Overview
- The company reported that a recent del‑brax study met its predefined endpoints and reproduced prior signs of target engagement and muscle‑protection that informed the selected dosing for FORTITUDE‑3.
- Del‑brax is an investigational antibody‑oligonucleotide conjugate that delivers small interfering RNA to muscle cells to suppress aberrant DUX4 gene expression, the presumed driver of FSHD.
- The program holds FDA Orphan Drug and Fast Track designations and an EMA Orphan Drug designation, positioning Novartis to seek accelerated regulatory interactions based on the new data.
- Del‑brax entered Novartis’ pipeline through the February 2026 acquisition of Avidity Biosciences for $12 billion, adding several neuromuscular assets now in mid‑ to late‑stage development.
- FSHD affects tens of thousands in the U.S. and Europe and causes progressive muscle weakness, so the company’s next steps — full data review, regulator meetings, and completion of the ~200‑patient Phase 3 trial — will determine whether del‑brax can become the first disease‑modifying treatment.