Novartis is making another push at alpha-synuclein in Parkinson’s disease through a $200 million upfront licensing agreement with Arrowhead Pharmaceuticals.
The drugmaker intends to advance Arrowhead’s preclinical siRNA program, ARO-SNCA, to look toward alpha-synuclein for Parkinson’s and other synuclein-related disorders. Progress in this area has been elusive, with several pharma efforts falling short, notably including Novartis’ own UCB-partnered antibody, minzasolmin, which failed a phase 2 Parkinson’s study in 2024.
Novartis is backing Arrowhead’s siRNA strategy with a $200 million upfront payment, plus as much as $2 billion in potential development, regulatory and commercial milestones, along with tiered royalties if the program succeeds.
In exchange, the Swiss pharma secures exclusive global rights to advance and market ARO-SNCA, which applies Arrowhead’s TRiM RNAi platform to deliver the therapy subcutaneously against the gene responsible for producing alpha-synuclein.
The agreement also gives Novartis the option to nominate further collaboration targets outside of Arrowhead’s pipeline, which the pharma could then pursue with the TRiM platform.
Fiona Marshall, president of biomedical research at Novartis, stated in the release that advancing RNA medicines for Parkinson’s and other neurodegenerative conditions would demand entirely new strategies for delivery into the brain. She further explained that Arrowhead’s TRiM platform could be particularly well-suited to provide the broad and efficient distribution within critical brain regions required to unlock the full therapeutic benefit of RNA-based treatments in neurodegeneration.
Arrowhead’s RNA technology has long attracted the interest of major players like GSK, Takeda, Sarepta Therapeutics, Amgen and Sanofi, particularly for programs in cardiometabolic and rare diseases. The company is currently awaiting an FDA verdict on plozasiran, its therapy for the rare inherited condition familial chylomicronemia syndrome.
Arrowhead has a comparatively smaller footprint in the central nervous system space, though its collaboration with Sarepta includes a clinical-stage program targeting spinocerebellar ataxia type 2, a rare neurodegenerative disease.
Arrowhead CEO Christopher Anzalone, Ph.D., noted that the TRiM platform has produced strong preclinical data, showing successful delivery into the CNS through subcutaneous dosing, including penetration into deep brain regions.
Anzalone stated in the morning release that moving these findings into upcoming clinical trials could mark a major advance for neurodegenerative disorders and historically challenging CNS gene targets. He also emphasized that the company was eager to work with Novartis to advance ARO-SNCA into trials for synucleinopathies such as Parkinson’s disease as quickly as possible, while also pursuing future collaborative programs.
This news comes on the back of Novartis’ recent announcement that the firm will part ways with 58 employees working at its New Jersey headquarters. These layoffs come after a separate workforce reduction earlier this year that impacted 426 staff members. Announced in May, those cuts were tied to a restructuring of the company’s cardiovascular business, driven by the looming loss of Entresto’s exclusivity and the ongoing expansion and introduction of newer heart medicines.
