The blood-brain barrier keeps pathogens out but it also keeps drugs from reaching the organ. Novartis is boosting its capabilities for brain drug delivery by acquiring a platform technology that Sironax developed to bring drugs across the protective membrane. The deal comes as scrutiny mounts on Novartis’s neuroscience pipeline following recent clinical trial setbacks.
Novartis has been looking at the Sironax technology for more than a year. Last summer, the Swiss pharmaceutical company secured an exclusive option to buy the platform. That deal permitted Novartis to evaluate the technology during an option period, after which it could buy it. Sironax announced Wednesday that Novartis will exercise that right by paying $125 million.
Sironax was co-founded in 2017 by Xiaodong Wang, the scientist who earlier in his career co-founded the cancer drug company BeiGene, now called BeOne Medicines. Waltham, Massachusetts-based Sironax describes its platform technology as “brain delivery modules” that are engineered to transport therapies across the blood-brain barrier. Those modalities include monoclonal antibodies, peptides, proteins, gene therapies, and other large molecule drugs. Sironax’s research includes neurodegeneration and neuroinflammation in diseases such as amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS).
“In diseases like ALS, MS, where you need [central nervous system] access, you’re able to do that, so that’s very differentiating as well for us,” Sironax CEO Shefali Agarwal said in an interview earlier this year.
The deal with Novartis allows Sironax to retain the right to develop, manufacture, and commercialize select assets the biotech developed using its technology. Sironax currently has three drugs in Phase 1b/2 testing: SIR2501 is a SARM1 inhibitor in development for ALS and chemotherapy-induced peripheral neuropathy; SIR4156 is a NAMPT activator in development for metabolic dysfunction; and SIR9900 is a brain-penetrating RIPK1 inhibitor for inflammatory and immunological diseases.
Measured by revenue, neuroscience is the smallest of Novartis’s four core therapeutic areas, but the company has been trying to change that. The neuroscience pipeline includes programs for MS, Alzheimer’s, and various neuromuscular disorders. Those programs represent multiple drug modalities that could use the Sironax technology.
Novartis is looking for some wins after some disappointing clinical trial outcomes. Last week, the drugmaker announced delpacibart etedesiran, an antibody oligonucleotide conjugate, failed its Phase 3 test in myotonic dystrophy type 1. The drug was one of three drug candidates from Novartis’s $12 billion Avidity Biosciences acquisition last year. Last month, Novartis said it would discontinue development of antibody drug lifonebart (formerly VHB937B1) following its Phase 2 failure in ALS.
Setbacks have hit other parts of the Novartis pipeline. The company has paused clinical trials evaluating an experimental cell therapy for autoimmune diseases and neurological disorders. In late August, the company was notified of life-threatening immune reactions in three patients. Those patients later died. Ahead of Labor Day weekend, Novartis reported pelacarsen, an antisense oligonucleotide, failed its Phase 3 test in cardiovascular disease.
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