Esperion Therapeutics update on Phase III programme for ETC-1002 (bempedoic acid) to treat familial hypercholesterolemia and atherosclerotic cardiovascular disease

Esperion Therapeutics Inc. remains on track to initiate the ETC-1002 (bempedoic acid) Phase III development programme by the end of 2015. The programme will support a proposed label indication for the use of ETC-1002, an oral, once-daily LDL-C lowering therapy, "as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH), or atherosclerotic cardiovascular disease (ASCVD), who require additional lowering of LDL-C." Approximately nine million patients in the USA fall into these two combined categories. The ETC-1002 Phase III development programme will include clinical studies in patients with ASCVD and HeFH, and the study designs will be finalised once minutes from the FDA meeting are received. In the fourth quarter, Esperion plans to finalise development plans for the fixed-dose combination of ETC-1002 and ezetimibe and will file a separate Investigational New Drug (IND) application with the FDA.

Comment: ETC-1002 targets ATP citrate lyase, an enzyme on the same cholesterol biosynthesis pathway as the enzyme targeted by statins. The drug does not reduce LDL-C as much as the PCSK9 inhibitors, but it does have the benefit of being an oral therapy.