The Medicines Company announced the discontinuation of the clinical development program for MDCO-216, its investigational cholesterol efflux promoter.

MDCO-216 is a complex of dimeric recombinant apolipoprotein A-1 Milano (APoA-1 Milano) and a phospholipid (POPC) being developed to improve cardiovascular outcomes by reducing plaque burden in patients with atherosclerotic disease. It works by mimicking pre-beta HDL and inducing cholesterol efflux. 

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The decision for discontinuation was based on data from the recent MILANO-PILOT trial which did not demonstrate drug effects on intracoronary atherosclerotic plaque sufficient to support further development; its safety profile, however, was considered excellent. Also, the decision to discontinue further investment in the clinical development of MDCO-216 was based on the highly positive results reported from the ORION-1 trial evaluating the company’s PCSK9 synthesis inhibitor. 

The ORION-1 (n=501), a placebo-controlled, double-blind, randomized Phase 2 study, showed significant and durable LDL-C reduction, further supporting a triannual (and potentially biannual) dosing regimen with high standards of safety and and tolerability. 

The investment in developing MDCO-216 was through a clinical proof-of-concept. “Unfortunately, the efficacy data from MILANO-PILOT do not support a prudent decision to make the significant, near-term investment required to move MDCO-216 forward,” stated Clive Meanwell, MD, PhD, Chief Executive Officer of The Medicines Company. 

The Medicines Company is supporting the close-out of the MILANO-PILOT trial and its results will be presented at the American Heart Association (AHA) Scientific Sessions.  

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