The most advanced experimental drug for the most common form of adult muscular dystrophy has failed to beat placebo on its main measure, leaving thousands of American families in the same position they were in a week ago, with no approved treatment for the disease itself.
Novartis announced on Sept. 8 that its global Phase 3 HARBOR study of del-desiran in myotonic dystrophy type 1 did not show a statistically significant improvement over placebo on video hand opening time, a measure of how quickly a person can release a grip. The company said it saw clinical activity in secondary endpoints and exploratory analyses, and will review the full dataset and talk to regulators about a path forward.
