Novartis announced on Tuesday that its RNA drug candidate del-desiran failed to meet the primary endpoint in a late-stage trial for myotonic dystrophy type 1 (DM1), a rare muscle disease. The trial, which aimed to measure the drug's ability to improve hand muscle relaxation, did not show significant improvement over a placebo. However, researchers observed evidence of clinical activity on secondary goals and in exploratory analyses.
The setback comes as Novartis continues to evaluate the full dataset from the trial. Shreeram Aradhye, Novartis' chief medical officer, stated that developing therapies for complex diseases like DM1 remains challenging, and setbacks are part of scientific progress. The company remains committed to identifying the most appropriate development path for the del-desiran program and advancing innovative approaches for people living with DM1 and other serious neuromuscular diseases.
Novartis acquired del-desiran and its technology platform from Avidity Biosciences late last year for $12 billion. The platform uses targeted antibodies to shuttle pieces of engineered genetic material into muscle cells to silence disease-causing genes. Novartis also acquired other drugs, including del-zota and del-brax, which are in testing for Duchenne muscular dystrophy and facioscapulohumeral muscular dystrophy, respectively.
The trial failure led to a 13% drop in Novartis shares, with trading just under $139 per share by late Tuesday morning. Jefferies analyst Michael Leuchten noted that the Avidity acquisition came with controversy, raising questions about Novartis' due diligence and business development approach. Leuchten's team had previously projected peak annual sales of $1.5 billion for del-desiran and an 80% probability of success for the development program.
Cantor Fitzgerald analyst Eric Schmidt suggested that the high degree of variability in the hand relaxation test may have tripped up Novartis' study. The company also faces competition from Dyine Therapeutics, a Massachusetts-based biotechnology company working on a similar therapy for DM1. Following the Novartis news, Dyine Therapeutics' stock price dropped by 22%, and Cantor's team reduced the probability of success forecast for Dyine's medicine from 50% to 25%.
