STAT+: Neuromuscular drug from Novartis fails in key study, adding to pressure on company — SkimNews
Get the Health newsletter
Daily health & science — research, biotech, public health, the studies worth knowing. Free.
- Novartis said its experimental drug del-desiran failed to meet the primary endpoint in the Phase 3 HARBOR trial, not outperforming placebo on video hand opening time (vHOT), a measure of the hand's ability to relax after squeezing.
- Del-desiran was being tested for myotonic dystrophy type 1 (DM1), a rare neuromuscular condition that causes progressive muscle stiffness and weakness.
- The del-desiran failure is Novartis' second major trial collapse in a matter of days, intensifying scrutiny of the company's pipeline and acquisition strategy.
- The trial setbacks come as Novartis navigates a major patent cliff, though the company did secure a victory with a multiple sclerosis drug last week.
Why it matters: Two late-stage trial collapses in quick succession put Novartis' late-stage pipeline under a microscope just as the company faces a major patent cliff on existing blockbusters. DM1 patients lose a candidate aimed at progressive muscle stiffness, and investors will press management on whether acquisition strategy is replenishing late-stage assets fast enough.
Ask SkimNews
