Insmed

Insmed's treprostinil palmitil inhalation powder (TPIP) met its primary endpoint with a statistically significant 35% placebo-adjusted reduction in pulmonary vascular resistance in patients with pulmonary arterial hypertension.

The Non-Cystic Fibrosis Bronchiectasis (NCFB) market is projected to grow from USD 1.7 billion in 2024 to USD 7.5 billion by 2035, representing a robust CAGR of 14.62%.

• The European Medicines Agency is reviewing accelerated assessment requests for three innovative drugs - apitegromab, brensocatib, and diazoxide choline - based on their potential major public health impact. • Scholar Rock's apitegromab, Insmed's brensocatib, and Soleno's diazoxide choline are being evaluated for their therapeutic innovation potential in addressing significant unmet medical needs. • The fast-track designation, if granted, would expedite the regulatory review process for these promising therapeutics, potentially bringing them to patients more quickly.

AN2 Therapeutics has selected Quality of Life-Bronchiectasis (QOL-B) respiratory domain as the new primary efficacy endpoint for their Phase 3 EBO-301 trial in treatment-refractory MAC lung disease.

The FDA has accepted Insmed's New Drug Application for brensocatib, setting a PDUFA target action date of August 12 under Priority Review designation.

Insmed's novel gene therapy candidate for Duchenne Muscular Dystrophy (DMD) has received IND clearance, paving the way for clinical trials.

The FDA has accepted Insmed's New Drug Application for brensocatib in bronchiectasis, granting Priority Review with a PDUFA target action date of August 12, 2025.

• The non-cystic fibrosis bronchiectasis (NCFB) market is experiencing growth due to rising prevalence and increased awareness, leading to early diagnosis and treatment. • Over 15 companies are actively developing more than 15 NCFB drugs, aiming to improve the treatment landscape for this chronic lung condition. • Key players like Insmed, AstraZeneca, and Verona Pharma are advancing promising therapies such as brensocatib, benralizumab and ensifentrine through clinical trials. • Recent clinical trial milestones, including Phase III results for brensocatib and Phase II enrollment for AP-PA02, signal progress in addressing unmet needs in NCFB treatment.

Insmed anticipates filing a New Drug Application (NDA) for brensocatib in Q4 2024, with a potential launch in mid-2025, targeting bronchiectasis treatment.

Subgroup analyses from the Phase 3 ASPEN trial demonstrate that brensocatib shows a consistent positive impact on pulmonary exacerbation rates across most pre-specified subgroups.