Novartis shares fall 14% after the Phase 3 HARBOR study for del-desiran fails to improve hand opening time in patients with DM1

Patients with the progressive muscle-stiffening illness did not open a clenched hand faster in a statistically meaningful way compared to a dummy treatment. This failure occurred shortly after the late-stage trial of pelacarsen, another Novartis asset, failed to reduce cardiovascular risk despite lowering blood fat levels. Additionally, the company paused several Phase 2 trials for the CAR T cell therapy rapcabtagene autoleucel following three patient deaths. Shreeram Aradhye, the president of development and chief medical officer at Novartis, stated that developing therapies for a complex disease like DM1 remains challenging and that setbacks are part of scientific progress. The company is currently reviewing the full data and will consult with regulators regarding next steps. Novartis continues to work on two other drugs from its $12 billion acquisition of Avidity Biosciences.