Ionis reports positive data from study of olezarsen for hypertriglyceridemia
Ionis Pharmaceuticals has reported positive topline outcomes from the randomised, global Phase III Essence study of Tryngolza (olezarsen) in individuals with moderate hypertriglyceridemia and, or at risk for, atherosclerotic cardiovascular disease (ASCVD).
This condition is characterised by fasting triglyceride (TG) levels between ≥150mg/dL and <500mg/dL.
The multicentre, double-blind, placebo-controlled Essence study was carried out with the TIMI Study Group, the company's research partner.
It enrolled 1,478 subjects aged 18 years and above diagnosed with or at risk for ASCVD, and 9% of them had fasting triglycerides of ≥500mg/dL at baseline.
Subjects received either 50mg or 80mg of olezarsen or a placebo every four weeks via subcutaneous injection for 12 months.
The primary endpoint focused on the percentage change from baseline in fasting TG levels at the six-month mark, compared to the placebo.
Key secondary endpoints were changes in TG levels at 12 months, the proportion of subjects achieving fasting TG levels of <150mg/dL, and the percentage variations in other lipid parameters against the placebo over the treatment period.
The trial, which included subjects already on standard of care, lipid-lowering medications, achieved its primary endpoint.
A significant decrease in TG levels was observed, with a placebo-adjusted 61% decrease at the 80mg monthly dose and a 58% decrease at the 50mg dose over a six-month period, compared to the placebo.
In addition, the therapy met all key secondary endpoints, with the majority of subjects achieving normal TG levels of <150mg/dL.
Olezarsen also demonstrated a favourable tolerability and safety profile in the trial.
Ionis Pharmaceuticals global cardiovascular development senior vice-president Sam Tsimikas said: 'Following the Food and Drug Administration (FDA) approval and encouraging launch of Tryngolza (olezarsen) for people living with familial chylomicronaemia syndrome (FCS), a rare, genetic form of severely elevated TGs, these data support olezarsen's potential tobenefit the much broader population of people living with severe hypertriglyceridemia (sHTG).'
The investigational RNA-targeted medicine is designed to decrease the body's apolipoprotein C III (apoC-III) production, a protein for regulating the metabolism of triglyceride in the blood.
Last November, Ionis announced the design for a pivotal Phase III trial of ION582, an investigational treatment for Angelman syndrome (AS), following discussions with the US FDA.
"Ionis reports positive data from study of olezarsen for hypertriglyceridemia" was originally created and published by Clinical Trials Arena, a GlobalData owned brand.
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Novartis is investigating a broad portfolio of RLTs, exploring new isotopes, ligands and combination therapies to look beyond gastroenteropancreatic neuroendocrine tumors (GEP-NETs) and prostate cancer and into breast, colon, lung and pancreatic cancer. Novartis has established global expertise, with specialized supply chain and manufacturing capabilities across its network of RLT production sites. To support growing demand for RLTs, we have expanded production capabilities in Millburn (NJ), Zaragoza (Spain), Ivrea (Italy) and a state-of-the-art facility in Indianapolis (IN). In Carlsbad (CA), Novartis is establishing its third US-based RLT manufacturing site to support expanded use of RLTs, create resiliency in its manufacturing network and optimize the delivery of medicines to patients on the West Coast. DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as 'potential,' 'can,' 'will,' 'may,' 'could,' 'trend,' 'potentially,' 'upcoming,' 'progression,' 'progress,' 'investigating,' 'investing,' 'look beyond,' or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for Pluvicto, or regarding potential future revenues from Pluvicto. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. 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In particular, our expectations regarding Pluvicto could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise. About Novartis Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide. Reimagine medicine with us: Visit us at and connect with us on LinkedIn, Facebook, X/Twitter and Instagram. References Data on file. Pluvicto [prescribing information]. Millburn, NJ: Advanced Accelerator Applications USA, Inc.; 2025. An International Prospective Open-label, Randomized, Phase III Study Comparing 177Lu-PSMA-617 in Combination With SoC, Versus SoC Alone, in Adult Male Patients With mHSPC (PSMAddition). identifier: NCT04720157. 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AJR Am J Roentgenol. 2017;209(2):277-288. doi:10.2214/AJR.17.18264 Jurcic JG, Wong JYC, Knoc SJ, et al. Targeted radionuclide therapy. In: Tepper JE, Foote RE, Michalski JM, eds. Gunderson & Tepper's Clinical Radiation Oncology. 5th ed. Elsevier, Inc. 2021;71(3):209-249 # # # Novartis Media RelationsE-mail: Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
