Key Highlights
- Arrowhead Pharmaceuticals (NASDAQ: ARWR) has entered into a global license and collaboration agreement with Novartis for ARO-SNCA, a preclinical siRNA therapy targeting alpha-synuclein for the treatment of synucleinopathies, including Parkinson’s Disease.
- Novartis will gain an exclusive worldwide license to develop, manufacture, and commercialize ARO-SNCA, while also selecting additional CNS collaboration targets using Arrowhead’s Targeted RNAi Molecule (TRiM™) platform.
- Upon closing, Arrowhead will receive an upfront payment of $200M, and remains eligible for up to $2B in development, regulatory, and sales milestones, plus tiered royalties up to low double digits.
- Arrowhead will complete preclinical work required for CTA filing, after which Novartis will lead global development and commercialization.
- The agreement underscores Novartis’ commitment to neurodegenerative diseases, leveraging RNAi as a novel modality to overcome barriers in CNS delivery.
Strategic Significance
For Arrowhead, the deal represents its largest CNS collaboration to date, validating the TRiM™ platform’s ability to achieve broad CNS distribution via subcutaneous delivery. For Novartis, it strengthens its RNA medicines portfolio, aligning with its broader neuroscience strategy to alter disease trajectories in Parkinson’s and related disorders.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops RNA interference (RNAi) therapies that silence disease-causing genes. Its proprietary TRiM™ platform enables precise and durable gene silencing with subcutaneous delivery, advancing programs across cardiometabolic, pulmonary, oncology, and CNS diseases. Headquartered in Pasadena, California, Arrowhead is committed to translating genetic insights into transformative medicines for patients worldwide.
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