Novartis neuromuscular drug fails pivotal trial, escalating pipeline pressures

An experimental treatment for a rare neuromuscular disorder did not improve muscle function in a Phase 3 study, Novartis announced Tuesday, marking the company’s second significant trial failure within days.

The dual setbacks are likely to intensify scrutiny of Novartis’ pipeline and acquisition strategy as the company confronts a major patent cliff, even after a recent victory with a multiple sclerosis drug. Shares fell up to 10% in early Swiss trading.

The Phase 3 HARBOR trial tested del-desiran in myotonic dystrophy type 1 (DM1), a disorder causing progressive muscle stiffness and weakness. Novartis stated the drug missed its primary endpoint of beating placebo on video hand opening time (vHOT), which measures the hand’s ability to relax after gripping.

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