Date: 2011-05-04
Type of information:
phase: 1
Announcement: initiation
Company: Santaris Pharma (Denmark)
Product: SPC5001
Action mechanism: SPC5001 is a RNA-targeted drug inhibiting PCSK9, which is a protein involved in removing low-density lipoprotein cholesterol (LDL-C) or “bad” cholesterol from the bloodstream
Disease: high cholesterol
Therapeutic area: Metabolic diseases - Cardiovascular diseases
Country:
Trial details: The randomized, dose-escalation, double-blind, placebo-controlled Phase 1 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.
Latest
news: Santaris Pharma has advanced SPC5001 into Phase 1 clinical trials for the treatment of high cholesterol. SPC5001 is a mRNA-targeted drug that inhibits a new target, Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9), a protein involved in removing low density lipoprotein cholesterol (LDL-C) or “bad” cholesterol from the bloodstream. Santaris Pharma also plans to advance SPC4955 inhibiting apolipoprotein B (apoB) for the treatment of high cholesterol in the first half of 2011. In yet a third approach, in February 2011 Santaris Pharma obtained an exclusive license from Massachusetts General Hospital for intellectual property related to the regulation of miR-33, an important microRNAthat regulates high density lipoprotein (HDL) levels or “good” cholesterol.
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 or the “good” cholesterol in the blood.
