Zodasiran for cholesterol and triglyceride lowering in patients with hyperlipidemia: final report of phase 1 basket trial
Authors' conclusion
These results indicate a favorable safety profile for zodasiran, with promise for correcting isolated hypercholesterolemia and moderate-to-severe hypertriglyceridemia
Study context
- Population
- Patients on lipid-lowering therapy with hyperlipidemia, familial hypercholesterolemia or moderate-to-severe hypertriglyceridemia13 in FH open-label extension
- Participants
- 9 / 17 / 6
- Exposure
- Zodasiran across hyperlipidemia cohorts
- Hyperlipidemian = 9
- Familial hypercholesterolemian = 17
- Moderate-to-severe hypertriglyceridemian = 6
- Outcome
- Serious treatment-related adverse events
Methodological detailsplacebo-controlled (hyperlipidemia cohort), 48-week open-label extension (FH cohort), Zodasiran subcutaneously on days 1 and 29; every 12 weeks in the extension
- Placebo-controlled (hyperlipidemia cohort)
- 48-week open-label extension (FH cohort)
- Zodasiran subcutaneously on days 1 and 29; every 12 weeks in the extension
Main finding
As reported in the abstract
- No serious treatment-related adverse events observed
Observational data: associations, not effects.
Secondary outcomes
Serum ANGPTL3 and triglycerides, all cohorts · week 16 (12 weeks postdosing)
- Reduction in serum ANGPTL3
- ≤−85.4%
- Reduction in triglycerides
- ≤−67.1%
- ANGPTL3 reduction sustained to end of open-label extension (FH cohort)
Safety
- No elevations in hepatic aminotransferases, bilirubin or glycated hemoglobin
- No drug discontinuations
Original publication
Zodasiran for cholesterol and triglyceride lowering in patients with hyperlipidemia: final report of phase 1 basket trial. Nat Med. Published 7 April 2026. doi:10.
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