Novartis announced that its global Phase III HARBOR study for delpacibart etedesiran (del-desiran) in myotonic dystrophy type 1 failed to meet the primary endpoint of video hand opening time versus placebo. The company observed evidence of clinical activity in secondary endpoints and is evaluating the full dataset with health authorities to determine the future development path. Novartis maintains its five-year sales CAGR guidance of 5-6% for 2025-2030. The filing also highlighted positive developments elsewhere in the neuromuscular pipeline, including FDA priority review designation for delpacibart zotadirsen in Duchenne muscular dystrophy and planned regulatory discussions for delpacibart braxlosiran in facioscapulohumeral muscular dystrophy based on Phase I/II biomarker data. Novartis is an innovative medicines company developing pharmaceuticals to treat serious diseases.

Novartis announced that its Phase III Lp(a)HORIZON trial for pelacarsen failed to meet its primary endpoint, showing no reduction in cardiovascular events compared to placebo despite achieving lower lipoprotein (a) levels. The study enrolled 8,323 patients with elevated Lp(a) and established cardiovascular disease. Novartis stated the findings provide important evidence on the relationship between Lp(a) lowering and outcomes but did not demonstrate risk reduction in the overall population. Pelacarsen is an investigational antisense oligonucleotide developed under a license from Ionis Pharmaceuticals to inhibit Lp(a) production. Novartis AG operates as an innovative medicines company focused on discovering, developing, and commercializing pharmaceutical products globally.

Novartis announced positive topline results from its Phase III REMODEL-1/-2 trials for remibrutinib, an oral BTK inhibitor, in patients with relapsing multiple sclerosis (RMS). The trials met their primary endpoint, demonstrating significant superiority over teriflunomide in reducing the annualized relapse rate and inflammatory brain lesions. Remibrutinib also showed clinically meaningful reductions in disability progression endpoints, including a positive trend in 3-month confirmed disability progression and nominally significant results for 6-month confirmed disability progression in a combined analysis. The drug exhibited a favorable safety profile with no liver safety signals across more than 4,500 clinical trial participants. Novartis plans to present this data at MSToronto2026 and intends to submit regulatory applications globally for remibrutinib in RMS. Novartis is an innovative medicines company developing pharmaceuticals to treat serious diseases.

Novartis announced that the FORTITUDE Phase I/II study of delpacibart braxlosiran (del-brax) met its primary and key secondary endpoints. The biomarker cohort, involving 51 patients with facioscapulohumeral muscular dystrophy (FSHD), showed reductions in KHDC1L and creatine kinase levels, indicating target engagement and reduced muscle damage. Del-brax is an investigational antibody oligonucleotide conjugate (AOC) acquired through the February 2026 Avidity Biosciences transaction. Following these results, Novartis plans to engage global regulatory authorities regarding the data while its Phase III study continues to enroll 200 patients. Novartis is a pharmaceutical company that develops innovative medicines.