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Novartis will gain the exclusive worldwide licence to research, develop, manufacture, and commercialise Arrowhead\u2019s siRNA therapy ARO-SNCA. Credit: Taljat David via Shutterstock.com. <\/p>\n
Novartis is returning to the alpha-synuclein\u00a0protein as a possible treatment for Parkinson\u2019s disease, signing a licensing deal worth up to $2.2bn for Arrowhead Pharmaceuticals\u2019 preclinical candidate.<\/p>\n
Novartis, which experienced a Phase II failure with its own alpha-synuclein-targeting candidate late last year, will pay $200m upfront for the exclusive worldwide licence to research, develop, manufacture, and commercialise Arrowhead\u2019s siRNA therapy ARO-SNCA. <\/p>\n
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As part of the deal, Arrowhead is in line to receive a further $2bn in milestone payments, along with royalties on sales if the drug is approved in the future. The transaction is expected to close in the second half of 2025.<\/p>\n Arrowhead\u2019s ARO-SNCA is built upon the company\u2019s targeted RNAi molecule (TRiM) platform. Arrowhead uses RNAi, a natural mechanism used by your cells to regulate the expression of genes, to silence specific mRNAs and reduce the production of proteins that cause disease. <\/p>\n ARO-SNCA itself silences the alpha-synuclein gene \u2013 abnormal forms of the alpha-synuclein protein can misfold and clump together in the brain, leading to neurological diseases.\u00a0<\/p>\n Treatment of Parkinson\u2019s disease will be the first indication targeted by Novartis when it has its hands on ARO-SNCA. Further synucleinopathies will follow as part of the collaboration, the company stated.<\/p>\n Arrowhead\u2019s CEO Christopher Anzalone said the candidate has already \u201cgenerated impressive preclinical results\u201d, including distribution to deep brain regions via subcutaneous injection.<\/p>\n Anzalone said: \u201cWe look forward to working with Novartis to bring ARO-SNCA for the treatment of synucleinopathies, such as Parkinson\u2019s disease, into clinical trials as soon as possible and to collaborate on additional programs in the future.\u201d<\/p>\n Arrowhead\u2019s platform has already been tapped by several big pharma companies in recent years, with Takeda, GSK and Sanofi all agreeing deals with the RNA-specialist. There was also a deal with rare disease specialist Sarepta Therapeutics that involved a hefty $500m upfront payment to Arrowhead.<\/a> In terms of its own pipeline, the US biotech is currently awaiting an approval decision<\/a> from the US Food and Drug Administration (FDA) due in November 2025 for plozasiran to treat the rare genetic disorder familial chylomicronemia syndrome.<\/p>\n Alpha-synuclein\u2019s many suitors<\/p>\n For Swiss drugmaker Novartis, the licensing deal marks a second attempt at unlocking the alpha-synuclein target, following a setback for one of its own candidates earlier this year. Minzasolmin, an oral drug co-developed with UCB, flunked a Phase IIa trial<\/a> in December 2024. The alpha-synuclein misfolding inhibitor missed the study\u2019s primary and secondary endpoints, resulting in programme termination.<\/p>\n Novartis\u2019 biomedical research president Fiona Marshall said: \u201cWe believe that one way to effectively target core drivers in Parkinson\u2019s and other neurodegenerative diseases requires completely novel approaches to deliver RNA medicines to the brain. <\/p>\n \u201cWe see Arrowhead\u2019s 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.\u201d<\/p>\n
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