Phase 1 inclisiran trial results published in New England Journal of Medicine

The findings add to the clinical evidence supporting PCSK9 as a therapeutic target for significantly lowering LDL cholesterol.
The findings add to the clinical evidence supporting PCSK9 as a therapeutic target for significantly lowering LDL cholesterol. | File photo
Alnylam Pharmaceuticals published results from its Phase 1 clinical trial involving inclisiran in The New England Journal of Medicine.
"The published findings from our Phase 1 trial with inclisiran add to the clinical evidence supporting PCSK9 as a therapeutic target for significantly lowering LDL cholesterol, as well as the ability of RNAi therapeutic candidates to inhibit synthesis of liver-derived target proteins in a potent and durable manner,” Alnylam Executive Vice President of Research and Development Dr. Akshay Vaishnaw said. “We believe that inclisiran represents an innovative and differentiated approach for the treatment of hypercholesterolemia.”
Results show that 300mg doses reduce PCSK9.
“We look forward to presenting the interim results of the ORION-1 Phase 2 data at AHA this week including Day 90 follow-up results for all 501 patients and a preliminary analysis of Day 180 follow-up for up to 200 patients,” The Medicines Co. Vice President David Kallend said. “Based on the strong results from the Phase 1 study, we hope to further elucidate the hypothesis of triannual or biannual dosing of inclisiran with the ORION-1 results.”