Novartis once again tries on Parkinson’s Island to raise $200 million for Arrow’s RNAi therapy
Parkinson's disease is associated with the accumulation of specific brain proteins that are currently not approved to address the therapy. Novartis is one of the companies pursuing this goal, but its partnership program failed in the clinic. The pharmaceutical giant is trying again, this time to get the right to preclinical RNA therapy from Arrowhead.
Novartis has obtained an exclusive global license to develop and commercialize arrow therapy ARO-SNCA, under the terms of the deal announced Tuesday. After the deal is over, Arrow will receive a $200 million advance from Novartis.
Arrows specializes in the therapies that utilize cellular mechanisms called RNA interference (RNAI). Using small interfering RNA (siRNA), its therapy triggers this interfering mechanism to suppress gene expression, thus shooting down the production of the protein that causes the disease. Although Arrowhead has not yet obtained any approved siRNA therapy, its research has led to partnerships with GSK, Amgen, Takeda Pharmaceutical and Sarepta Therapeutics.
The target of ARO-SNCA is alpha synuclein. In Parkinson's disease, a misfolded version of this protein accumulates in the brain, helping the disease progression. Arrowhead has not spoken publicly about Aro-SNCA so far. In a Tuesday announcement, Arrowhead President and CEO Christopher Anzalone said the therapy comes from its target RNAi molecule (TRIM) platform technology, and its preclinical results demonstrate the delivery to the central nervous system, including deep in the brain.
Novartis believes Parkinson's therapy has enough potential to reach an agreement. The protocol puts Arrows in charge of preclinical work to support clinical trial applications. After that, Novartis will take over the development of assets and potential commercialization. This collaboration could extend to other diseases driven by aggregation of alpha synuclein. Novartis can also choose other targets as long as they go beyond the current pipeline of the arrow. Arrowhead could receive milestone payments of up to $2 billion. If the alliance's drugs arrive in the market, then biotech will receive royalties from sales.
“We believe that one approach to effectively targeting the core drivers in Parkinson's disease and other neurodegenerative diseases requires a completely novel approach to delivering RNA drugs to the brain,” Fiona Marshall, president of biomedical research at Novartis, said in a prepared statement. “We believe that the pruning technology of arrows has great potential to achieve widespread and efficient delivery types in key brain structures, which is essential to see the full benefit of RNA drugs in neurodegeneration.”
Novartis first tries to use alpha synuclears through a UCB partnership that begins in 2021. The alliance spans two small molecule inhibitors of problem proteins. Last December, the UCB reported that one of the molecules failed to achieve the primary or secondary goals of the mid-term clinical trial. Novartis Pipeline no longer lists these molecules.
This is not the first time Novartis has reached an RNAi transaction based on preclinical data. In 2023, Novartis paid $500 million to obtain DTX Pharma, a startup whose lead program is preparing for phase one test in Charcot-Marie-Marie-tooth disease, a rare neuromuscular disease. During the deal, Novartis executives told Medcity News that in addition to DTX's main assets, the Pharma giant attracted the startup's platform technology.
Novartis' first experience in RNA interference was also through commercial transactions. Cholesterol-lowering drugs containing the brand LEQVIO cholesterol-containing drugs came from Novartis for $9.7 billion in acquisition of the drug company. The drug's revenue in 2024 was $754 million, an increase of 112% from the previous year.
The Novartis agreement with Arrows is expected to end by the end of this year. Companies that have achieved clinical development using alpha synuclein-targeted drugs include SANOFI, AC Immune and Ionis Pharmaceuticals.
Photo: Adrian Moser/Bloomberg, via Getty Images
