On Tuesday, Novartis announced that its del-desiran drug, aimed at treating myotonic dystrophy type 1, did not show a statistically significant improvement in hand opening time compared to a placebo in its global phase III HARBOR study.
This failure is particularly concerning as it represents the third setback for the company in drug trials within a week, leading to a 10% decline in its stock price, which could result in the worst trading day in the company's history.
Novartis is currently reviewing the full dataset from the HARBOR study and plans to consult with health authorities to determine the future development path for del-desiran. The drug is part of Novartis's neuromuscular pipeline, which was bolstered by the acquisition of Avidity Biosciences for approximately $12 billion last year.
Shreeram Aradhye, Novartis's president of development and chief medical officer, acknowledged the challenges in developing therapies for complex diseases like myotonic dystrophy type 1, emphasizing the company's commitment to advancing innovative treatments despite these setbacks