Sirnaomics Ltd. (the “Company”, Stock Code: 2257.HK, together with its subsidiaries, the “Group” or “Sirnaomics”), a leading biopharmaceutical company engaging in discovery and development of advanced RNAi therapeutics, announced today that the Group has completed IND-enabling studies for STP125G, an siRNA therapeutics targeting Apolipoprotein C3 (ApoC3), based on its proprietary GalAheadTM mxRNA technology. The safety and efficacy results from the non-human primate (NHP) studies strongly support for an IND filing with the U.S. FDA for initiating a Phase I clinical study of STP125G for cardiovascular disease indications.

ApoC3 is a widely known player in triglyceride metabolism, and it has been recently recognized as a polyhedric factor which may regulate several pathways beyond lipid metabolism by influencing cardiovascular, metabolic, and neurological disease risk. High levels of triglycerides (TG) have been shown to be associated with increased risk of cardiovascular diseases. For severe hypertriglyceridemia (sHTG) patients whose TG level is more than 1000 mg/dL, the risk of developing acute pancreatitis is 5 to 10 times to that in the general population. Down-regulation of ApoC3 using siRNA or antisense oligonucleotides has been shown to be effective in lowering TG in sHTG patients.

During an efficacy evaluation of STP125G with non-human primate model (N = 4), we observed a dose-dependent silencing activity among 1 mg/kg, 3 mg/kg and 10 mg/kg doses with a strong safety profile. The maximum target silencing efficacy was achieved at 10 mg/ kg dosage around week 4 and was maintained for an additional 9 weeks (the total length of this 13-week study). The safety evaluation of STP125G using non-human primate model (N = 4) demonstrated an excellent safety readout with a single subcutaneous administration at 50 mg/kg, 100 mg/kg or 250 mg/kg. The maximum target silencing efficacies were like the level of 10 mg/kg for all three high dosages.

“STP125G is the second drug candidate based on our GalAheadTM mxRNA technology that has shown excellent safety and potent efficacy results with the NHP models. Its long-lasting silencing activity against ApoC3 may provide better therapeutic benefit to patients suffering cardiovascular conditions, than those of antisense and other siRNA drugs.” Dr. Patrick Lu, Founder, Chairman of the Board, Executive Director, President and Chief Executive Officer of Sirnaomics, indicated. “Those data readouts further validated STP125G as a novel siRNA therapeutic candidate for treatment of hypertriglyceridemia and other cardiovascular diseases, using our proprietary GalAheadTM-based delivery technology”