Novartis misses primary endpoint in myotonic dystrophy study

16 hours ago

(AWP Alliance News) - Novartis AG - Basel, Switzerland-based pharmaceutical company - Announces results from its global phase three Harbor study evaluating delpacibart etedesiran, also known as del-desiran, for the treatment of patients with myotonic dystrophy type one. Currently there are no approved treatment options for the progressive neuromuscular disease. However, Novartis says del-desiran failed to demonstrate a statistically significant improvement versus placebo in relation to the primary endpoint of video hand opening time. Del-desiran is an investigational antibody oligonucleotide conjugate that has been designed to target the underlying cause of myotonic dystrophy type one.

"Despite decades of research, there are still no approved treatment options for DM1, and patients and caregivers continue to face a significant daily burden," says President, Development and Chief Medical Officer Shreeram Aradhye. "Developing therapies for a complex disease like DM1 remains challenging, and setbacks are part of scientific progress. As we continue to evaluate the full Harbor dataset, we remain committed to identifying the most appropriate development path for the del-desiran program and advancing innovative approaches for people living with DM1 and other serious neuromuscular diseases."

Current stock price: CHF125.46

12-month change: up 23%

By Elijah Dale, Alliance News senior reporter Asia-Pacific

Comments and questions to newsroom [at] alliancenews.com