vortosiran (RBD4059) / Suzhou Ribo - LARVOL DELTA

A Single Ascending Doses Phase I Study to Evaluate the Safety and Pharmacokinetics of RBD4059 in Healthy Subjects (clinicaltrials.gov) - P1 | N=32 | Completed | Sponsor: Suzhou Ribo Life Science Co. Ltd. | Active, not recruiting ➔ Completed

Trial completion

Suzhou Ribo Life Science Co., Ltd….announced at China Pharmaceutical Innovation Conference (CPIC) in Shanghai, the positive results from a Phase 2a study in Europe evaluating vortosiran (RBD4059), Ribo's siRNA therapy targeting coagulation Factor XI (FXI), in patients with chronic coronary artery disease. (Press Release Hub) - "The randomized, double-blind, placebo-controlled study demonstrated that vortosiran in patients receiving standard of care, aspirin, treatment was generally well tolerated and achieved profound, dose-dependent, and long-lasting suppression of FXI activity (NCT06717074, clinicaltrials.gov). Patients with chronic CAD with previous myocardial infarction on aspirin were investigated in the current trial. Patients completing the high dose group vortosiran dosing regimen, at a maintenance dose of 400mg, achieved a mean maximum reduction in FXI activity of 92%, which sustained for several months following dosing."

P2a data • Coronary Artery Disease

A Study to Evaluate Safety, Pharmacokinetics and Pharmacodynamics of RBD4059 in Participants With Stable Coronary Artery Disease (clinicaltrials.gov) - P2 | N=30 | Completed | Sponsor: Ribocure Pharmaceuticals AB | Active, not recruiting ➔ Completed

Trial completion • Cardiovascular • Coronary Artery Disease

A Study to Evaluate Safety, Pharmacokinetics and Pharmacodynamics of RBD4059 in Participants With Stable Coronary Artery Disease (clinicaltrials.gov) - P2 | N=30 | Active, not recruiting | Sponsor: Ribocure Pharmaceuticals AB | Trial completion date: Dec 2025 ➔ Apr 2026 | Trial primary completion date: Dec 2025 ➔ Apr 2026

Trial completion date • Trial primary completion date • Cardiovascular • Coronary Artery Disease

RNA interference therapy targeting coagulation factor XI - a first-in-human trial of RBD4059. (PubMed, Blood Adv) - P1 | "Modeling of the available PD data supports clinical dosing schemes of 3-6 months to achieve a robust antithrombotic effect. Altogether, these observations support the further clinical development of RBD4059 to provide an siRNA-based therapeutic option targeting FXI for patients inadequately treated with or contraindicated for thrombosis prophylaxis."

First-in-human • Journal • P1 data • Cardiovascular • Hematological Disorders • Thrombosis

A Study to Evaluate Safety, Pharmacokinetics and Pharmacodynamics of RBD4059 in Participants With Stable Coronary Artery Disease (clinicaltrials.gov) - P2 | N=30 | Active, not recruiting | Sponsor: Ribocure Pharmaceuticals AB | Recruiting ➔ Active, not recruiting

Enrollment closed • Cardiovascular • Coronary Artery Disease

Inhibition of Factor XI Using RBD4059: A Novel GalNAc-siRNA With Potent and Durable Antithrombotic Effects. (PubMed, JACC Basic Transl Sci) - "In this study, RBD4059 demonstrated potent FXI inhibition with long duration in mice and monkeys, antithrombotic effects in mouse thrombosis models, and no prolonged bleeding in a mouse tail bleeding model. These findings make RBD4059 a promising candidate for the development of a safe and efficient antithrombotic drug."

Journal • Cardiovascular • Hematological Disorders • Thrombosis

A Study to Evaluate Safety, Pharmacokinetics and Pharmacodynamics of RBD4059 in Participants with Stable Coronary Artery Disease (clinicaltrials.gov) - P2 | N=30 | Recruiting | Sponsor: Ribocure Pharmaceuticals AB

New P2 trial • Cardiovascular • Coronary Artery Disease

A Single Ascending Doses Phase I Study to Evaluate the Safety and Pharmacokinetics of RBD4059 in Healthy Subjects (clinicaltrials.gov) - P1 | N=32 | Active, not recruiting | Sponsor: Suzhou Ribo Life Science Co. Ltd. | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2024 ➔ Dec 2025 | Trial primary completion date: Oct 2024 ➔ Dec 2025

Enrollment closed • Trial completion date • Trial primary completion date

First-in-human study to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of RBD4059, a GalNAc-siRNA drug targeting coagulation factor XI (FXI), in healthy subjects (ESC 2024) - P1 | "As of January 25, 2024, 24 subjects (with follow-up to week 24) with demographics and baseline clinical characteristics shown in Table 1 were enrolled. RBD4059 was well tolerated (Table 2), all related AEs were grade 1. There was no evidence of any clinically relevant bleeding events."

Clinical • P1 data • PK/PD data • Cardiovascular

A Single Ascending Doses Phase I Study to Evaluate the Safety and Pharmacokinetics of RBD4059 in Healthy Subjects (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: Suzhou Ribo Life Science Co. Ltd. | N=64 ➔ 40 | Trial completion date: Mar 2025 ➔ Dec 2024

Enrollment change • Trial completion date

A Single and Multiple Ascending Doses Phase I Study to Evaluate the Safety and Pharmacokinetics of RBD4059 in Healthy Subjects (clinicaltrials.gov) - P1 | N=64 | Recruiting | Sponsor: Suzhou Ribo Life Science Co. Ltd. | Not yet recruiting ➔ Recruiting | Trial completion date: Oct 2024 ➔ Mar 2025

Enrollment open • Trial completion date

Inhibition of Factor XI using a GalNAc-siRNA RBD5049: a novel antithrombotic drug with high potency and long duration (ESC 2023) - "Enoxaparin sodium (Lovenox) with clinical equivalent dose (4 mg/kg) was used as a control. RBD4059 has demonstrated high potency/efficacy and long duration regarding FXI inhibition and with large safety margin. As the first siRNA based molecule targeting FXI, RBD4059 has now entered Phase 1 trial in human healthy volunteers."

Cardiovascular • Thrombosis • Venous Thromboembolism

A Single and Multiple Ascending Doses Phase I Study to Evaluate the Safety and Pharmacokinetics of RBD4059 in Healthy Subjects (clinicaltrials.gov) - P1 | N=64 | Not yet recruiting | Sponsor: Suzhou Ribo Life Science Co. Ltd.

New P1 trial