Novartis is taking another shot at alpha-synuclein as a target for treating Parkinson’s disease via a $200 million upfront licensing deal with Arrowhead Pharmaceuticals.
The Swiss pharma plans to use Arrowhead’s preclinical siRNA program, dubbed ARO-SNCA, to target alpha-synuclein in order to treat Parkinson’s and additional synucleinopathies. Other pharmas have struggled to get their alpha-synuclein candidates to perform, including Novartis’ own UCB-partnered antibody minzasolmin, which flunked a phase 2 Parkinson's trial last year.
Still, Novartis has enough faith in Arrowhead’s approach to hand over $200 million, with up to $2 billion in development, regulatory and sales milestones set to come the biotech’s way should the bet pay off, along with tiered royalties.
In return, the pharma gains the exclusive rights to develop and commercialize the ARO-SNCA program, which uses Arrowhead’s RNAi platform, called TRiM, to subcutaneously deliver the siRNA therapy that targets the gene encoding the alpha-synuclein protein.
As part of the deal, Novartis can also select additional collaboration targets beyond Arrowhead’s own pipeline for the pharma to develop using the TRiM platform.
“We believe that one way to effectively target core drivers in Parkinson’s and other neurodegenerative diseases requires completely novel approaches to deliver RNA medicines to the brain,” Fiona Marshall, Ph.D., president of biomedical research at Novartis, said in the Sept. 2 release.
“We see Arrowhead’s TRiM technology as having great potential to achieve the type of widespread and effective delivery in key brain structures that will be necessary to see the full benefit of RNA medicines in neurodegeneration,” Marshall added.
Over the years, Arrowhead’s RNA tech has caught the attention of the likes of Takeda, Amgen, Sarepta Therapeutics, GSK and Sanofi, especially when it comes to cardiometabolic and rare disease applications. The biotech is currently awaiting an FDA approval decision for plozasiran to treat a rare genetic disorder called familial chylomicronemia syndrome.
Arrowhead is less well known in the CNS space, although one of the range of clinical-stage candidates from its Sarepta collaboration is aimed at a neurodegenerative disorder called spinocerebellar ataxia type 2.
Arrowhead CEO Christopher Anzalone, Ph.D., said the TRiM platform has already “generated impressive preclinical results demonstrating delivery to CNS, including distribution to deep brain regions, after subcutaneous administration.”
“The potential translation of these results in upcoming clinical trials would represent an important leap forward for neurodegenerative diseases and gene targets in the CNS that have been historically difficult to address,” Anzalone added in this morning’s release.
“We look forward to working with Novartis to bring ARO-SNCA for the treatment of synucleinopathies, such as Parkinson’s disease, into clinical trials as soon as possible and to collaborate on additional programs in the future,” the CEO said.