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.
Nazem Atassi, Global Head of Neuroscience and Gene Therapy Development at Novartis, said the results replicate the target engagement and downstream muscle protection observed in earlier dose-escalation cohorts. He added that the data support the dosing regimen selected for the ongoing Phase 3 trial.
Atassi said the company is reviewing the full set of biomarker and clinical findings and plans to engage with global regulators as development progresses.
Del-Brax Targets The Root Cause Of FSHD
Del-brax is an investigational antibody oligonucleotide conjugate (AOC), designed to address the underlying cause of FSHD by suppressing aberrant DUX4 gene expression.
The therapy combines monoclonal antibody targeting with oligonucleotide precision to deliver small interfering RNA into muscle cells.
Novartis said del-brax remains the only investigational therapy to demonstrate disease-modifying potential for FSHD in clinical studies.
The treatment has received Orphan Drug and Fast Track designations from the FDA, as well as an Orphan Drug designation from the European Medicines Agency, and is currently being evaluated in Phase 3 development.
A Key Addition To Novartis' Neuromuscular Pipeline
Del-brax joined the Novartis neuroscience portfolio through the company's acquisition of Avidity Biosciences, completed in February 2026 for $12 billion.
The acquisition also added delpacibart-etedesiran, known as del-desiran, currently in Phase 3 development for myotonic dystrophy type 1, and delpacibart-zotadirsen (del-zota), in Phase 2 development for Duchenne muscular dystrophy.
The ongoing Phase 3 FORTITUDE-3 trial is enrolling 200 FSHD patients aged 16 to 70 years and is evaluating the efficacy and safety of del-brax across multiple clinical and patient-reported outcome measures.NVS Price Action: Novartis shares were up 2.57% at $151.91 during premarket trading on Thursday, according to Benzinga Pro data.
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