Novartis AG (NYSE:NVS) announced Thursday it terminated a mid-stage trial for an experimental amyotrophic lateral sclerosis (ALS) drug.
However, the Swiss pharmaceutical giant continues to invest heavily in central nervous system treatments, exercising a $125 million option to acquire global rights to a brain delivery platform from biotechnology firm Sironax.
ALS Drug Trial Discontinued
The Swiss drugmaker discontinued the development of VHB937 for ALS after the experimental drug missed its primary and secondary endpoints in a Phase 2 study.
The clinical trial thoroughly assessed the drug’s safety and efficacy in patients who have early-stage ALS within two years of symptom onset.
Following initial Media reports, the company confirmed the trial’s failure in an email statement to Reuters.
Despite the ALS setback, the pharmaceutical company continues evaluating VHB937 for Alzheimer’s disease, a progressive brain disorder.
Brain Delivery Platform Acquisition
On Thursday, Sironax announced that Novartis exercised an exclusive option to purchase its proprietary brain delivery platform.
Originating from a previous July 2025 agreement, the deal grants Sironax $125 million upon transaction closure, though the biotech firm retains rights to develop specific assets using the technology.
Sironax engineered the platform to cross the blood-brain barrier, successfully enabling the in vivo transport of large biologics, gene therapies, and monoclonal antibodies into the brain.
This technology aims to expand treatment possibilities for severe neurological disorders, including Parkinson’s, Huntington’s, and Alzheimer’s.
Advancing Clinical Programs
Beyond the acquisition, Sironax actively advances three clinical programs currently in Phase 1b/2 trials.
These include SIR2501, an FDA Fast Track-designated treatment for ALS and peripheral neuropathy; SIR4156, targeting metabolic dysfunction; and SIR9900, aimed at inflammatory and immunological diseases.
NVS Price Action: Novartis shares were up 1.37% at $140.19 at the time of publication on Thursday, according to Benzinga Pro data.
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