Novartis disclosed a Phase III setback for delpacibart etedesiran (del-desiran) in myotonic dystrophy type 1, failing to improve disease progression versus placebo in the HARBOR trial. The result follows other recent turbulence for the company, raising the stakes for next-wave DMPK readouts and development choices in neuromuscular disease. Separately, Biohaven’s epilepsy candidate BHV-7000 faced an FDA partial clinical hold tied to requests for additional information about preclinical safety. The hold came shortly after Biohaven drew interest in a $350 million transaction involving the asset, complicating timelines for trial enrollment and future regulatory leverage. For investors and operators, these two updates highlight how preclinical data requests and single-trial endpoints can quickly reshape development paths—especially in programs marketed around next-generation mechanisms.