Clinical TrialJun 11, 2026, 08:02 AM
Novartis del-brax Phase I/II FSHD study meets primary biomarker endpoint
AI Summary
Novartis announced positive results from the biomarker cohort of its FORTITUDE Phase I/II study for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD). The study met its primary and key secondary endpoints, demonstrating reductions in KHDC1L and creatine kinase levels, indicating strong target engagement and reduced muscle damage. Del-brax, an investigational antibody oligonucleotide conjugate (AOC), shows potential to be the first disease-modifying treatment for FSHD, a rare, progressive neuromuscular disease. Novartis plans to discuss these results with global regulatory authorities, while its Phase III study for del-brax is actively enrolling patients.
Key Highlights
- Novartis' delpacibart braxlosiran (del-brax) Phase I/II FORTITUDE study met primary and key secondary endpoints.
- The study showed reductions in KHDC1L (cDUX) and creatine kinase biomarker levels.
- Results indicate strong target engagement and reduction in muscle damage in FSHD patients.
- Del-brax is an investigational antibody oligonucleotide conjugate (AOC).
- It has the potential to be the first disease-modifying treatment for facioscapulohumeral muscular dystrophy (FSHD).
- Novartis plans to engage global regulatory authorities on the Phase I/II data.
- A Phase III study for del-brax is currently enrolling 200 patients aged 16-70 years.
- FSHD affects approximately 45,000-87,000 people in the US and EU.
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