Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)
Summary
The purpose of AROAPOC3-3001 is to evaluate the efficacy and safety of ARO-APOC3 (plozasiran) in adult participants with familial chylomicronemia syndrome (FCS). Participants who have met all eligibility criteria will be randomized to receive 4 doses of plozasiran or matching placebo administered subcutaneously. Participants who complete the randomized period will continue in a 2-year open-label extension period where all participants will receive plozasiran.
Timeline
- Start
- 2021-12-14
- Primary completion
- 2024-04-29
- Completion
- 2026-04-21
Outcome
Met primary endpoint
paper the primary end point) was -80% in the 25-mg plozasiran group, -78% in the 50-mg plozasiran group, and -17% in the placebo group (P<0.001). PMID 39225259 ↗
release “PALISADE successfully met the primary endpoint of lowering triglycerides and met all key secondary endpoints” arrowheadpharma.com ↗
release “PALISADE successfully met its primary endpoint and all multiplicity-controlled key secondary endpoints” arrowheadpharma.com ↗
release “In PALISADE, 25 mg REDEMPLO reduced triglycerides by a median of 80% from baseline versus a 17% reduction with placebo.” arrowheadpharma.com ↗
release “In the PALISADE study, Plozasiran met its primary endpoint and all key secondary endpoints” visirna.com ↗
Publications
- Loomba R, de-Madaria E, Afghani E, Singh VK, Leeper NJ. Clinical Trial: Plozasiran Prevents Recurrent Pancreatitis in Adults With Very Severe Hypertriglyceridemia-Results of a Post Hoc Analysis of the Phase 3 PALISADE Study. Aliment Pharmacol Ther. 2026 May;63(9):1236-1245. doi: 10.1111/apt.70623. Epub 2026 Apr 1.
- Watts GF, Rosenson RS, Hegele RA, Goldberg IJ, Gallo A, Mertens A, Baass A, Zhou R, Muhsin M, Hellawell J, Leeper NJ, Gaudet D; PALISADE Study Group. Plozasiran for Managing Persistent Chylomicronemia and Pancreatitis Risk. N Engl J Med. 2025 Jan 9;392(2):127-137. doi: 10.1056/NEJMoa2409368. Epub 2024 Sep 2.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Plozasiran | Small interfering RNA (siRNA) | 25 mg | Subcutaneous |
| Subject | Plozasiran | Small interfering RNA (siRNA) | 50 mg | Subcutaneous |