2024-10-29
Recently, BOCG's portfolio company, Ribo Life Science, announces that it 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 following news is from Ribo Life Science:
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.
About Ribo Life Science
Ribo is a world-leading clinical stage company devoted to the development of innovative RNAi-based therapeutics to address unmet clinical needs in the world. As a Nobel Prize winning discovery, RNAi (or RNA interference) is a natural mechanism within human cells that can control the production of proteins by specifically silencing its mRNA. With a cross-continent talent team and the fully integrated cutting edge RNAi technology platforms, Ribo has built an extensive pipeline of innovative RNAi-based medicines for treating liver diseases, cardio-metabolic diseases and other disorders.
About Ribocure
Ribocure Pharmaceuticals AB is a clinical stage biotech company with a business focus on the development of cutting-edge RNA therapeutics.
Ribocure’s primary focus lies in addressing the significant medical gaps in rare diseases involved in the fields of cardiovascular, renal, and oncology. The company is dedicated to the discovery and development of groundbreaking drug assets utilizing advanced oligonucleotide technologies.
Operating as a subsidiary of Ribo Life Science Co., Ltd., Ribocure leverages the advantages of a vertically integrated oligonucleotide research platform and excels in translational and clinical operations. Our comprehensive technological platforms encompass the entire process, from sequence design, screening, and optimization to delivery technologies and large-scale commercial CMC capacity.
In 2022, we successfully progressed three projects to the clinical stage, achieving an impressive average time frame of 20-24 months from concept to implementation.
