Argo Expands Collaboration with Novartis with siRNA Deal
The latest multi-asset licensing and option agreement between Argos and Novartis, will grant the Swiss pharma giant rights to key siRNA molecules, building on the companies’ partnership.
Chinese clinical-stage biotech, Argo Biopharmaceutical, has entered into an additional licensing and option agreement with Novartis for multiple cardiovascular assets. This latest deal adds to the existing collaboration agreements between the two companies and represents the third one that includes assets from Argo’s pipeline (1).
Under the terms of the most recent agreement, Novartis has been granted an option to license ex-China rights to two discovery-stage small interfering RNA (siRNA) molecules — for treatment of severe hypertriglyceridemia (sHTG) and mixed dyslipidemia — and rights for first negotiation for BW-00112 (ANGPTL3), which is currently in Phase II trials in the U.S. and China. There are reciprocal license and profit-sharing options for an additional hepatic-delivered siRNA asset which is in investigational new drug (IND)-enabling studies and expected to begin multi-territorial Phase I studies in 2026 (1).
“We are thrilled to deepen our collaboration with Novartis, a global leader in cardiovascular, renal, and metabolic areas. This new collaboration further supports the innovation engine Argo has built to deliver best-in-class siRNA therapeutics while building a top-tier clinical development team across multiple geographies,” said Dr. Dongxu Shu, Co-Founder, Chairman of the Board, and CEO of Argo Biopharma, in a company press release (1). “Argo’s ambition is to become a global biotech, and corporate development activities are an important component to expand the reach of our hepatic and ex-hepatic siRNA therapeutics.”
Cardiovascular diseases remain the leading cause of death globally. Responsible for over 20.5 million deaths in 2021, cardiovascular-related diseases are projected to rise to 35.6 million deaths by 2050 — driven by aging populations and persistent risk factors such as high systolic blood pressure and dyslipidemia (2). RNA interference (RNAi)-based drugs offer a novel and precise approach by silencing the specific genes responsible for producing harmful proteins that drive disease progression. Unlike traditional therapies that target proteins after they are formed, RNAi therapeutics act earlier in the pathological process by targeting messenger RNA (mRNA) to prevent the production of disease-causing proteins, enabling more effective intervention at the genetic level (3).
By selectively degrading mRNAs for key regulatory proteins, siRNAs have demonstrated effectiveness in managing cardiovascular risk factors, such as high cholesterol and hypertension. Additionally, siRNAs targeting ANGPTL3 — a liver-produced protein that inhibits enzymes responsible for breaking down triglycerides and cholesterol — have demonstrated an ability to lower key cardiovascular risk factors such as triglycerides, low-density lipoprotein cholesterol (LDL-C), and other atherogenic lipoproteins (4).
ANGPTL3 as a therapeutic target has resulted in many companies actively developing ANGPTL3 inhibitors, including Arrowhead Pharmaceuticals (Zodasiran [ARO-ANG3]), Eli Lilly (Solbinsiran) and Regeneron Pharmaceuticals (Evkeeza).
“Long-acting siRNAs which are designed to deeply and durably target disease-causing proteins represent an important paradigm shift in prevention and treatment of cardiovascular diseases. We are excited to build on our work with Argo through these new agreements, which include additional molecules,” added Dr. Shaun Coughlin, Global Head of Cardiovascular and Metabolism at Novartis Biomedical Research, in the press release (1). “Novartis is committed to advancing innovative treatments for patients with cardiovascular conditions, and our collaboration with Argo further strengthens our efforts to advance potential new therapies that address unmet medical needs.”
As part of the deal, Argo will receive an upfront payment of USD 160 million and eligibility for milestone and option payments valued up to USD 5.2 billion, along with tiered royalties on commercial sales. The preceding deal between the two companies, which was announced in January 2024, included a USD 185 million upfront and USD 4+ billion in milestones for select cardiovascular RNAi assets (1).
References
Argo Biopharma. Argo Biopharma Announces Multi-Asset License and Option Agreements with Novartis for Novel Molecules for Cardiovascular Diseases. Press Release, Sep. 3, 2025.
Chong, B.; Jayabaskaran, J.; Jauhari, S.M.; et al. Global Burden of Cardiovascular Diseases: Projections from 2025 to 2050. Eur. J. Prev. Cardiol., 2025, 32 (11), 1001–1015.
Rychahou, P.G.; Jackson, L.N.; Farrow, B.J.; Mark Evers, B.; RNA Interference: Mechanisms of Action and Therapeutic Consideration. Surgery, 2006, 140 (5), 719–725.
Watts, G.F.; Chan, D.C.; RNA Interference Therapy for Targeting ANGPTL3 and Atherogenic Lipoproteins: Findings and Implications of a Recent Phase I Study. Clin. Transl. Med., 2023, 13 (11), 1484.