Novartis Inks $2.2 Billion Deal to License Arrowhead's Preclinical siRNA Therapy ARO-SNCA for Parkinson’s Disease

Sep 03, 2025 00:00 CST Updated 00:00

Novartis

Drug Development and Manufacturing

Arrowhead Pharmaceuticals

Pharmaceutical R&D Developer

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On September 2, Arrowhead Pharmaceuticals announced a global licensing and collaboration agreement with Novartis for ARO-SNCA, a preclinical stage siRNA therapy targeting α-synuclein for the treatment of synucleinopathies such as Parkinson's disease, along with additional collaborative targets leveraging Arrowhead’s proprietary Targeted RNAi Molecule (TRiM™) platform.

According to the agreement, upon completion of the transaction, Novartis will pay Arrowhead.A $200 million upfront payment.Arrowhead is also eligible to receive up to$2 billion in development, regulatory, and sales milestone payments.Arrowhead is also eligible for tiered royalties on commercial sales, which could reach double digits. The deal is expected to be completed byCompleted in the second half of 2025.

Through this transaction, Novartis will obtain global exclusive rights to research, develop, manufacture, and commercialize ARO-SNCA, a preclinical stage program utilizing Arrowhead's TRiM™ platform for subcutaneous administration and delivery to the central nervous system. It aims to target the gene encoding α-synuclein as a potential treatment for patients with Parkinson’s disease and other synucleinopathies. Novartis will select additional collaborative targets outside of Arrowhead's current pipeline developed using the TRiM™ platform.

For all licensed programs under the agreement, Arrowhead will conduct and complete the necessary preclinical research activities to submit a Clinical Trial Application (CTA). Novartis will have full control over development, manufacturing, medical affairs, and commercialization activities.

Arrowhead’s products target the liver, lungs, muscles, and nervous system, with indications covering areas such as hepatitis B, metabolic disorders, and cardiovascular diseases.Currently, Arrowhead has 16 products in its pipeline,Five products have entered Phase III clinical trials.

R&D Pipeline

Arrowhead Pharmaceuticals was founded in 1989 and listed on NASDAQ in 2004.As a pioneer and leader in the global siRNA field, Arrowhead treats refractory diseases by silencing disease-causing genes.Arrowhead therapy utilizes a wide range of RNA chemical combinations and efficient delivery methods to trigger the RNAi mechanism, inducing rapid, deep, and long-lasting knockdown of target genes and inhibiting the production of pathogenic target proteins.

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