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Novartis AG (NYSE:NVS) has suffered a major setback after its experimental drug del-desiran failed a late-stage trial for myotonic dystrophy type 1, a rare muscle-wasting disease with no approved treatments. The drug failed to show a statistically significant improvement over placebo on the trial's primary endpoint, video hand opening time. Del-desiran was one of the key assets acquired through Novartis' $12 billion acquisition of Avidity Biosciences, making the failure particularly damaging to investor confidence in the company's acquisition-led pipeline strategy.
The setback came just days after Novartis AG (NYSE:NVS)'s cholesterol drug pelacarsen also failed a major late-stage study. The two failures have increased pressure on CEO Vas Narasimhan as Novartis seeks to replace revenue from established medicines such as Entresto ahead of future patent expirations. The company nevertheless reaffirmed its target of 5%-6% annual sales growth through 2030.
Novartis AG (NYSE:NVS) still has the scale, cash generation, and diversified commercial portfolio to absorb setbacks in individual drug programs. Although del-desiran failed its late-stage trial in myotonic dystrophy, the failure does not necessarily invalidate Novartis' broader pipeline or the other therapies acquired through Avidity. The company is evaluating the full dataset and plans to engage with regulators, leaving some possibility that the program could have a future depending on the underlying data and trial interpretation.
