Novartis DM1 miss raises stakes for next wave of DMPK readouts

The Phase III miss for Novartis’ del-desiran leaves open whether the most advanced attempt to treat myotonic dystrophy type 1 by targeting DMPK had activity that was poorly captured by its primary endpoint or failed more broadly across measures of muscle function. Whether data from the company’s HARBOR study can reveal where the molecular-to-clinical chain broke is less clear.

Novartis AG (SIX:NOVN; NYSE:NVS) announced Tuesday that delpacibart etedesiran (del-desiran) did not significantly improve the primary endpoint of video hand opening time (vHOT) versus placebo in the 150-patient study, but the company said it saw evidence of activity in secondary endpoints and exploratory analyses, including muscle strength and daily living activities. Novartis has not disclosed numerical results or said when it will present the full dataset, and has not indicated whether HARBOR included biomarker analyses...