Novartis' $12B Avidity deal falters as DM1 therapy misses Phase 3 endpoint

Novartis' recent acquisition of Avidity Biosciences was intended to bring a novel therapy for Myotonic dystrophy type 1 (DM1) into its portfolio.

The drug, del-desiran, an antibody-oligonucleotide conjugate targeting the DMPK protein, completed a Phase 3 trial but failed to show a statistically significant improvement in hand opening time for patients.

The trial miss erased much of the $12 billion value that analysts had attached to the deal, prompting a sharp downgrade of the transaction’s outlook.

Avidity’s pipeline also includes candidates for Duchenne muscular dystrophy and facioscapulohumeral muscular dystrophy, but the DM1 setback highlights the risk of large bets on rare disease programs.

The episode underscores the broader industry call for more, not fewer, bold investments in difficult-to-treat conditions despite recent M&A losses.