A Study of STX-1150 in Participants With Elevated Low-Density Lipoprotein Cholesterol (LDL-C)

STX-1150 is an investigational product designed to epigenetically silence the PCSK9 gene. It comprises an mRNA and a guide RNA (gRNA) delivered in a lipid nanoparticle (LNP) for intravenous (IV) infusion.

STX-1150 epigenetically silences the expression of the PCSK9 gene in the liver, thereby reducing circulating PCSK9 and LDL-C levels. The active components, an mRNA and a gRNA are encapsulated in lipid nanoparticles (LNPs) for targeted hepatic delivery. The gRNA precisely guides the complex to a specific locus within the PCSK9 gene promoter.

By reducing PCSK9 expression, STX-1150 prevents the degradation of LDL receptors (LDL-R), leading to increased LDL-R levels on hepatocytes and enhanced clearance of LDL-C from the bloodstream. This targeted and durable epigenetic silencing represents a promising therapeutic strategy for long-term LDL-C reduction, particularly benefiting patients with elevated LDL-C or a high risk for Atherosclerotic Cardiovascular Disease (ASCVD).

Inclusion criteria

• Elevated serum LDL-C with or without LDL-C lowering medication
• Willing and able to give informed consent before initiation of any study-related procedures and willing to comply with all required study procedures

Exclusion criteria

• Patients with history of an ASCVD event </= 6 months.
• Any uncontrolled or serious disease, or any medical or surgical condition that may interfere with participation
• Diagnosis of familial hypercholesterolemia
• Active or history of liver disease
• Previous treatment with PCSK9-inhibitor or other prior treatment within a specified timeframe
• Clinically significant abnormal laboratory values