Skip to main content

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

Call to Look Again

More than a dozen researchers penned a letter in Science saying a previous investigation into the origin of SARS-CoV-2 did not give theories equal consideration.

Not Always Trusted

In a new poll, slightly more than half of US adults have a great deal or quite a lot of trust in the Centers for Disease Control and Prevention, the Hill reports.

Identified Decades Later

A genetic genealogy approach has identified "Christy Crystal Creek," the New York Times reports.

Science Papers Report on Splicing Enhancer, Point of Care Test for Sexual Transmitted Disease

In Science this week: a novel RNA structural element that acts as a splicing enhancer, and more.