The Medicines Company (NASDAQ:MDCO) is up 4% premarket on light volume in response to its announcement that the FDA has agreed to a Phase 3 program for cholesterol med inclisiran that will support a New Drug Application (NDA).
The late-stage program will include clinical trials in patients with atherosclerotic cardiovascular disease (ASCVD) and familial hypercholesterolemia (FH) and will collectively enroll ~3,000 subjects. The primary endpoint will be the change in LDL-C (bad cholesterol) from baseline over an 18-month period.
Separate from the registration studies, the company will also conduct a large-scale (n=14,000) cardiovascular outcomes study in patients with ASCVD or other risk factors (e.g., diabetes) to determine the effects of lowering LDL-C with inclisiran on cardiovascular outcomes. The primary endpoint will be a composite of coronary heart disease death, non-fatal myocardial infarction and fatal/non-fatal ischemic stroke.
The company says it expects to submit its NDA for ASCVD and FH at the end of 2019.
Inclisiran (PCSK9si) is an investigational GalNAc-conjugated RNAi therapeutic targeting PCSK9, a protein regulator of LDL receptor metabolism. In contrast to PCSK9 inhibitors (like Amgen's Repatha and Regeneron and Sanofi's Praluent), PCSK9si works by turning off PCSK9 synthesis in the liver. MDCO is collaborating with Alnylam (NASDAQ:ALNY) on its development under their 2013 agreement.