By Fineline Cube 
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today unveiled a worldwide licensing and collaboration agreement with Novartis (NYSE: NVS) to advance its pre‑clinical siRNA candidate ARO‑SNCA, designed to silence the alpha‑synuclein gene in Parkinson’s disease and other synucleinopathies.
Deal Highlights
- Exclusive Worldwide License – Novartis gains the right to research, develop, manufacture, and commercialize ARO‑SNCA, and to select additional targets for the TRiM platform.
- Upfront Payment – Novartis will pay USD 200 million upon closing.
- Milestone Potential – Up to USD 2 billion in development, regulatory, and sales milestones, plus tiered royalties in the low double digits.
- Timeline – Expected to close in the second half of 2025, with Arrowhead completing all pre‑clinical work required for a clinical trial application (CTA).
ARO‑SNCA & the TRiM Platform
| Feature | Description |
|---|---|
| Mechanism | siRNA that knocks down alpha‑synuclein mRNA, reducing toxic protein aggregation in the CNS. |
| Delivery | Subcutaneous administration via Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform, engineered for efficient CNS uptake. |
| Clinical Readiness | Pre‑clinical data demonstrate robust target engagement, favorable pharmacokinetics, and an excellent safety profile. |
Strategic Implications
- For Arrowhead – The deal unlocks significant capital and milestone upside while shifting the development burden to Novartis, allowing Arrowhead to focus on future pipeline assets.
- For Novartis – Access to a cutting‑edge RNAi technology and a first‑in‑class Parkinson’s candidate expands its neuro‑oncology and neurodegenerative disease portfolio.
- For Patients – ARO‑SNCA represents a novel therapeutic modality that could slow or halt disease progression in Parkinson’s disease and related synucleinopathies.-Fineline Info & Tech