Arrowhead Pharmaceuticals disclosed that the two‑year open‑label extension study of its RNAi therapy plozasiran, designed to lower apolipoprotein C‑III, produced sustained triglyceride reductions and a favorable safety profile in patients with severe hypertriglyceridemia. The data were presented at the American College of Cardiology’s 75th Annual Scientific Session and Expo, which ran from March 28‑30, 2026.
In the extension cohort, 96 % of patients with severe hypertriglyceridemia achieved triglyceride levels below 500 mg/dL, and 63 % reached levels under 150 mg/dL. No acute pancreatitis events were reported over the two‑year period, a key safety endpoint for this high‑risk population.
Dr. Christie M. Ballantyne, principal investigator of the study, highlighted the “dramatic and meaningful reductions in TGs” and noted that the absence of pancreatitis events was “particularly reassuring.” Arrowhead CEO Dr. Christopher Anzalone emphasized that the results reinforce the company’s “strong execution” and position the company well for the upcoming Phase III readouts and a potential supplemental New Drug Application later in 2026.
The long‑term data arrive on the heels of Arrowhead’s Q1 2026 financial results, which showed net income of $30.8 million and revenue of $264 million—an impressive turnaround from a net loss the previous year. The company’s pipeline, including the planned completion of Phase III trials (SHASTA‑3, SHASTA‑4, and MUIR‑3) by mid‑2026, is now bolstered by these robust safety and efficacy findings.
Regulatory support for plozasiran is strong, with U.S. FDA orphan and Fast Track designations and an EMA orphan designation. Arrowhead’s recent launch of REDEMPLO (tirzepatide) for familial chylomicronemia syndrome in Q1 2026 further demonstrates its transition to a commercial‑stage company, while the new data position plozasiran as a leading candidate in the limited hypertriglyceridemia market.
Overall, the two‑year data strengthen Arrowhead’s competitive stance, provide critical evidence for regulatory submissions, and underscore the company’s capacity to translate its RNAi platform into a marketable therapy for a high‑risk patient population.
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