A Single Ascending Doses Phase I Study to Evaluate the Safety and Pharmacokinetics of RBD4059 in Healthy Subjects

Summary

This is a Randomized, Single-blind, Placebo-Controlled Phase I Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of Subcutaneously Administered RBD4059 in Healthy Subjects. The study will be performed in single ascending dose (SAD) phase in healthy subjects. The decision to escalate to subsequent dose levels will be made by the SRC based on the review of all available safety information and PK/PD data in each cohort. RBD4059, a GalNAc conjugated siRNA, is an FXI-targeted new molecular entity independently developed by Ribo using its proprietary siRNA delivery system - RIBO-GalSTARTM.

Timeline

Start

2023-03-21

Primary completion

2025-12

Completion

2025-12

Outcome

Met primary endpoint

release “Primary endpoint for safety and tolerability has been met – RBD4059 demonstrated favorable safety profile with no identified adverse safety signals across the investigated dose-range.” ribocure.com ↗

Publications

• Gabrielsen A, Ueckert S, Nilsson C, Egnell AC, Fu J, Wikstrom J, Svedlund S, Friend R, Zhang H, Liang Z, Gao S, Gan LM. RNA interference therapy targeting coagulation factor XI: a first-in-human trial of RBD4059 (vortosiran). Blood Adv. 2026 Apr 14;10(7):2541-2548. doi: 10.1182/bloodadvances.2025018348.

Drugs

Indications

No indication recorded.