Ionis Pharma Posts Strong Phase 3 Data for Cholesterol Drug
Olezarsen shows an 85% reduction in acute pancreatitis events, a critical complication of severe hypertriglyceridemia, paving the way for a regulatory filing.
Ionis Pharmaceuticals has unveiled compelling Phase 3 trial results for its novel cholesterol-lowering drug, olezarsen, demonstrating a dramatic reduction in a life-threatening complication associated with severe hypertriglyceridemia (sHTG). The data, presented at the American Heart Association Scientific Sessions, showed the treatment cut episodes of acute pancreatitis by 85% in the study population, a clinically significant outcome that could position the drug as a new standard of care.
The Carlsbad, California-based biotech, with a market capitalization of approximately $11.9 billion, saw its shares react to the news, though they experienced a slight pullback of 4.66% to $70.12 in recent trading. The stock remains near its 52-week high of $76.78, reflecting sustained investor optimism in the company's pipeline.
The pivotal CORE and CORE2 studies evaluated olezarsen in patients with sHTG, a condition characterized by dangerously high levels of triglycerides in the blood. According to the company's official announcement, the trials met their primary endpoints, with patients receiving the drug experiencing a placebo-adjusted mean reduction in triglycerides of up to 72%.
More critically, the study highlighted the drug's protective effect against acute pancreatitis, a painful and potentially fatal inflammation of the pancreas that is a primary risk for patients with uncontrolled triglyceride levels. The 85% reduction in these events addresses a major unmet medical need in this patient population. Furthermore, 86% of patients treated with olezarsen saw their triglyceride levels fall below the 500 mg/dL threshold, a key marker for pancreatitis risk.
"These groundbreaking results show olezarsen has the potential to become the new standard of care for patients with sHTG, a serious and life-threatening condition," said Brett P. Monia, Ph.D., chief executive officer of Ionis. The company confirmed it is on track to submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for olezarsen by the end of 2025.
The positive data adds another layer of potential for olezarsen, which is also under review for a separate, rarer condition. An earlier New Drug Application for the treatment of familial chylomicronemia syndrome (FCS) was granted Priority Review by the FDA, with a regulatory decision date set for December 19, 2024. This dual-track regulatory progress underscores the drug's broad applicability in treating diseases driven by elevated triglycerides.
Wall Street has responded favorably to the drug's clinical momentum and Ionis's broader strategy. Analysts at Stifel have projected peak sales for olezarsen could reach $1.7 billion for the sHTG indication alone. Following strong third-quarter earnings, other firms have also revised their outlooks, with TD Cowen raising its price target on Ionis to $99 and Leerink Partners adjusting its target to $72, both maintaining buy-equivalent ratings. The consensus rating among analysts remains a 'Strong Buy', with an average price target of $83.11.
As Ionis prepares its sNDA filing, the focus will be on translating this strong clinical data into a commercial success. With a favorable safety profile reported in the trials and a clear clinical benefit, olezarsen is poised to become a significant new therapy for a patient group with limited options and a cornerstone of Ionis's growing commercial portfolio.