Novartis's Phase III HARBOR study of del-desiran in myotonic dystrophy type 1 failed to show statistically significant improvement over placebo on its primary endpoint of video hand opening time. The company reaffirmed its 5-6 percent compound annual sales growth target for 2025-2030 and pointed to progress in its broader AOC pipeline.
Novartis's late-stage del-desiran study in myotonic dystrophy type 1 missed its primary endpoint, a setback for the Swiss drugmaker's neuromuscular pipeline. The company reaffirmed its target of 5-6 percent compound annual sales growth from 2025 to 2030.
"Developing therapies for a complex disease like DM1 remains challenging, and setbacks are part of scientific progress," Shreeram Aradhye, President of Development and Chief Medical Officer at Novartis, said. The company is evaluating the full dataset and will consult health authorities on the program's path forward.
The Phase III HARBOR study, which enrolled about 150 people with DM1 over 54 weeks, "did not demonstrate statistically significant improvement versus placebo on the primary endpoint of video hand opening time," a measure of hand myotonia, Novartis said in a statement. Evidence of clinical activity appeared in secondary endpoints and exploratory analyses, while safety findings were generally consistent with earlier data. Del-desiran, an antibody oligonucleotide conjugate (AOC) that targets the toxic DMPK messenger RNA behind the disease, had received Orphan Drug, Fast Track and Breakthrough Therapy designations from the US Food and Drug Administration.
The miss lands a second blow to the pipeline in as many days, after shares fell more than 3 percent on news that Novartis's cholesterol drug failed a closely watched study. Del-desiran was one of three AOC therapies added through the acquisition of Avidity Biosciences, and the company stressed the platform still has momentum: the FDA granted priority review to delpacibart zotadirsen (del-zota) in Duchenne muscular dystrophy, and Novartis plans to meet with the agency on delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy after positive Phase I/II biomarker data.
The trial failure removes a potential revenue contributor from a pipeline that carries no approved DM1 treatment, a market with high unmet need. Investors will watch whether Novartis can salvage del-desiran through a secondary-endpoint or subgroup analysis, and how the setback shapes the broader AOC platform's valuation ahead of the del-zota regulatory decision.
This article is for informational purposes only and does not constitute investment advice.
