- Insmed's Phase 3 ASPEN study of brensocatib shows significant reduction in annualized pulmonary exacerbation rates.
- Brensocatib (INSM, Financial) demonstrated statistically significant efficacy with a 25mg dose reducing lung function decline.
- FDA's Priority Review is underway, with an action date set for August 12, 2025.
Insmed Incorporated (INSM) has announced the publication of positive results from its pivotal Phase 3 ASPEN study of brensocatib in the New England Journal of Medicine. The study, which is the largest clinical trial ever conducted for bronchiectasis, demonstrated significant efficacy in treating non-cystic fibrosis bronchiectasis, a chronic inflammatory pulmonary condition currently without approved therapies.
The trial's primary endpoint was met with both 10mg and 25mg doses showing reduced annualized exacerbation rates of 1.02 and 1.04, respectively, compared to 1.29 for the placebo. Notably, the 25mg dose achieved a significant reduction in the rate of lung function decline, marking a potential breakthrough in treatment. The drug's safety profile was favorable, with common side effects including COVID-19, nasopharyngitis, cough, and headache.
Brensocatib, a DPP1 inhibitor, is currently under Priority Review by the FDA, with a PDUFA target action date of August 12, 2025. If approved, it would be the first approved therapy for bronchiectasis and the first DPP1 inhibitor, offering a novel mechanism of action for addressing neutrophil-mediated inflammation.
Approximately 500,000 patients in the U.S., 600,000 in the EU5, and 150,000 in Japan suffer from bronchiectasis, highlighting an area of unmet medical need. The ASPEN study included 1,680 adult patients and 41 adolescent patients, assessing the drug's efficacy over a 52-week period.
