addition to assessing the effect of treatment on exacerbation rate, ASPEN will evaluate other related measures, such as the time to the first exacerbation. The trial also will analyze changes in quality of life — assessed with the Quality of Life Questionnaire – Bronchiectasis — and in lung function, determined by forced expiratory volume (FEV). FEV measures how much air a person can exhale during a forced breath. Adverse events also will be monitored.

“We believe this [ASPEN trial] is a significant step toward enabling regulatory submissions and potentially bringing brensocatib to patients in need,” Flammer said.