Novartis AG (ADR) (NYSE:NVS) shares fell as much as 10% after the company's del-desiran failed to meet the primary endpoint in its pivotal Phase 3 HARBOR study evaluating the drug in myotonic dystrophy type 1.
The study did not show a statistically significant improvement over placebo on video hand opening time, a novel measure of hand myotonia that served as the trial's primary endpoint.
Novartis said it is evaluating the full HARBOR dataset and will engage with health authorities to determine the most appropriate development path for del-desiran.
The setback marks Novartis' second major late-stage trial failure in a matter of days, following the recent failure of its cardiovascular drug pelacarsen.
The read-through hit other companies developing treatments for myotonic dystrophy type 1. Dyne Therapeutics shares dropped roughly 21%, while Sarepta Therapeutics Inc (NASDAQ:SRPT) fell about 10%, as investors reassess whether targeting toxic DMPK RNA will reliably translate into functional improvement for patients.
