Skip to main content
Premium Trial:

Request an Annual Quote

Alnylam Doses First Patients in Cholesterol Drug Trial

Premium

Alnylam Pharmaceuticals said this week that it has begun dosing patients in a previously announced phase I study of its siRNA-based hypercholesterolemia drug ALN-PCS.

ALN-PCS comprises siRNAs against proprotein convertase subtilisn/kexin type 9, which has been shown to regulate low-density lipoprotein receptor levels and play a role in plasma LDL clearance. It incorporates a second-generation lipid nanoparticle technology called MC3.

The phase I study is being conducted in the UK and is designed to test single, escalating doses of the drug in up to 32 healthy volunteers with elevated baseline low-density lipoprotein levels.

“The primary objective of the study is to evaluate the safety and tolerability of a single dose of ALN-PCS, with patients being enrolled into five sequential cohorts of increasing doses ranging from 0.015 to 0.25 mg/kg,” Alnylam said. “Secondary objectives include characterization of plasma and urine pharmacokinetics of ALN-PCS, and assessment of pharmacodynamic effects of the drug on plasma PCSK9 protein and LDLc levels measured from serial blood samples prior to and following dosing.”

The Scan

Support for Moderna Booster

An FDA advisory committee supports authorizing a booster for Moderna's SARS-CoV-2 vaccine, CNN reports.

Testing at UK Lab Suspended

SARS-CoV-2 testing at a UK lab has been suspended following a number of false negative results.

J&J CSO to Step Down

The Wall Street Journal reports that Paul Stoffels will be stepping down as chief scientific officer at Johnson & Johnson by the end of the year.

Science Papers Present Proteo-Genomic Map of Human Health, Brain Tumor Target, Tool to Infer CNVs

In Science this week: gene-protein-disease map, epigenomic and transcriptomic approach highlights potential therapeutic target for gliomas, and more