Get ready for a breakthrough in the treatment of severe hypertriglyceridemia (sHTG)! The latest study results are a game-changer, offering new hope for patients with this challenging condition.
Sobi, a leading biopharma company, has announced positive findings from their pivotal Phase 3 trials, CORE and CORE2, which investigated the effectiveness of olezarsen in managing sHTG. The results are nothing short of remarkable, with a highly significant reduction in fasting triglyceride levels, up to 72% at six months, and this improvement was sustained for a full year!
But here's where it gets even more impressive: olezarsen not only lowered triglyceride levels but also significantly reduced the risk of acute pancreatitis, a potentially life-threatening complication of sHTG. In fact, olezarsen showed an 85% reduction in acute pancreatitis events, a first in the treatment of this condition. And get this, 86% of patients treated with olezarsen achieved triglyceride levels below the risk threshold, a truly impactful outcome.
The studies included a diverse group of patients, with 43% having extremely high triglyceride levels often associated with multifactorial chylomicronemia syndrome (MCS). The positive results were consistent across this subgroup, demonstrating the broad effectiveness of olezarsen.
These groundbreaking findings were presented at the American Heart Association Scientific Sessions and simultaneously published in The New England Journal of Medicine, solidifying their credibility and impact.
Dr. Nicholas Marston, a cardiologist and presenting author, emphasized the significance of these results, especially for lipid specialists like himself who treat sHTG patients. He highlighted the major consequences of acute pancreatitis and the limited effectiveness of conventional therapies, making these new data a welcome advancement in the field.
Lydia Abad-Franch, Head of R&D and Chief Medical Officer of Sobi, expressed her encouragement and belief in the promise of olezarsen. She believes it offers new hope for improving the quality of life for patients with sHTG, who have long faced limited treatment options. Sobi is now moving forward with the European submission for olezarsen in MCS, planned for next year.
The CORE and CORE2 studies enrolled nearly 1,100 patients, making it the largest pivotal program ever conducted in sHTG. The studies met their primary endpoint, demonstrating the effectiveness of olezarsen across different doses. Olezarsen not only reduced triglyceride levels but also showed a highly statistically significant reduction in acute pancreatitis events, based on a comparison of 22 events in the placebo group versus only 7 events in the olezarsen group.
In terms of safety, olezarsen demonstrated a favorable profile. Adverse events were balanced across treatment arms, and serious adverse events occurred less frequently in the olezarsen group compared to placebo. The most common side effect was injection site reactions, which were mostly mild and more frequent with olezarsen.
An ongoing open-label extension study of olezarsen for sHTG is providing further insights, with over 90% of patients who completed CORE and CORE2 choosing to continue into this extension.
Sobi is preparing to submit a marketing authorization application for olezarsen in MCS to the European Medicines Agency (EMA) in 2026, while their partner Ionis is on track to submit a supplemental new drug application for sHTG to the FDA by the end of this year.
Severe hypertriglyceridemia (sHTG) is a serious condition characterized by extremely high triglyceride levels (≥500 mg/dL) and an increased risk of acute pancreatitis and other morbidities. Acute pancreatitis is a medical emergency, causing severe abdominal pain, prolonged hospitalization, and potential organ damage. Preventing the first attack is crucial, especially in individuals with triglyceride levels ≥880 mg/dL and a history of acute pancreatitis episodes, as their risk of future attacks is significantly higher.
Olezarsen, an investigational RNA-targeted medicine, is designed to lower the body's production of apoC-III, a protein that regulates triglyceride metabolism in the blood. It is approved in the U.S. and the European Union as TRYNGOLZA® for adults with familial chylomicronemia syndrome (FCS).
Olezarsen is being developed by Ionis Pharmaceuticals, with Sobi holding exclusive rights outside the U.S., Canada, and China to commercialize it as a potential treatment for FCS and severely elevated triglycerides.
Sobi, a global biopharma company, is dedicated to transforming the lives of people with rare diseases through innovative breakthroughs. With a presence across Europe, North America, the Middle East, Asia, and Australia, Sobi is committed to making a difference.
For more information and media inquiries, please visit Sobi's website (https://www.sobi.com/en) and LinkedIn page (https://www.linkedin.com/company/sobi/).
