STAT+: Neuromuscular drug from Novartis fails in key study, adding to pressure on company

Andrew covers the biopharma industry, scientific research, and public health across the continent. You can reach Andrew confidentially on Signal at drewqjoseph.71.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 raise questions 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 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. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe