On April 10, 2024, Ribo and Ribocure announce the completion of enrolment and dosing of the Ph1 clinical trial with world’s first and most advanced anti-thrombotic siRNA drug, RBD4059, targeting coagulation factor XI (FXI).
The Phase I clinical trial is a randomized, single-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics, and potency of RBD4059 in healthy subjects. The study results will be used to inform and progress upcoming Proof of Principle and Proof of Concept studies in target patient populations.
Anticoagulant drugs are cornerstones of the prevention and treatment of thrombosis in many patient populations, including high-risk coronary artery disease, peripheral artery disease, end-stage renal disease (ESRD), atrial fibrillation (AF) and orthopedic surgery, etc. Common and currently used anticoagulants, such as direct oral anticoagulants (DOAC), Vitamin K antagonists (VKAs) and heparin, are all associated with various degree of clinical bleeding risk and skewed benefit-risk that prohibits its wider use, in e.g. frail patients or patients with higher bleeding risk and great unmet need of anti-thrombotic therapy.
RBD4059 is a GalNac-conjugated siRNA drug based on the RIBO-GalSTARTM liver targeting technology platform and achieves its anti-thrombotic effects by suppressing FXI concentrations and inhibiting the intrinsic pathway of the coagulation cascade. People with naturally occurring low levels of FXI are at lower risk of having thrombosis without signs of increased bleeding. Therefore, inhibiting FXI holds the promise to be an efficacious antithrombotic therapy with minimal bleeding risk compared to current available therapies.
RBD4059 demonstrated good tolerability, potency and durability during the Ph1 study, and trial data will be shared at upcoming major scientific meetings. Based on the safety and efficacy data observed in Ph1, a Ph2 CTA application has been submitted to EMA, aiming for proof of principle and dose confirmation in a target patient population.
