Skip to main content
Premium Trial:

Request an Annual Quote

Alnylam Presents Positive Phase I Data on Cholesterol Drug Candidate


Alnylam Pharmaceuticals last week released data from a phase I study of its siRNA-based hypercholesterolemia drug ALN-PCS, reporting that a single dose of the agent could significantly and durably lower both its target and low-density lipoprotein cholesterol levels.

In the study, 32 healthy volunteers with elevated LDL were enrolled into six sequential dose cohorts, ranging from 0.015 mg/kg to 0.4 mg/kg. Patients were randomized to receive either drug or placebo.

According to Alnylam, drug treatment led to “rapid, dose-dependent, and durable reductions in LDL-C of up to 50 percent relative to baseline and placebo, with a statistically significant mean reduction of 41 percent at the 0.4 mg/kg dose level.”

Additionally, treatment triggered an up to 84 percent reduction in plasma levels of the drug's target, proprotein convertase subtilisn/kexin type 9, with a mean reduction of 68 percent in the highest dose group. A dose-dependent decrease in the proportion of subjects achieving target levels of LDL-C was also observed.

“The effects of a single dose of ALN-PCS support a once-monthly dose administration regimen for future studies,” Alnylam said.

No serious adverse events related to ALN-PCS were observed, and there was no significant change compared to baseline in levels of high-density lipoprotein.

ALN-PCS comprises siRNAs that are delivered using the so-called MC3 lipid nanoparticle technology, to which both Alnylam and partner Tekmira Pharmaceuticals have laid claim (GSN 3/17/2011).

Earlier this year, after releasing positive preliminary phase I data, Alnylam said that it would not advance ALN-PCS into later-stage clinical testing without a partner (GSN 1/12/2012).

The Scan

More Boosters for US

Following US Food and Drug Administration authorization, the Centers for Disease Control and Prevention has endorsed booster doses of the Moderna and Johnson & Johnson SARS-CoV-2 vaccines, the Washington Post writes.

From a Pig

A genetically modified pig kidney was transplanted into a human without triggering an immune response, Reuters reports.

For Privacy's Sake

Wired reports that more US states are passing genetic privacy laws.

Science Paper on How Poaching Drove Evolution in African Elephants

In Science this week: poaching has led to the rapid evolution of tuskless African elephants.