Novartis shares tumbled after del-desiran (delpacibart etedesiran), the lead asset it gained by acquiring Avidity Biosciences for $12 billion in February 2026, failed its Phase III trial.

The HARBOR trial, in myotonic dystrophy type 1 (DM1) — an inherited disease that progressively weakens muscles — missed its primary endpoint: a statistically significant improvement over placebo in video Hand Opening Time (a measure of the muscle-relaxation problem central to DM1) through week 54.

Why the drug was interesting

Del-desiran is an antibody-oligonucleotide conjugate (AOC) — a muscle-targeting antibody (aimed at transferrin receptor 1) linked to a small interfering RNA designed to degrade the toxic DMPK messenger RNA that drives DM1. The AOC approach was the crown jewel of the Avidity deal, so the miss cuts deep.

Why it matters

Investors reacted sharply: Novartis fell ~11% in Zurich and ~14% on the NYSE — its worst single-day drop since March 2020. “Developing therapies for a complex disease like DM1 remains challenging, and setbacks are part of scientific progress,” said CMO Shreeram Aradhye. Novartis says it will review the full HARBOR dataset and talk to regulators about a path forward. For a company that paid a premium for a platform, the result is a reminder that even elegant biology has to clear the clinic. Business/clinical news, not investment advice.