Phase III trial of PCSK9si meets all endpoints in CV disease and elevated LDL-cholesterol

The Medicines Company announced positive topline results from the first pivotal Phase III clinical trial evaluating the efficacy, safety, and tolerability of PCSK9si (inclisiran) to decrease LDL-cholesterol (LDL-C) in patients with atherosclerotic cardiovascular disease through twice-yearly dosing. The ORION-11 study of inclisiran sodium 300 mg met all primary and secondary endpoints with efficacy consistent with findings from Phase I and II studies. The trial showed that inclisiran was well tolerated and confirmed that the safety profile was at least as favorable as that demonstrated in the ORION-1 Phase II and ORION-3 open label extension studies. Detailed efficacy, tolerability and safety data from ORION-11 will be presented during a late-breaking science session at the European Society of Cardiology’s ESC Congress 2019.

Comment: The sequential release of topline Phase III data readouts for the ORION-9 and ORION-10 studies are expected to continue later in the third quarter in advance of anticipated regulatory submissions in the U.S. in the fourth quarter of 2019 and in Europe in the first quarter of 2020. Patients who have completed their respective Phase III studies are now enrolling into ORION-8, an open-label, long-term extension study where patients completing ORION-9, ORION-10 and ORION-11 will receive inclisiran for three years to evaluate the efficacy, safety and tolerability of long-term dosing of inclisiran.