Novartis' experimental myotonic dystrophy drug fails pivotal Phase 3 trial
Novartis announced that its Phase 3 HARBOR study of del-desiran did not meet its primary endpoint in patients with myotonic dystrophy type 1.
The Swiss pharmaceutical firm reported that del-desiran, an investigational neuromuscular therapy, failed to show a statistically significant improvement over placebo on the video hand opening time measure. The Phase 3 HARBOR trial targeted myotonic dystrophy type 1, a rare disease characterized by progressive muscle stiffness and weakness. This setback follows another recent trial disappointment for Novartis, raising questions about its drug pipeline and recent acquisition strategy, even as the company celebrated a separate multiple sclerosis drug success.
Why it matters
The miss signals challenges for Novartis' pipeline and could affect future treatments for a rare, debilitating muscle disease.
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