Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, and The Medicines Company (Nasdaq:MDCO), a leading biopharmaceutical development and cardiovascular product company, today announced that results from the Phase 1 study of inclisiran (in-CLEE-si-ran), the recommended International Nonproprietary Name (INN) for ALN-PCSsc, were published in The New England Journal of Medicine (NEJM). Inclisiran is an investigational RNAi therapeutic targeting PCSK9 - a genetically validated protein regulator of LDL receptor metabolism - being developed for the treatment of hypercholesterolemia. The paper can be found online here.

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Results from the study showed doses =300 mg (single or multiple doses) significantly reduced PCSK9 and LDL cholesterol for at least 6 months. Moreover, inclisiran was found to be generally well tolerated.

"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," said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President of R&D and Chief Medical Officer of Alnylam. "We believe that inclisiran represents an innovative and differentiated approach for the treatment of hypercholesterolemia."

Inclisiran is currently being studied in the ORION-1 Phase 2 study by The Medicines Company. With more than 500 patients enrolled, ORION-1 is the largest study of a GalNAc-siRNA conjugate to date. The Medicines Company recently announced positive top-line results from the day 90 interim analysis of the Phase 2 study demonstrating significant and durable LDL-C reduction that validates the potential for a triannual or biannual dosing regimen. Top-line results also showed that inclisiran was generally well tolerated, with no evidence of drug-related elevations of liver enzymes, neuropathy adverse events, or changes in renal function. The Medicines Company plans to present complete interim results from the ongoing study in a Late-Breaking Clinical Trial Session at the American Heart Association (AHA) Scientific Sessions on November 15, 2016.

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