An experimental drug for a rare neuromuscular condition failed to improve muscle function in a pivotal study, Novartis said Tuesday, the company’s second major trial collapse in a matter of days.
The two study flops will likely stoke concerns about Novartis’ pipeline and its acquisition strategy as it navigates a major patent cliff, though the company did see a victory with a multiple sclerosis drug last week. The stock was down by as much as 10% in early trading in Switzerland.
The Phase 3 HARBOR trial was testing the drug del-desiran in a condition called myotonic dystrophy type 1, known as DM1, which causes progressive muscle stiffness and weakness. Novartis said the drug did not meet its primary endpoint of outperforming placebo on improving video hand opening time, or vHOT, a measure of the hand’s ability to relax after squeezing.
