Pfeiffer Palm Teller Syndrome

HONG KONG, GERMANTOWN, Md. and SUZHOU, China, July 12, 2024 /PRNewswire/ -- 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 GalAhead™ 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 GalAhead™ 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 GalAhead™-based delivery technology". - End - About ApoC3 and STP125G ApoC3 is an important emerging target linking hypertriglyceridemia with cardiovascular disease (CVD). ApoC3 is a potent modulator of many established CVD risk factors, and is found on chylomicrons, VLDL, LDL, and HDL particles. Many studies show that in humans, apoC3 levels are an independent risk factor for CVD, and its presence on lipoproteins may promote their atherogenicity. Recent findings of the role of ApoC3 has been implicated in HDL metabolism and in the development of atherosclerosis, inflammation, and ER stress in endothelial cells. ApoC3 has been recently considered an important player in insulin resistance mechanisms, lipodystrophy, diabetic dyslipidemia, and postprandial hypertriglyceridemia (PPT). The emerging evidence of the involvement of ApoC3 in the pathogenesis of Alzheimer's disease open the way to further study if modification of ApoC3 level slows disease progression. Furthermore, ApoC3 is clearly linked to cardiovascular disease (CVD) risk, and progression of coronary artery disease (CAD) as well as the calcification of aortic valve and recent clinical trials has pointed out the inhibition of ApoC3 as a promising approach to manage hypertriglyceridemia and prevent CVD. Several evidences highlight the role of ApoC3 not only in triglyceride metabolism but also in several cardio-metabolic pathways. STP125G is a single-stranded siRNA therapeutics targeting ApoC3 mRNA, based on Sirnaomics proprietary GalAhead™ mxRNA technology. About Sirnaomics Sirnaomics is an RNA therapeutics biopharmaceutical company that focuses on the discovery and development of innovative drugs for indications with unmet medical needs and large market opportunities. Sirnaomics is the first clinical-stage RNA therapeutics company to have a strong presence in both Asia and the United States. Based on its proprietary delivery technologies, a polypeptide nanoparticle RNAi platform and GalNAc RNAi platform, GalAhead™, Sirnaomics has established an enriched drug candidate pipeline. STP122G, which represents the first drug candidate utilizing the Group's GalAhead™ mxRNA technology, is currently in Phase I development. STP125G is the second siRNA therapeutics based on Sirnaomics proprietary GalAhead™ mxRNA technology, targeting ApoC3 mRNA for cardiovascular disease treatment. STP237G is the first dual-targeted drug based on a GalAhead™ muRNA technology and is in the late stage of preclinical evaluation. The Group has also had multiple successes with oncology applications through its clinical programs for STP705 and STP707. With the expansion of the Group's clinical pipeline and establishment of the Group's manufacturing facility, Sirnaomics focuses on a transition from a biotech company to a biopharma corporation. Learn more at: . SOURCE Sirnaomics