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Novartis del-desiran fails to meet primary endpoint in phase 3 study for DM1 treatment

Novartis del-desiran fails to meet primary endpoint in phase 3 study for DM1 treatment

智通财经智通财经2026/09/08 05:51
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Novartis (NVS.US) announced that its Phase III HARBOR study evaluating del-desiran for the treatment of type 1 myotonic dystrophy (DM1) did not meet its primary endpoint. Data showed that, compared with placebo, patients did not show a statistically significant improvement in video hand opening time (vHOT). Novartis stated that the study observed signs of clinical activity in secondary endpoints and exploratory analyses, with overall safety consistent with previous data. The company will further analyze the full data and determine the subsequent development path after communicating with regulatory authorities.
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