Novartis has suffered another setback after its experimental drug del-desiran failed to meet the main goal of a late-stage clinical trial for myotonic dystrophy type 1.
The global Phase III HARBOR trial did not show a statistically significant improvement compared with a placebo in patients’ ability to open their hands. Novartis said it is reviewing the complete trial data and will discuss the next steps with health regulators.
The result comes shortly after the company reported disappointing results for pelacarsen, which failed to lower the risk of cardiovascular events in a late-stage study. The drugmaker also recently paused eight clinical trials of its experimental cell therapy, rap-cel, following the deaths of three participants.
Del-desiran was among three antibody-oligonucleotide therapies added to Novartis’ neuromuscular pipeline through its roughly $12 billion acquisition of Avidity Biosciences last year.
Despite the setbacks, Novartis said it continues to expect average annual sales growth of 5% to 6% through 2030. Analysts at Jefferies, however, questioned whether the target can be achieved without further acquisitions.
The company has also reported encouraging developments elsewhere in its pipeline. Last week, Novartis said its experimental multiple sclerosis treatment remibrutinib showed a meaningful delay in disability progression in a late-stage trial.