Insmed Incorporated issued the following announcement on June 8.
Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today announced that 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.
Post-ATS Conference Call Details
Insmed will host a conference call on Wednesday, June 24, 2020 at 4:00 pm ET. Shareholders and other interested parties may participate in the conference call by dialing (888) 317-6003 (domestic) or (412) 317-6061 (international) and referencing conference ID number 9628054. The call will also be webcast live on the company's website at www.insmed.com.
A replay of the conference call will be accessible approximately one hour after its completion through July 8, 2020, by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and referencing replay access code 10144366. A webcast of the call will also be archived for 90 days under the Investor Relations section of the Company's website at www.insmed.com.
About WILLOW
WILLOW was a randomized, double-blind, placebo-controlled, parallel-group, multi-center, multi-national, Phase 2 study to assess the efficacy, safety and tolerability, and pharmacokinetics of brensocatib administered once daily for 24 weeks in patients with non-cystic fibrosis bronchiectasis (NCFBE). WILLOW was conducted at 116 sites and enrolled 256 adult patients diagnosed with NCFBE who had at least two documented pulmonary exacerbations in the 12 months prior to screening. Patients were randomized 1:1:1 to receive either 10 mg or 25 mg of brensocatib or matching placebo. The primary efficacy endpoint was the time to first pulmonary exacerbation over the 24-week treatment period in the brensocatib arms compared to the placebo arm.
About Brensocatib (Formerly INS1007)
Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase I (DPP1) being developed by Insmed for the treatment of patients with bronchiectasis. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase, in neutrophils when they are formed in the bone marrow. Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. Brensocatib may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs.
About Non-Cystic Fibrosis Bronchiectasis
Non-cystic fibrosis bronchiectasis (NCFBE) is a severe, chronic pulmonary disorder in which the bronchi become permanently dilated due to a cycle of infection, inflammation, and lung tissue damage. The condition is marked by frequent pulmonary exacerbations requiring antibiotic therapy and/or hospitalizations. Symptoms include chronic cough, excessive sputum production, shortness of breath, and repeated respiratory infections, which can worsen the underlying condition. NCFBE affects approximately 340,000 to 520,000 patients in the U.S. Today, there are no approved therapies specifically targeting NCFBE in the U.S., Europe, or Japan for the treatment of patients with NCFBE.
About Insmed
Insmed Incorporated is a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases. Insmed's first commercial product, ARIKAYCE® (amikacin liposome inhalation suspension), is the first and only therapy approved in the United States for the treatment of refractory Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. MAC lung disease is a chronic, debilitating condition that can cause severe and permanent lung damage. Insmed's earlier-stage clinical pipeline includes brensocatib, a novel oral reversible inhibitor of dipeptidyl peptidase 1 with therapeutic potential in non-cystic fibrosis bronchiectasis and other inflammatory diseases, and treprostinil palmitil, an inhaled formulation of a treprostinil prodrug that may offer a differentiated product profile for rare pulmonary disorders, including pulmonary arterial hypertension. For more information, visit www.insmed.com.
Original source can be found here.
