Novartis acquires blood-brain barrier technology for $125M amid pipeline setbacks

On September 16, 2026, Novartis exercised its option to acquire Sironax's blood-brain barrier drug delivery platform for $125 million. The acquisition aims to bolster Novartis's neuroscience pipeline following a series of clinical setbacks, including the simultaneous discontinuation of its experimental ALS drug VHB937 (lifonebart) after a failed Phase 2 trial. Sironax will retain rights to its early-stage clinical assets, including SIR2501, using the deal's proceeds to fund their development.

Novartis acquires Sironax platform

• ▪Novartis secured an exclusive option to evaluate and potentially acquire Sironax's platform in the summer of 2025.
• ▪Sironax was co-founded in 2017 by scientist Xiaodong Wang, who also co-founded the cancer drug company BeiGene, now known as BeOne Medicines.
• ▪Novartis exercised an option on September 16, 2026, to acquire full ownership of Sironax's brain delivery platform for $125 million.

Blood-brain barrier delivery technology

• ▪The blood-brain barrier acts as a protective filter that shields the brain from toxic substances but also prevents beneficial medicines from reaching the organ.
• ▪Sironax's platform technology uses brain delivery modules designed to transport large molecule drugs, including monoclonal antibodies, peptides, proteins, and gene therapies, across the blood-brain barrier.

Sironax retained clinical programs

• ▪Under the September 2026 acquisition agreement, Sironax retains the rights to develop, manufacture, and commercialize select clinical assets developed using its platform technology.
• ▪Sironax is advancing three early-stage clinical programs, including SIR2501 for amyotrophic lateral sclerosis, SIR4156 for metabolic dysfunction, and SIR9900 for inflammatory and immunological diseases.
• ▪Sironax's primary asset, SIR2501, is a SARM1 inhibitor that has received a fast track designation from the U.S. Food and Drug Administration.

Novartis neuroscience pipeline setbacks

• ▪Novartis's antibody oligonucleotide conjugate, delpacibart etedesiran, failed a Phase 3 trial for myotonic dystrophy type 1 in September 2026.
• ▪Novartis paused clinical trials evaluating its experimental cell therapy, rap-cel, after three patients died from life-threatening immune reactions in August 2026.
• ▪Novartis's antisense oligonucleotide, pelacarsen, failed a Phase 3 clinical trial for cardiovascular disease in late August 2026.

VHB937 ALS trial failure

• ▪The experimental drug VHB937 is designed to stabilize TREM2, a protein that regulates immune responses, inflammation, and waste clearance in the brain.
• ▪Novartis is continuing to recruit patients for a mid-stage clinical trial evaluating the experimental drug VHB937 in patients with Alzheimer's disease.
• ▪The Phase 2 trial of VHB937 enrolled 251 patients with early-stage amyotrophic lateral sclerosis to evaluate the drug's efficacy and safety in delaying disease progression.
• ▪Novartis discontinued development of its experimental amyotrophic lateral sclerosis drug VHB937, also known as lifonebart, after it failed a Phase 2 trial in September 2026.