Novo Nordisk halts two IL-6 heart trials as Novartis suffers Phase 3 setbacks; Ionis wins first Alexander disease approval

Novo Nordisk announced it has ended two late‑stage studies of its investigational IL‑6 inhibitor ziltivekimab, which was being evaluated for cardiovascular disease. The decision follows an earlier Phase 3 failure and has led analysts to question the viability of IL‑6 as a therapeutic target in this space.

Novartis reported a double setback, with a Phase 3 trial of del‑desiran in myotonic dystrophy type 1 missing its primary endpoint and another Phase 3 cardiovascular study also failing to demonstrate benefit. Both misses dampen expectations for the company’s recent acquisitions.

Ultragenyx disclosed that its Phase 3 trial in Angelman syndrome did not show a clinical benefit, adding another disappointment in the rare‑disease arena.

In contrast, Ionis Pharmaceuticals received FDA approval for its targeted therapy for Alexander disease, marking the first approved treatment for this rare neurodegenerative disorder.

The mixed results underscore the challenges of developing cardiovascular and rare‑disease drugs, even as investor interest in biopharma remains robust.