On October 28, 2024, Ribocure Pharmaceuticals AB and Suzhou Ribo Life Science Ltd (Ribo) received authorization from the Swedish Medicinal Product Agency (MPA), to initiate a Phase II clinical trial in Sweden with the lipid-lowering siRNA drug RBD5044 that targets APOC3. The trial will evaluate efficacy and safety in patients with mixed dyslipidemia.
A randomized, double-blind, placebo-controlled, multi-center phase II trial evaluating the efficacy and safety of RBD5044 in participants with mixed dyslipidemia will be run. The trial sites include Ribocure Clinic, a specialized phase II unit for high quality functional imaging proof of principle trials, based in GoCo Health Innovation City, Gothenburg, as well as sites in Lund and Uppsala, Sweden.
RBD5044 is a GalNAc-conjugated small interfering RNA (siRNA) drug based on the RIBO-GalSTARTM liver targeting technology platform. The inhibition of APOC3 by siRNA-mediated knockdown is a highly potent, high precision and long-lasting mechanism to reduce plasma levels of atherogenic ApoC-III containing triglyceride-rich particles.
Plasma ApoC3 levels predict coronary events independently of LDL cholesterol values. In addition, genetic studies demonstrate that APOC3 Loss-Of-Function mutations are associated with lower plasma triglycerides and about a 40% lower risk of cardiovascular disease. Thereby, RBD5044 has the potential to reduce residual cardiovascular disease by targeting a lipid-related risk that is not amenable to current lipid-lowering therapies. In patients with severe hypertriglyceridemia, the inhibition of ApoC-III and lowering of plasma triglyceride levels might also reduce recurrent pancreatitis events.
RBD5044 is the second siRNA targeting APOC3 world-wide to enter clinical stage. Based on Ph1 safety and efficacy data, we are optimistic about the potential of RBD5044 to be best-in-class in treating mixed dyslipidemia and isolated hypertriglyceridemia. “Hypertriglyceridemia, both in fasting and postprandial states, has been shown to be closely linked to residual cardiovascular risk and currently lacks an effective therapeutic option. We are committed to accelerating the development of RBD5044 to address this significant unmet clinical need. The approval marks our fourth clinical trial authorization in 2024, a milestone that reflects our dedication to advancing oligonucleotide therapeutics. I am incredibly proud of our team, which has consistently shown its ability to not only develop efficacious and safe siRNAs, but also navigating the regulatory landscapes with speed and precision”, says Li-Ming Gan, CEO of Ribocure.