The Medicines Company and Alnylam Pharmaceuticals have entered an agreement with the US Food and Drug Administration (FDA) for the Phase III clinical programme of inclisiran to treat atherosclerotic cardiovascular disease (ASCVD) and familial hypercholesterolemia (FH).

Inclisiran is an investigational GalNAc-conjugated RNAi therapeutic candidate that targets a genetically validated protein regulator of LDL receptor called PCSK9.

The clinical trials in the Phase III programme are designed to enrol a total of up to 3,000 patients with ASCVD or FH.

Set to be conducted over a period of 18 months, the patients will be administered with a total of four 300mg subcutaneous doses of the drug during the entire treatment period.

All trials will measure the low-density lipoprotein-cholestrol (LDL-C) change from baseline as the primary endpoint.

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By GlobalData

Alnylam chief executive officer John Maraganore said: “The Medicines Company’s agreement with the FDA supports a clear path forward for further inclisiran development and regulatory review.

"Indeed, we’re pleased with the excellent progress made by our colleagues at The Medicines Company to advance this potential, innovative medicine to patients in need, and we intend to fully support them in their efforts.

"The Medicines Company’s agreement with the FDA supports a clear path forward for further inclisiran development and regulatory review."

“Moreover, we believe the planned development path for inclisiran positions this promising potential medicine to contribute meaningfully to our Alnylam 2020 goals.”

The Medicines Company also intends to conduct a trial for cardiovascular outcomes in around 14,000 participants with ASCVD and / or risk equivalents such as diabetes.

The trial's primary endpoint will be a combination of coronary heart disease death, non-fatal myocardial infarction and fatal and non-fatal ischemic stroke.

Based on the trial results, the firms plan to submit a new drug application (NDA) for inclisiran at the end of 2019 for ASCVD and FH.

Image: New Phase III clinical programme for subcutaneous inclisiran. Photo: courtesy of arztsamui at