Novartis Drug Fails Late-Stage Trial for Muscle-Wasting Disease

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Novartis Drug Fails Late-Stage Trial for Muscle-Wasting Disease

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Novartis’s experimental drug, del-desiran (formerly AOC 1001), failed to meet its primary goal in a late-stage clinical trial for myotonic dystrophy type 1 (DM1), a rare and progressive muscle-wasting disease. The Phase 3 HARBOR study, which involved approximately 150 participants, did not demonstrate a statistically significant improvement in video hand opening time (vHOT), a measure of hand myotonia, compared to a placebo.

DM1 is a severe, inherited neuromuscular disorder with no approved therapies, affecting an estimated 80,000 people in the United States and Europe. The disease is caused by a genetic mutation leading to the accumulation of toxic messenger RNA (mRNA) in muscle cells, resulting in progressive muscle weakness, myotonia, and other systemic issues. Del-desiran was designed as an antibody oligonucleotide conjugate (AOC) to target and degrade this disease-causing DMPK mRNA.

Despite this setback, Novartis noted that evidence of clinical activity was observed in secondary endpoints and exploratory analyses, and the safety findings were consistent with previous data. The company plans to evaluate the full HARBOR dataset and consult with health authorities to determine the future development path for del-desiran.

This failure represents the third clinical trial disappointment for Novartis in a week. Earlier in September 2026, an experimental heart drug, pelacarsen, failed to reduce cardiovascular events in a late-stage study despite lowering lipoprotein(a) levels. Additionally, trials for an experimental cell therapy for autoimmune diseases were temporarily paused after three patient deaths. Del-desiran had previously received Orphan Drug, Fast Track, and Breakthrough Therapy designations from the U.S. Food and Drug Administration (FDA), highlighting the urgent need for treatments for DM1.

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