Santaris Pharma, a clinical-stage biopharmaceutical company focused on the research and development of mRNA and microRNA targeted therapies, has advanced SPC5001 into phase 1 clinical trials for the treatment of high cholesterol. SPC5001 is a mRNA-targeted drug that inhibits the exciting new target, Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9), a protein involved in removing low density lipoprotein cholesterol (LDL-C) from the bloodstream.

The randomized, dose-escalation, double-blind, placebo-controlled phase I study will assess safety, tolerability, pharmacokinetics, and pharmacodynamics of SPC5001. The study aims to enroll 32 healthy volunteers and 8 patients with familial hypercholesterolemia who will be randomized to receive weekly subcutaneous injections of SPC5001 or placebo. Santaris Pharma A/S aims to first use SPC5001 in patients with familial hypercholesterolemia, a genetic disorder characterized by high cholesterol levels, specifically very high levels of LDL-C in the blood and often leads to early cardiovascular disease.

In preclinical studies, SPC5001 provided fast-acting, potent and long-lasting inhibition of PCSK9 and provided reductions of mean LDL cholesterol by 50% in non-human primates with a sustained reduction of 74% in the highest responder. SPC5001 did not change HDL (high-density lipoprotein) levels in the blood.