Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) has just released positive topline results from the pivotal phase 3 core and core2 studies of Olezarsen in individuals with severe hypertriglyceridemia (SHTG). The studies revealed that Olezarsen demonstrated a highly statistically significant placebo-adjusted mean reduction in fasting triglycerides of up to 72% and a highly statistically significant reduction in acute pancreatitis events of 85%, with favorable safety and tolerability.
In these studies, which make up the largest pivotal program for SHTG, nearly 1,100 patients were involved and were required to be on standard of care lipid-lowering therapy throughout the treatment period. The results showed that both 80 mg and 50 mg monthly doses of Olezarsen met the primary endpoint, with a highly statistically significant placebo-adjusted mean reduction in fasting triglyceride levels at six months.
The studies also met the secondary endpoint of reduction in acute pancreatitis events, with Olezarsen demonstrating a highly statistically significant 85% reduction in events compared to placebo. Furthermore, the drug demonstrated a favorable safety and tolerability profile, with adverse events generally balanced across treatment groups, and serious adverse events occurring less frequently in the Olezarsen groups compared to placebo.
Ionis plans to submit a supplemental new drug application (SNDA) to the U.S. Food and Drug Administration by the end of the year. Additionally, detailed data from the studies will be presented at an upcoming medical conference.
In another study, detailed data from the phase 3 essence study evaluating Olezarsen in individuals with moderate hypertriglyceridemia and elevated cardiovascular risk were recently published in the New England Journal of Medicine and presented during a session at the European Society of Cardiology Congress. The study supported the safety database of Olezarsen and demonstrated a favorable safety and tolerability profile.
The core and core2 studies were global, multicenter, randomized, double-blind, placebo-controlled trials investigating the safety and efficacy of Olezarsen for SHTG. Participants aged 18 and older with triglyceride levels ≥500 mg/dl were enrolled and were required to be on standard of care therapies for elevated triglycerides throughout the treatment period.
Severe hypertriglyceridemia (SHTG) is defined by severely high triglycerides (≥500 mg/dl) and is characterized by an increased risk of acute pancreatitis and other morbidities. It is considered a medical emergency and can cause debilitating abdominal pain, leading to prolonged hospitalization and sometimes permanent organ damage. The current standard of care therapies for SHTG and lifestyle modifications do not sufficiently or consistently lower triglyceride levels or reduce the risks of SHTG in all patients.
Olezarsen is an investigational RNA-targeted medicine designed to lower the body's production of apoc-III, a protein produced in the liver that regulates triglyceride metabolism in the blood. It is approved in the U.S. as Tryngolza® as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia (FCS).
Ionis Pharmaceuticals, Inc. has been a pioneer in RNA-targeted medicines and continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing.
These results position Ionis to set a new treatment standard for individuals with SHTG who are at risk of debilitating acute pancreatitis attacks, marking a major step forward in delivering transformative care to those who need it most. As a result of these announcements, the company's shares have moved 34.03% on the market, and are now trading at a price of $57.1425. Check out the company's full 8-K submission here.
