Novartis AG - Form 6-K Filing on Del-brax Clinical Trial Results

Novartis AG announced positive results from the Phase I/II FORTITUDE study of del-brax, an investigational RNA therapeutic for facioscapulohumeral muscular dystrophy (FSHD). The study met its primary and key secondary endpoints, demonstrating reductions in KHDC1L and creatine kinase levels, indicating target engagement and reduced muscle damage. Del-brax shows potential as the first disease-modifying treatment for FSHD, a progressive neuromuscular disease. Novartis plans to engage with global regulatory authorities based on these findings and is currently enrolling patients in a Phase III study. Del-brax, an antibody oligonucleotide conjugate (AOC), targets the root cause of FSHD by suppressing DUX4 expression. This therapy has received Orphan Drug and Fast Track designations from the FDA and Orphan Drug designation from the EMA. The acquisition of Avidity Biosciences added del-brax and two other AOC therapies to Novartis's neuroscience pipeline, strengthening their focus on neuromuscular diseases.