Novartis licenses US$2.2B Parkinson’s drug from Arrowhead Pharmaceuticals

DENMARK—Novartis is once again focusing on the alpha-synuclein protein as a potential treatment for Parkinson’s disease by signing a licensing deal with Arrowhead Pharmaceuticals worth up to US$2.2 billion.

Despite a setback last year when Novartis’ own drug targeting alpha-synuclein failed in Phase II trials, the pharmaceutical giant has chosen to invest US$200 million upfront for an exclusive global license.

This license allows Novartis to develop, manufacture, and commercialize Arrowhead’s preclinical RNA interference (RNAi) therapy, known as ARO-SNCA.

Under this agreement, Arrowhead stands to earn an additional US$2 billion in milestone payments, along with royalties if the drug reaches the market.

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

ARO-SNCA is based on Arrowhead’s targeted RNAi molecule (TRiM) technology, which uses a natural cellular process to silence specific gene messages, thereby reducing the production of harmful proteins.

AR0-SNCA specifically targets the gene responsible for producing alpha-synuclein. Abnormal forms of this protein can misfold and accumulate in the brain, which is strongly linked to neurological disorders like Parkinson’s.

Novartis plans to initially focus on Parkinson’s disease as the primary indication and aims to explore other diseases related to alpha-synuclein as part of their collaboration.

Christopher Anzalone, CEO of Arrowhead, highlighted that ARO-SNCA has already demonstrated promising preclinical results.

He noted the therapy’s ability to reach deep brain areas effectively through subcutaneous injections.

Anzalone expressed enthusiasm about working with Novartis to advance ARO-SNCA into clinical trials for Parkinson’s and related diseases, while also exploring additional programs.

Arrowhead has a proven track record, having partnered with major pharmaceutical companies such as Takeda, GSK, and Sanofi, all of which have licensed RNAi-based technologies.

Additionally, the biotech recently secured a significant $500 million upfront deal with Sarepta Therapeutics.

Arrowhead is also awaiting FDA approval for plozasiran, a treatment for the rare genetic disorder familial chylomicronemia syndrome, with a decision expected by November 2025.

Novartis is making a second attempt to target alpha-synuclein with this new deal.

Their previous effort involved the oral drug minzasolmin, which was co-developed with UCB.

Unfortunately, this drug failed to meet its primary and secondary goals during a Phase IIa trial conducted in December 2024, resulting in the termination of that program.

Fiona Marshall, president of biomedical research at Novartis, emphasized the importance of innovative RNA medicine delivery methods to the brain.

She praised Arrowhead’s TRiM platform for its potential to effectively distribute treatments to brain regions that are crucial for addressing neurodegenerative diseases.

Alpha-synuclein has proven difficult for many drug developers.

 Roche and Prothena’s antibody, prasinezumab, yielded mixed Phase II results in early-stage Parkinson’s patients, but it has moved into Phase III trials nonetheless.

Similarly, Lundbeck’s alpha-synuclein-targeting drug Lu AF82422 showed no significant benefit for multiple system atrophy patients in a February 2024 Phase II readout, but the company has also advanced the candidate to Phase III.

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