Santaris Pharma has advanced a new discovery research candidate, SPC5001, which will attack PCSK9, an important new target recently identified for the treatment of high cholesterol.

PCSK9 is a protein that is involved in regulating LDL, which is a major risk factor for coronary heart disease, heart attack and stroke.

Preclinical data presented at a recent PCSK9 conference showed that SPC5001 provided the potent, specific and long-lasting inhibition of PCSK9, and lowered LDL cholesterol in primates by up to 74%.

Santaris Pharma vice-president and chief scientific officer Henrik Orum said that there is a lot of interest in PCSK9 as an exciting new target for the treatment of high cholesterol.

“Having demonstrated that SPC5001 effectively lowers LDL or the ‘bad’ cholesterol in preclinical studies, we are quickly moving forward with plans to advance SPC5001 into clinical studies,” Orum said.

The drug has been developed using Santaris’s LNA Drug Platform and Drug Discovery Engine, which combines the company’s proprietary LNA chemistry with specialised drug development capabilities to rapidly deliver potent single-stranded LNA-based drug candidates for a range of diseases.