Novartis AG (NOVN.SW) announced on Thursday that the biomarker cohort of the Phase 1/2 FORTITUDE trial for Del-brax met the primary and secondary endpoints in treating Facioscapulohumeral Muscular Dystrophy, or FSHD.
Company Profile
Novartis is a commercial-stage pharmaceutical company specialized in developing and commercializing therapies in areas such as cardiovascular disease, oncology, immunology, neuroscience, and radioligand therapy.
Facioscapulohumeral Muscular Dystrophy
It is a form of neuromuscular disease caused by the abnormal expression of the DUX4 gene, which is characterized by progressive muscle weakness, pain, fatigue, and disability. There are currently no approved therapies for this disease condition. The estimated number of people affected by the disease in the U.S. and EU is between 45,000 and 87,000.
Del-brax
Del-brax, or Delpacibart Braxlosiran, is an investigational antibody oligonucleotide conjugate, or AOC, in a new class of RNA therapeutics. It delivers siRNA to suppress DUX4 gene expression in patients with FSHD. Previously, it has received orphan drug designation by the FDA and EMA, with fast-track designation granted by the FDA for its treatment of FSHD.
Phase 1/2 FORTITUDE
It is a randomized, double-blind, placebo-controlled trial evaluating safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of del-brax in patients with FSHD.
The trial has three dose cohorts, with cohorts A & B evaluating del-brax administered at 2 mg/kg or 4 mg/kg doses to assess its safety and dose regimen.
Accordingly, the 2 mg/kg dose was selected for biomarker cohort C and was administered every 6 weeks in 51 FSHD patients over 12 months. The study's primary endpoint assessed the change in KHDC1L, which is a DUX4 biomarker, in patients' plasma. The secondary endpoint assessed change from baseline in creatine kinase levels.
Key Highlights
According to the company, the Phase 1/2 FORTITUDE trial met its primary and secondary endpoints of reduction in KHDC1L and creatine kinase levels. Del-brax also demonstrated a consistent safety profile, indicating target engagement and reduction in muscle damage in patients with FSHD.
Del-brax is currently enrolling 200 patients with FSHD for the Phase 3 FORTITUDE-3 trial. The primary endpoint will assess quantitative muscle testing or QMT in the U.S. and the 10-meter walk/run test (10MWRT) in Europe.
NOVN.SW is currently trading on the Swiss Stock Exchange at CHF 121.50, up 2.07%.
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