Arrowhead Pharmaceuticals, Novartis Ink $2.2 Billion Deal for Parkinson’s Therapy and RNAi Platform

Arrowhead Pharmaceuticals Inc. has signed a global licensing and collaboration agreement with Novartis for ARO-SNCA, a preclinical small interfering RNA (siRNA) therapy targeting alpha-synuclein for synucleinopathies, including Parkinson’s disease. The deal also includes additional collaboration targets leveraging Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform.

Under the terms, Arrowhead will receive a $200 million upfront payment and is eligible for up to $2 billion in milestone payments, plus royalties on commercial sales.

The partnership aims to advance ARO-SNCA into clinical trials and explore further applications of Arrowhead’s TRiM platform, which has shown promise in delivering RNA-based therapies to the central nervous system (CNS). The collaboration aligns with Novartis’s focus on neurodegenerative diseases and genetic medicines, bolstering its neuroscience pipeline.

“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,” said Christopher Anzalone, Ph.D., President and CEO at Arrowhead. “Our TRiM platform has generated impressive preclinical 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 working 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 programs in the future.”

Fiona Marshall, Ph.D., President of Biomedical Research at Novartis, said, “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.”

The transaction is expected to close in the second half of 2025.