Novartis' $12 billion Avidity drug fails Phase 3 trial in myotonic dystrophy

Novartis reported that its antibody-oligonucleotide conjugate del-desiran did not meet the primary endpoint in the Phase 3 HARBOR study for myotonic dystrophy type 1, showing no significant improvement in hand opening time compared with placebo.

Although the primary goal was missed, the trial observed some signals of activity in secondary and exploratory endpoints, suggesting the drug may still have biological effects that warrant further investigation.

The setback follows a similar failure of pelacarsen in a cardiovascular outcome trial, and the news sent Novartis shares down about 12% in pre-market trading, raising concerns about the recent $12 billion acquisition of Avidity Biosciences.