Key Takeaways:
Novartis AG said Thursday its experimental drug delpacibart braxlosiran (del-brax) lowered two blood markers linked to facioscapulohumeral muscular dystrophy in a Phase 1/2 study, supporting the Swiss drugmaker's $12 billion bet on RNA-targeting therapies.
"We are now evaluating the totality of the biomarker and clinical data and look forward to discussions with global regulatory agencies," Nazem Atassi, Novartis' global head of neuroscience and gene therapy development, said in a statement.
The 90-patient trial tested two doses of del-brax against placebo across three cohorts. Patients receiving the drug showed reduced levels of KHDC1L, a biomarker regulated by the DUX4 gene that drives FSHD pathology, and creatine kinase, a marker of muscle damage. The safety profile was consistent with previous results, the company said.
The data "at least partially validates Novartis' decision to acquire Avidity," Jefferies analyst Michael Leuchten wrote in a note. Shares of Novartis rose about 3% on Thursday morning.
Del-brax is an antisense oligonucleotide conjugate designed to silence the DUX4 gene, which is expressed incorrectly in FSHD patients. The condition causes progressive muscle weakness in the face, shoulders, arms and other areas, affecting an estimated 45,000 to 87,000 people in the U.S. and European Union. No disease-modifying treatment currently exists.
Novartis acquired the drug through its $12 billion takeover of Avidity Biosciences in October 2025. The deal brought three AOC candidates, including del-desiran for myotonic dystrophy type 1 — with data expected in the second half of 2026 — and del-zota for Duchenne muscular dystrophy, now in mid-stage development.
The company plans to discuss the Phase 1/2 results with regulators globally. A late-stage study evaluating del-brax on muscle strength and a 10-meter walk-and-run test is already enrolling patients.
The positive readout strengthens Novartis' position in rare neuromuscular diseases and could open a path to accelerated approval. Investors will watch for del-desiran data later this year as the next catalyst for the AOC platform.
This article is for informational purposes only and does not constitute investment advice.
