Novartis is licensing ARO-SNCA, a preclinical siRNA therapy for synucleinopathies, a group of neurodegenerative disorders including Parkinson’s disease.
Arrowhead Pharmaceuticals has notched another major partnership with billion-dollar potential. Novartis has signed on to develop the RNA interference specialist’s preclinical Parkinson’s disease program for $200 million upfront.
On the back end, Arrowhead could be eligible for up to $2 billion in milestones, plus royalties on future sales, according to a Tuesday morning press release.
The deal adds to the list of heavyweight partners Arrowhead has collected, which already included Amgen, GSK and Takeda. Its biggest partnership, though, is with Sarepta Therapeutics; the November 2024 deal is worth upwards of $11 billion if all the programs are a success.
Novartis is licensing ARO-SNCA, a preclinical siRNA therapy for synucleinopathies, or neurodegenerative disorders such as Parkinson’s, that are caused by the buildup of the protein alpha-synuclein in the brain. The deal also includes other targets using Arrowhead’s Targeted RNAi Molecule, or TRiM, platform that have yet to be named, according to the release.
Arrowhead will continue with preclinical work for the programs up to a clinical trial application, when Novartis will take over.
Besides the partnered programs, Arrowhead has a wholly owned pipeline that includes lead asset plozasiran. The drug is currently awaiting a November decision from the FDA to treat familial chylomicronemia syndrome (FCS), a rare genetic metabolic disorder that prevents the body from breaking down fats.
The company is also working on two obesity assets, including ARO-ALK7, which is meant to silence the ACVR1C gene, in turn reducing the risk of obesity-related metabolic complications.
CEO Chris Anzalone told BioSpace last month that he aspires to build Arrowhead into a company like Vertex or Regeneron by building long-term value as an independent entity.
Novartis’ neurodegenerative pipeline is stacked with assets in multiple sclerosis, myasthenia gravis and Huntington’s disease. The program is anchored by approved therapies Mayzent and Fabhalta.
This is not Novartis’ first foray into siRNA therapies, however. The Swiss pharma has Leqvio, which is approved for lowering lipoprotein cholesterol. The drug was licensed from Alnylam, Arrowhead’s more-senior peer in the RNAi world, in January 2020.
