Study of ARO-ANG3 in Healthy Volunteers and in Dyslipidemic Patients

Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetcs and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial hypercholesterolemia and severe hypertriglyceridemia.

Timeline

Start

2019-01-07

Primary completion

2021-05-17

Completion

2021-05-17

Outcome

Met primary endpoint

paper No serious treatment-related adverse events, the primary endpoint of the trial, were observed. PMID 41946917 ↗

Publications

• Watts GF, Scott R, Sullivan D, Baker J, Clifton P, Hamilton J, Given B, Melquist S, Zhou R, Gaudet D, Goldberg IJ, Knowles JW, Leeper NJ, Hegele RA, Ballantyne CM. Zodasiran for cholesterol and triglyceride lowering in patients with hyperlipidemia: final report of phase 1 basket trial. Nat Med. 2026 Apr;32(4):1432-1443. doi: 10.1038/s41591-026-04307-8. Epub 2026 Apr 7.
• Watts GF, Schwabe C, Scott R, Gladding PA, Sullivan D, Baker J, Clifton P, Hamilton J, Given B, Melquist S, Zhou R, Chang T, San Martin J, Gaudet D, Goldberg IJ, Knowles JW, Hegele RA, Ballantyne CM. RNA interference targeting ANGPTL3 for triglyceride and cholesterol lowering: phase 1 basket trial cohorts. Nat Med. 2023 Sep;29(9):2216-2223. doi: 10.1038/s41591-023-02494-2. Epub 2023 Aug 25.
• Ying Q, Chan DC, Watts GF. Angiopoietin-like protein 3 inhibitors and contemporary unmet needs in lipid management. Curr Opin Lipidol. 2021 Jun 1;32(3):210-212. doi: 10.1097/MOL.0000000000000747. No abstract available.

Drugs

Indications