Arrowhead Pharmaceuticals has entered into a global licensing and collaboration agreement with Novartis for ARO-SNCA, Arrowhead’s pre-clinical stage siRNA therapy against alpha-synuclein for the treatment of synucleinopathies, such as Parkinson’s Disease, and for other additional collaboration targets that will utilise Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform. Upon closing, Arrowhead will receive USD 200 million as an upfront payment, and is eligible to receive up to USD 2 billion in potential milestone payments plus royalties on commercial sales.

Under the terms of the agreement, Novartis will receive an exclusive worldwide licence to research, develop, manufacture and commercialise ARO-SNCA, a pre-clinical stage programme that utilises Arrowhead’s TRiM platform for subcutaneous administration and delivery to the CNS designed to target the gene that encodes the alpha-synuclein protein as a potential treatment for patients with Parkinson’s Disease, and other synucleinopathies. Novartis will select additional collaboration targets outside of Arrowhead’s current pipeline to be developed using the TRiM platform.

For all licensed programmes under the agreement, Arrowhead will conduct and complete pre-clinical research activities necessary to enable a Clinical Trial Application (CTA) filing. Novartis will then assume sole control over development, manufacturing, medical affairs and commercialisation activities.

 “With a robust neuroscience pipeline and clear commitment to neurodegenerative diseases and genetic medicines, Novartis is a compelling partner for Arrowhead in the CNS space. Our TRiM platform has generated impressive pre-clinical results demonstrating delivery to CNS, including distribution to deep brain regions, after subcutaneous administration. The potential translation of these results in upcoming clinical trials would represent an important leap forward for neurodegenerative diseases and gene targets in the CNS that have been historically difficult to address. We look forward to work with Novartis to bring ARO-SNCA for the treatment of synucleinopathies, such as Parkinson’s Disease, into clinical trials as soon as possible and to collaborate on additional programmes in the future,” said Christopher Anzalone, Ph.D., President and CEO, Arrowhead.

Upon closing, Novartis will make a USD 200 million upfront payment to Arrowhead.

Arrowhead is also eligible to receive development, regulatory and sales milestone payments of up to USD 2 billion. It is further eligible to receive tiered royalties on commercial sales up to the low double digits. The transaction is expected to close in the second half of 2025, subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions.

Fiona Marshall, Ph.D., President–Biomedical Research, Novartis, added, “Neurodegenerative conditions such as Parkinson’s Disease affect millions of patients worldwide. Novartis aspires to transform the lives of patients and families living with these diseases, by advancing medicines that significantly alter the course of the disease. We believe that one way to effectively target core drivers in Parkinson’s and other neurodegenerative diseases requires completely novel approaches to deliver RNA medicines to the brain. We see Arrowhead’s TRiM technology as having great potential to achieve the type of widespread and effective delivery in key brain structures that will be necessary to see the full benefit of RNA medicines in neurodegeneration.”