Arrowhead Pharmaceuticals has entered into a global licensing and collaboration agreement with Novartis for its preclinical siRNA therapy, ARO-SNCA.
ARO-SNCA is intended for the treatment of synucleinopathies, a group of neurodegenerative diseases, including Parkinson’s Disease.
Under the terms of the licensing agreement, Novartis obtains an exclusive worldwide license to develop and commercialise ARO-SNCA and other targets.
Arrowhead Pharma will receive an upfront payment of $200m and is eligible to earn up to $2bn in milestone payments, alongside royalties on future commercial sales.
The transaction is anticipated to close in the second half of 2025, pending regulatory approvals and other customary conditions.
Novartis Biomedical Research president Fiona Marshall said: “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 collaboration will leverage Arrowhead’s Targeted RNAi Molecule (TRiM) platform for targeting alpha-synuclein, a protein linked to Parkinson’s Disease.
Arrowhead will conduct preclinical research to facilitate a clinical trial application. Novartis will oversee subsequent development, manufacturing, and marketing.
Gibson, Dunn & Crutcher served as legal advisor to Arrowhead on this transaction.
Arrowhead president and CEO Christopher Anzalone said: “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.”
In November last year, Arrowhead Pharma signed a licensing and collaboration agreement with US-based biotechnology company Sarepta Therapeutics.
The agreement covers multiple clinical and preclinical programmes in rare, genetic diseases of the muscle, central nervous system (CNS), and lungs.
