"First-in-class" oral therapy approved by FDA for breakthrough therapy

Insmed has announced that the U.SFDA has awarded brensocatib (formerly in1007) breakthrough therapy for the treatment of non-cystic fibrosis bronchodilatrification (NCFBE) adult patientsBrensocatib is a new oral, reversible dipeptide peptidease 1 (DPP1) inhibitor currently developed by Insmed to treat bronchial dilation and other inflammatory diseasesThe company recently launched a clinical trial to test the efficacy of this therapeutic treatment for patients with COVID-19non-cystic fibrosis bronchodilatal disease is a serious chronic lung disease that causes permanent expansion of the bronchial tube due to the circulation of infection, inflammation and damage to lung tissueSymptoms of the disease include chronic cough, sputum, shortness of breath, and repeated respiratory infections, which can worsen the underlying conditionCurrently, there is no approved treatment specifically for NCFBE patients in the United States, Europe or JapanBrensocatib is an oral, reversible DPP1 small molecule inhibitorDPP1 is an enzyme responsible for activating the neutrophil serine protease (NSPs) in neutrophilsNeutrophils are the most common white blood cell type and play an essential role in killing pathogens and medialinflammationIn chronic inflammatory lung disease, neutrophils accumulate in the airways and cause NSP to become hyperactive, causing damage to lung tissue and inflammationBrensocatib can reduce tissue damage to inflammatory diseases such as bronchial dilation by inhibiting DPP1 to reduce the activation of NSPsthis breakthrough therapy is based on positive results from THE Phase 2 clinical trial WILLOWCompared to placebo, brensocatib reached the primary endpoint of the studyThe 10 mg and 25 mg dose groups took significantly longer for the first lung deterioration during the 24-week treatmentIn addition, brensocatib treatment led to a lower frequency of lung deterioration compared to placeboSpecifically, the 10 mg group decreased by 36% (p-0.041) and the 25 mg dose group by 25% compared to the placebo group (p-0.167)FDA's Breakthrough Therapy identified as designed to accelerate the development and evaluation of treatments for serious or life-threatening diseasesThese preliminary clinical evidence in the study suggests that they may provide significant improvements compared to existing therapiesBreakthrough Therapy identified the ability for more frequent communication and meetings with the FDA, the ability to roll-in listing applications and access to priority reviews, and close guidance on improving the efficiency of development projects"We are pleased that the FDA has awarded brensocatib the breakthrough therapy recognition for patients with NCFBE," said DrMartina Flammer, Chief Medical Officer ofInsmedThis indicates that the FDA recognizes the potential of brensocatib as a 'first-in-class' therapyThere are currently no approved treatments specifically for this severe chronic lung disease in the United States, Europe or JapanWe look forward to continuing to work with the FDA to advance the development of brensocatib to address this urgent medical need"