Novartis announced that del-brax, an antisense oligonucleotide conjugate, achieved its primary endpoint in a Phase 1/2 study for a muscle-wasting disease. The data marks a key clinical win for the RNA therapy, which was acquired through Novartis' $12 billion deal with Avidity Biosciences.
The trial evaluated the drug's efficacy and safety in patients with the degenerative condition, though specific efficacy rates and patient population details were not disclosed in the release. The therapy is designed to target the underlying genetic cause of the disease.
As a Phase 1/2 study, this data supports advancing del-brax into later-stage trials. The regulatory pathway for such an RNA therapy typically requires further pivotal studies before FDA or EMA submission, and no PDUFA date has been set.
Novartis shares saw minimal movement following the announcement. The $12 billion Avidity acquisition positions Novartis prominently in the RNA therapeutics space, competing with other antisense and siRNA developers.
Analysts caution that early-stage data, while encouraging, may not predict late-stage success. The muscle-wasting disease field has seen previous setbacks, and the full dataset has yet to be published in a peer-reviewed journal.