Ionis Pharmaceuticals has secured FDA Breakthrough Therapy designation for its antisense oligonucleotide olezarsen, a first‑in‑class therapy that dramatically lowers triglycerides and reduces acute pancreatitis events in adults with severe hypertriglyceridemia. Phase 3 CORE and CORE2 trials demonstrated a 72 % reduction in fasting triglycerides and an 85 % reduction in pancreatitis events, the most substantial benefit seen to date for any investigational drug in this indication.
The designation extends olezarsen’s approved use beyond familial chylomicronemia syndrome—where it is marketed as TRYNGOLZA—to a broader population of patients with severe hypertriglyceridemia. Breakthrough status enables more frequent FDA interactions and may accelerate the review timeline, positioning the drug as a potential blockbuster asset. Ionis plans to file a supplemental New Drug Application by the end of 2025.
Ionis’s Q3 2025 earnings showed revenue up 17 % year‑over‑year to $157 million, with TRYNGOLZA contributing $32 million. Management raised 2025 revenue guidance to $875 million–$900 million and adjusted operating loss guidance to $275 million–$300 million, reflecting confidence in growth and disciplined cost management. CEO Brett P. Monia described the quarter as a watershed moment, noting that two independent launches are underway and two more are anticipated in 2026, underscoring the company’s strategy to deliver a steady cadence of new medicines.
Dr. Sam Tsimikas, Ionis’s senior vice president of global cardiovascular development, emphasized that the Breakthrough designation reflects the groundbreaking nature of the CORE and CORE2 data, which lowered triglycerides below the risk threshold for pancreatitis in the vast majority of patients. The drug’s mechanism—reducing hepatic production of apoC‑III—addresses a critical unmet need in a population of roughly 3 million U.S. patients with severe hypertriglyceridemia, of whom 1 million are high risk. Current therapies provide modest triglyceride reductions and fail to prevent pancreatitis attacks, whereas olezarsen’s 85 % reduction in pancreatitis events offers a transformative therapeutic option.
Ionis’s broader pipeline, including zilganersen for Alexander disease, and its evolution into a fully integrated commercial‑stage biotechnology company reinforce its competitive position. The company’s robust cash balance supports continued investment in development and commercialization while maintaining financial flexibility.
The announcement was well received by investors, reflecting confidence in olezarsen’s potential to reshape the cardiometabolic market and drive future revenue growth.
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