- A Phase 2 proof-of-concept, double-blind, placebo-controlled randomized study, MILANO-PILOT, assessing The Medicines Company's (NASDAQ:MDCO) MDCO-216 for reducing atherosclerotic plaque burden failed to achieve the efficacy results necessary for an early end to the trial. In a report, the Independent Data Monitoring Committee recommended the study continue citing the inconclusiveness of the interim data from 40 subjects. Results from the full group of 120 patients will be presented at the American Heart Association Scientific Sessions in mid-November.
- MDCO-216 a complex of a recombinant protein, apolipoprotein A-1 Milano, and phospholipid (POPC). It mimics pre-beta high density lipoprotein (HDL) and induces cholesterol efflux (flowing out), the first step in the removal of deposited cholesterol from the walls of blood vessels (reverse cholesterol transport). The company wants to prove that it improves cardiovascular outcomes.
- Also on tap for the AHA in November is interim data from the Phase 2 ORION-1 study assessing PCSK9si in 501 patients with atherosclerotic cardiovascular disease and elevated LDL-C despite maximum tolerated doses of LDL-C-lowering drugs. The study is comparing the effects of different doses of PCSK9si as well as evaluating the potential for quarterly or bi-annual dosing. The primary endpoint is the percent change in LDL-C from baseline at Day 180.
- PCSK9si, a GalNAc-conjugated RNAi therapeutic that targets PCSK9, is under development for the treatment of high cholesterol (hypercholesterolemia). PCSK9si turns off PCSK9 synthesis in the liver which is different that anti-PCSK9 monoclonal antibodies [Amgen's (AMGN) Repatha (evolocumab) and Sanofi (SNY)/Regeneron's (REGN) Praluent (alirocumab)] which bind to PCSK9 in the blood.
- MDCO is down 8% after hours on light volume.