Pelacarsen

Today’s agreement with Argo Biopharmaceutical follows a deal in January 2024 that saw Novartis hand over $185 million upfront.\n Novartis has returned to Argo Biopharmaceutical with $160 million in upfront cash to explore various cardiovascular meds in the Chinese biotech’s siRNA pipeline.The latest deal will allow Novartis to explore ANGPTL3—an RNAi therapeutic in phase 2 trials—in a combination study for dyslipidemia. The Swiss pharma will also have first right of negotiation to secure the license to ANGPTL3 further down the line.This morning’s multifaceted agreement also gives Novartis the license to a preclinical liver-delivered siRNA candidate outside of China. The drug is due to enter an international phase 1 study next year, and the companies have the option to share profits in both China and the U.S.Finally, Novartis has secured the option to license the ex-China rights for two discovery-stage, next-gen molecules being developed for mixed dyslipidemia and a related lipid disorder called hypertriglyceridemia.As well as the $160 million upfront fee, Argo will also be in line for milestone and option payments that could add up to $5.2 billion, alongside tiered royalties. Novartis also plans to participate in Argo’s next equity financing round.Today’s agreement follows a deal in January 2024 that saw Novartis hand over $185 million upfront for rights to two of Argo\'s clinical-phase RNAi cardiovascular disease programs.Dongxu Shu, Ph.D., and Patrick Shao, Ph.D., both of whom once worked at Arrowhead Pharmaceuticals, co-founded Argo in 2021. When they announced the first collaboration with Novartis last year, Shu called the alliance the “first significant overseas out-licensing transaction in the RNAi field from a Chinese biotech company.”In this morning’s release, Shu said the company was “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,” Shu added. “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.” Novartis’ cardiovascular offering already includes the bad-cholesterol–lowering med Leqvio alongside the heart failure medication Entresto. The pharma’s late-stage pipeline features pelacarsen, an antisense med aimed at preventing cardiovascular events in patients with elevated levels of lipoprotein(a).“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,” Shaun Coughlin, M.D., Ph.D., global head of cardiovascular and metabolism at the Novartis Institute for Biomedical Research, said in today’s release.“We are excited to build on our work with Argo through these new agreements, which include additional molecules,” Coughlin added.Back in June, Novartis doled out $25 million upfront as part of a four-year pact with ProFound Therapeutics to create new treatments for heart disease.

PRESS RELEASE Two VictORION studies will highlight Leqvio’s impact on patient quality of life, and as a cholesterol lowering monotherapyLp(a)FRONTIERS APHERESIS study will assess effect of pelacarsen on reducing need for lipoprotein apheresis – a cholesterol removal procedure similar to dialysisPARACHUTE-HF study will highlight the efficacy and safety of Entresto to treat heart failure with reduced ejection fraction due to chronic Chagas diseaseAdditional presentations will assess the safety of new pipeline asset abelacimab in atrial fibrillation Basel, August 18, 2025 – Novartis will present data from 19 abstracts across its cardiovascular portfolio at the 2025 European Society of Cardiology (ESC) Congress in Madrid from August 29 to September 1, 2025. “The latest data we are presenting at ESC will showcase how Novartis is pioneering groundbreaking treatments that can transform cardiovascular outcomes,” said Ruchira Glaser, M.D., Global Head, Cardiovascular, Renal and Metabolic Development Unit, Novartis. “With established treatments Entresto and Leqvio, we continue to explore new ways to improve patient care whilst also investigating broader populations who may benefit. With promising pipeline assets, pelacarsen and abelacimab, we are tackling areas of significant unmet need to address the myriad of factors that cause or worsen heart disease.” Key highlights of data accepted by ESC include: Medicine Presentation Title Presentation Details Pelacarsen Pelacarsen reduces the need for lipoprotein apheresis in patients with elevated lipoprotein(a) and cardiovascular disease: The Phase 3 Lp(a)FRONTIERS APHERESIS trial Station 12 (Research Gateway) Moderated ePoster August 29, 09:15-10:00 CEST Pelacarsen Association of elevated lipoprotein(a) with future major adverse cardiovascular events in recurrent ASCVD patients: analysis of a large US electronic health record database Station 2 (Research Gateway) Moderated ePoster August 30, 17:15 – 18:00 CEST Pelacarsen Economic burden of elevated lipoprotein(a) in secondary prevention of atherosclerotic cardiovascular disease cohort from England Discovery Pole (North Convention Centre) Poster August 29, 15:15 CEST Leqvio VICTORION-Difference study: Inclisiran-based strategy vs standard of care Madrid (Main Auditorium) Hot Line August 30, 17:00 CEST Leqvio VICTORION-Mono China: efficacy and safety of inclisiran as monotherapy in Chinese adults with low or moderate ASCVD risk and elevated LDL-C Discovery Pole (North Convention Centre) Poster August 29, 10:00 CEST Abelacimab Prior anticoagulation experience, bleeding risk, and safety of factor XI inhibition in atrial fibrillation: an analysis of AZALEA-TIMI 71 Science Box 3 (Research Gateway) Poster September 1, 14:30 CEST Abelacimab Renal function and factor XI inhibition in atrial fibrillation: a pre-specified analysis of AZALEA-TIMI 71 Science Box 3 (Research Gateway) Poster September 1, 15:00 CEST Entresto PARACHUTE-HF: Randomized trial comparing sacubitril/valsartan with enalapril in patients with HFrEF caused by chronic Chagas cardiomyopathy Madrid (Main Auditorium) Hot Line September 1, 8:15 CEST About Novartis in Cardiovascular DiseaseAt Novartis, our mission is to ensure no heart is lost too soon. We envision a world where preventable CV deaths are no longer part of our lives. We’re proud of the positive impact we’ve made over the past 40 years and remain dedicated to tackling the most challenging problems in CVD. Through cutting-edge science and technology, we are focusing on areas of high unmet need, including scaling our xRNA platform across multiple risk factors and pioneering breakthroughs for genetically driven CVD risk factors and common heart conditions, including atrial fibrillation. We also work with patients, healthcare professionals, and organizations around the world to improve CV care beyond medicine alone. Together, we can help people with CVD enjoy longer, healthier lives and more time with their loved ones. DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. 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