Novartis Shares Slide After Neuromuscular Candidate Acquired with Avidity Fails Phase III Trial

Novartis stock experienced its largest single-day drop since the COVID-19 pandemic after the pharmaceutical company announced that its neuromuscular candidate delpacibart etedesiran (del-desiran) failed a crucial Phase III trial evaluating the antibody oligonucleotide conjugate (AOC) in patients with myotonic dystrophy type 1 (DM1).

Del-desiran fell short of the study's primary objective of demonstrating statistically significant improvement compared to placebo in video Hand Opening Time (vHOT), a widely used measure of hand myotonia. Despite this setback, Novartis acknowledged evidence of clinical activity in exploratory analyses and secondary endpoints, including hand grip strength, quantitative Muscle Testing composite score, muscle activity and participation scale, and the 10-Meter Walk/Run Test.

The company stated that it is thoroughly examining the complete HARBOR trial dataset and will engage with regulatory authorities to determine the most suitable development path for del-desiran. Novartis' president and chief medical officer, Shreeram Aradhye, emphasized that setbacks are inherent in scientific progress and that the company remains committed to advancing innovative therapies for individuals with DM1 and other severe neuromuscular diseases.

This failure marks Novartis' third clinical setback in a week, following the Phase III Lp(a)HORIZON trial failure of their co-developed pelacarsen and the recent suspension of eight trials assessing their autoimmune and neurological disease candidate rapcabtagene autoleucel (rap-cel) due to three patient deaths from immune effector cell-associated hemophagocytic syndrome.