Novartis Inks $2B Deal with Arrowhead for Parkinson's siRNA Therapy

Novartis has entered into a licensing agreement with Arrowhead Pharmaceuticals for ARO-SNCA, a preclinical small interfering RNA (siRNA) therapy targeting synucleinopathies, including Parkinson's disease. The deal, announced on Tuesday, involves a $200 million upfront payment to Arrowhead, with the potential for up to $2 billion in milestone payments plus royalties on future sales.

Expanding the RNAi Landscape

ARO-SNCA is designed to treat neurodegenerative disorders caused by the accumulation of alpha-synuclein protein in the brain. The agreement also covers additional undisclosed targets utilizing Arrowhead's Targeted RNAi Molecule (TRiM) platform. Arrowhead will continue preclinical development until the clinical trial application stage, at which point Novartis will assume responsibility for further advancement.

This partnership marks another significant collaboration for Arrowhead, adding to its existing relationships with pharmaceutical giants such as Amgen, GSK, and Takeda. The company's most substantial partnership to date remains its November 2024 deal with Sarepta Therapeutics, valued at up to $11 billion.

Novartis Strengthens Neurodegenerative Pipeline

For Novartis, this agreement bolsters its already robust neurodegenerative disease portfolio, which includes assets for multiple sclerosis, myasthenia gravis, and Huntington's disease. The Swiss pharmaceutical company's program is anchored by approved therapies Mayzent and Fabhalta.

This is not Novartis' first venture into siRNA therapies. The company previously licensed Leqvio, an approved treatment for lowering lipoprotein cholesterol, from Alnylam in January 2020. The addition of ARO-SNCA further solidifies Novartis' commitment to RNA interference technology in treating complex neurological conditions.

Arrowhead's Growing Pipeline and Aspirations

While expanding its partnerships, Arrowhead continues to develop its wholly-owned pipeline. The company's lead asset, plozasiran, is currently awaiting an FDA decision in November for the treatment of familial chylomicronemia syndrome (FCS), a rare genetic metabolic disorder affecting fat breakdown.

Arrowhead is also advancing two obesity-related assets, including ARO-ALK7, which aims to silence the ACVR1C gene to reduce the risk of obesity-related metabolic complications. CEO Chris Anzalone has expressed ambitions to build Arrowhead into a company rivaling industry leaders like Vertex or Regeneron, focusing on long-term value creation as an independent entity.