Insmed Receives FDA Breakthrough Therapy Designation for Non-Cystic Fibrosis Bronchiectasis Treatment

Insmed announced the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation for brensocatib (formerly known as INS1007) for the treatment of adult patients with non-cystic fibrosis bronchiectasis (NCFBE) for reducing exacerbations. Brensocatib is a novel oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) currently being developed by Insmed for the treatment of bronchiectasis and other inflammatory diseases.

The FDA's breakthrough therapy designation is designed to expedite the development and review of therapies that are intended to treat a serious or life-threatening disease and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy. The benefits of breakthrough therapy designation include more frequent communication and meetings with FDA, eligibility for rolling and priority review, intensive guidance on an efficient drug development program, and organizational commitment from the FDA involving senior managers.

"We are very pleased that the FDA has granted breakthrough therapy designation to brensocatib for treating patients with NCFBE, recognizing the strength of data from our Phase 2 WILLOW study and the potential for brensocatib to offer a novel, first-in-class treatment approach to bronchiectasis," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. "There are currently no approved therapies specifically targeting this severe and chronic pulmonary disease in the United States, Europe, or Japan. We look forward to continuing to work with the FDA as we advance the development of brensocatib to address this urgent medical need."

The breakthrough therapy designation for brensocatib is based on positive results from the global randomized, double-blind, placebo-controlled Phase 2 WILLOW study of brensocatib in adults with NCFBE. As previously announced, full results from this study will be presented during a virtual clinical trials session hosted by the American Thoracic Society (ATS) on Wednesday, June 24, 2020. Insmed will hold a conference call following the oral session during which the lead study investigator will further discuss the WILLOW data and Insmed management will provide a business assessment of brensocatib.

Insmed expects to initiate a Phase 3 program for brensocatib in bronchiectasis in the second half of 2020.