Novartis AG (NYSE:NVS) said Thursday that the biomarker cohort of its Phase 1/2 FORTITUDE study evaluating investigational therapy del-brax met both its primary and key secondary endpoints in patients with facioscapulohumeral muscular dystrophy (FSHD), a rare neuromuscular disease.
Trial Meets Endpoints
FSHD affects an estimated 45,000 to 87,000 people across the U.S. and Europe and is characterized by progressive muscle weakness, pain, fatigue, and disability.
The company reported reductions in KHDC1L (cDUX), a DUX4-regulated biomarker, and creatine kinase levels, a marker of muscle damage. According to Novartis, the findings indicate strong target engagement and a reduction in muscle injury among treated patients.
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