Trial results for Plozasiran in Chinese adults with Familial Chylomicronemia Syndrome (FCS) were posted on ClinicalTrials.gov on 2026-02-27. The Phase 3 study demonstrated a median reduction of -89.5% in fasting serum triglycerides for the Plozasiran 50 mg dose at Month 10, compared to a 38.8% increase for the placebo group.
Background
Familial Chylomicronemia Syndrome (FCS) is a rare genetic disorder characterized by severely elevated triglyceride levels due to impaired clearance of chylomicrons. This condition can lead to recurrent episodes of acute pancreatitis, abdominal pain, and other serious health complications. Effective management often requires strict dietary restrictions, and therapeutic options are limited.
Trial design
This completed Phase 3 clinical study (NCT05902598) enrolled 37 Chinese adults with Familial Chylomicronemia Syndrome. The randomized, double-blinded, placebo-controlled trial had a randomized period of approximately 112 weeks, including a treatment period of up to 104 weeks. The study evaluated the efficacy and safety of Plozasiran injection, comparing two active treatment arms (Plozasiran 25 mg and Plozasiran 50 mg) against a placebo group.
Key results
The study reported significant reductions in key lipid parameters:
- Percent Change From Baseline in Fasting Serum Triglyceride (TG) at Month 10:
- Plozasiran 25 mg: Median -85.7%
- Plozasiran 50 mg: Median -89.5%
- Placebo: Median 38.8%
- Percent Change From Baseline in Fasting Serum TG at Months 10 and 12 (Averaged):
- Plozasiran 25 mg: Median -82.1%
- Plozasiran 50 mg: Median -86.6%
- Placebo: Median 49.5%
- Percent Change From Baseline in Fasting Serum Apolipoprotein C3 (APOC3) at Month 10:
- Plozasiran 25 mg: Median -92.55%
- Plozasiran 50 mg: Median -91.83%
- Placebo: Median 19.91%
- Percent Change From Baseline in Fasting Serum APOC3 at Month 12:
- Plozasiran 25 mg: Median -83.98%
- Plozasiran 50 mg: Median -88.16%
- Placebo: Median 22.56%
Statistical analyses using the Wilcoxon (Mann-Whitney) rank sum test showed significant differences between active treatment groups and placebo:
- For fasting serum TG at Month 10, the median difference between Plozasiran 25 mg and placebo was -101.7 (95% CI: -167.8 to -35.5), with a p-value of 0.0022.
- For fasting serum TG at Month 10, the median difference between Plozasiran 50 mg and placebo was -103.2 (95% CI: -168.9 to -37.5), with a p-value of 0.0022.
- For fasting serum APOC3 at Month 10, the median difference between Plozasiran 25 mg and placebo was -112.73 (95% CI: -140.47 to -85.0), with a p-value of 0.0008.
- For fasting serum APOC3 at Month 10, the median difference between Plozasiran 50 mg and placebo was -115.25 (95% CI: -141.89 to -88.61), with a p-value of 0.0004.
What this means
The results from this Phase 3 trial indicate that Plozasiran significantly reduces fasting serum triglyceride and apolipoprotein C3 levels in Chinese adults with Familial Chylomicronemia Syndrome. The substantial median reductions observed across both doses, coupled with statistically significant differences compared to placebo, suggest that Plozasiran could offer a meaningful therapeutic option for managing this rare genetic disorder, potentially reducing the risk of associated complications.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT05902598, titled "A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome," were posted on 2026-02-27 on clinicaltrials.gov.