The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to brensocatib (INS1007; Insmed) for the treatment of adults with non-cystic fibrosis bronchiectasis (NCFBE) for reducing exacerbations.

Brensocatib is a novel oral, small molecule, reversible inhibitor of dipeptidyl peptidase 1 (DPP1), which is an enzyme responsible for the activation of neutrophil serine proteases (NSPs). The Company believes that the inhibition of DPP1 could potentially decrease the damaging effects of inflammatory lung diseases, such as NCFBE.  

The designation is based on positive data from the multicenter, double-blind, placebo-controlled phase 2 WILLOW study that evaluated the efficacy, safety and tolerability of brensocatib in 256 adults with NCFBE who had at least 2 documented pulmonary exacerbations in the previous 12 months. Patients were randomized to receive brensocatib 10mg, 25mg, or placebo once daily for 24 weeks. The primary end point was the time to first pulmonary exacerbation, defined as signs and symptoms plus intervention with systemic antibiotics.

Full study results will be presented at a virtual American Thoracic Society (ATS) session on June 24, 2020. The Company expects to initiate a phase 3 program for brensocatib in bronchiectasis in the second half of 2020.

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“There are currently no approved therapies specifically targeting this severe and chronic pulmonary disease in the United States, Europe, or Japan,” said Martina Flammer, MD, MBA, Chief Medical Officer of Insmed. :We look forward to continuing to work with the FDA as we advance the development of brensocatib to address this urgent medical need.”

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