Insmed partners patient group on bronchiectasis study

Insmed is joining forces with an organisation representing people living with the chronic lung disorder bronchiectasis to carry out a large-scale, real-world study of its recently approved therapy Brinsupri in the US.

The Bronchiectasis and NTM Association will work with the biopharma company on the observational study, called BEST-BE, which will measure how often adults with non-cystic fibrosis bronchiectasis (NCFB) experience exacerbations – a sudden worsening of symptoms – over two years.

NFCB is a highly debilitating condition in which the walls of the airways become thickened and damaged as a result of a cycle of inflammation and infections, leading to repeated exacerbations or attacks that require antibiotic treatment and can force patients to be hospitalised. Between 340,000 and 522,000 adults are receiving treatment for NFCB in the US.

DPP1 inhibitor Brinsupri (brensocatib), which is taken as a once-daily oral tablet, became the first therapy approved by the FDA for NFCB last year and can be used in patients aged 12 and over. The drug – which has been tipped as a future blockbuster – is thought to work by dampening down inflammation associated with incorrect functioning of neutrophils, a type of white blood cell.

BEST-BE receive funding from Insmed and will add to the data behind Brinsupri generated in the pivotal ASPEN trial, which showed that the drug cut the number of attacks experienced in a year by around 20% compared to placebo, whilst also prolonging the time to a first exacerbation and increasing the chances of patients becoming exacerbation-free over 52 weeks of follow-up.

Along with tracking exacerbation rates across a broad patient population, including those with concurrent nontuberculous mycobacterial (NTM) lung infections and immunocompromised individuals, BEST-BE will also assess health-related quality of life and the incidence and frequency of adverse events whilst taking Brinsupri.

It will feature a battery of innovative technologies, such as CRISPR-based detection of Mycobacterium avium complex (MAC) – a group of mycobacterial species that can cause progressive, destructive illness in vulnerable individuals – as well as AI-assisted CT scan analysis, and sputum rheology assessments.

Best-BE will enrol around 1,000 NCFB patients across 13 US states, and help to establish a biobank to support future research into the lung disorder.

The study "will follow diverse patient populations to see the long-term efficacy and safety profile of brensocatib, and how it affects disease progression in a real-world setting," said Charley Daley, chief research officer of the Bronchiectasis and NTM Association.

"This additional evidence could further shape clinical practice and help clinicians make informed treatment decisions for their patients with bronchiectasis."