Insmed's Arikayce missed the primary endpoint in a Phase II clinical trial for the treatment of non-tuberculous mycobacterial (NTM) lung infections, but the company emphasized significant results for the trial's secondary endpoint to keep its stock from taking a very steep dive.
Insmed's Arikayce missed the primary endpoint in a Phase II clinical trial for the treatment of non-tuberculous mycobacterial (NTM) lung infections, but the company emphasized significant results for the trial's secondary endpoint to keep its stock from taking a very steep dive.
Monmouth Junction, New Jersey-based Insmed will discuss the data with US and EU regulators to determine the next steps for...
With two deaths in non-ambulatory DMD patients, Sarepta is attempting damage control and will ask the US FDA to advise, setting up a possible confrontation with CBER director Prasad.
Deal Snapshot: Including its internal efforts with bimagrumab, Lilly has been seeking a muscle-sparing therapy to complement GLP-1 obesity drugs, such as Zepbound.
Regenxbio reported functional and biomarker data from an ongoing study of RGX-202, which it hopes to file for approval in Duchenne muscular dystrophy in 2026.
With its exon 53-skipping candidate already showing promise in 24-week data, Wave now has 48-week data showing improvements in muscle health and functional outcomes.
The company said it intends to maintain a focus on rare neuromuscular disease with apitegromab but could consider partnering in obesity.
The US firm was already looking to compete in ulcerative colitis, but now expands its ambitions by moving into rheumatoid arthritis as well.
Wales-based group launches with $140m series A.
