On September 10, 2024, Ribocure Pharmaceuticals AB and Suzhou Ribo Life Science Ltd (Ribo) received authorization from the Swedish Medicinal Product Agency (MPA), to initiate a first-in-human (FIH) Phase I clinical trial in Sweden with the siRNA drug, RBD7007 targeting C5.
This is a first-in-human (FIH) phase 1 trial to assess the safety, tolerability, and dose dependent pharmacokinetic (PK) and pharmacodynamic (PD) effects of RBD7007 in healthy participants. The clinical trial aims to inform the asset’s further development for treatment of complement-mediated diseases, where inhibition of complement factor 5 (C5) can provide a therapeutic benefit. The trial will include both single and multiple ascending dose regimens and findings from this trial will be used to determine dosage and administration protocols for future Phase 2 clinical trials.
RBD7007 is a N-acetylgalactosamine (GalNAc)-conjugated siRNA targeting the C5 mRNA in the liver. Preclinical studies have demonstrated that RBD7007 provides strong, long-lasting inhibition of complement pathway activation, along with an excellent safety profile, positioning it as a promising candidate for the treatment of complement-mediated diseases.
C5, a critical component of the complement system, plays a central role in innate immunity, helping to protect the body and regulate adaptive immune responses. The abnormal activation of C5 has been implicated in the pathogenesis of several diseases, like the autoimmune disorders Paroxysmal Nocturnal Hemoglobinuria (PNH), IgA nephropathy, and Myasthenia Gravis (MG). Existing antibody therapies that target C5 face limitations such as short half-lives and reduced duration of action.
Thus, there is a significant unmet clinical need for more potent, long acting, and safe therapeutic options. RBD7007 aims to address this need, offering significant benefits to patients affected by complement-mediated diseases.
About Suzhou Ribo Life Science Co. Ltd. and Ribocure Pharmaceuticals AB
Ribocure Pharmaceuticals AB is a Sweden-based, clinical stage biotech company and the international R&D Center of Suzhou Ribo Life Science Ltd., with focus on development of life-saving oligonucleotide therapies. Ribocure is located at GoCo Health Innovation City in Mölndal with its own Phase 2 Clinical Trial Unit (CTU) and an expertise in cardiovascular, pulmonary, renal and liver diseases, where advanced imaging and functional assessments are applied.
For more information, please visit www.ribolia.com and www.ribocure.com
