Patient enrolment has been completed in The Medicine Company’s ORION-1 study of PCSK9si, its investigational RNA interference (RNAi) proprotein convertase subtilisin/kexin type 9 synthesis inhibitor (PCSK9si).
The study—which started enrolling in January 2016—met its target of enrolling 480 patients with high cardiovascular risk and elevated LDL-C ahead of schedule. Follow-up of these patients will track the effects of different dosages of PCSK9si on LDL-C.
A previous study showed that a single dose of PCSK9si lowered LDL-C significantly for six months – supporting the potential for subcutaneous dosing as infrequently as twice a year. According to a company release, experts believe that such a dosing schedule could significantly improve patient adherence to treatment.
Interim three-month and six-month effectiveness and safety data from patients in ORION-1 are expected to be available, analysed and presented by the end of 2016.
“We and our partners at Alnylam are pleased to have completed enrolment of ORION-1. We believe that PCSK9si has significant potential as compared with anti-PCSK9 monoclonal antibodies (MAbs), a recently approved class of LDL-C lowering drugs,” says David Kallend, vice president and Global Medical director, The Medicines Company. “Assuming positive data from ORION-1, we are on track to initiate Phase 3 registration studies in early 2017. In addition, we plan on conducting studies directly comparing PCSK9si with anti-PCSK9 MAbs as well as studies in homozygous familial hypercholesterolemia starting this year.”