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Alnylam shares rally on phase 1 cholesterol-lowering drug results

Published: 14:14 20 Apr 2012 EDT

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Alnylam Pharmaceuticals (NASDAQ:ALNY) reported Friday positive results from its phase I clinical trial of ALN-PCS, a drug to treat severe hypercholesterolemia.

Hypercholesterolemia is the presence of high levels of cholesterol in the blood. High cholesterol levels can increase a person’s chance of getting heart disease, stroke, and other problems.

The drug ALN-PCS acts as an inhibitor that lowers bad cholesterol, also known as LDL-C. The new data was presented at a conference held by the American Heart Association in Chicago.

According to the results, a single dose of the medicine resulted in a "statistically significant" reduction of the protein PCSKP9, which regulates the LDL receptor, and lowered LDL-C by up to 50 percent.

Results also highlighted continued improved efficacy and tolerability for Alnylam’s second-generation lipid nano-particle delivery technology, the company said.

There was no major change compared to baseline in levels of high-density lipoprotein, or good cholesterol, it added.

"These new results show very robust, statistically significant, and dose-dependent lowering of both PCSK9 and LDL-C levels in a single dose study performed in the absence of statin co-administration," said senior vice president, Akshay K. Vaishnaw, in a statement.

The drug was shown to be safe and well tolerated. There were no serious adverse events related to the study drug administration, the company said.

The phase I randomized and placebo-controlled study was carried out on 32 healthy volunteer patients with an elevated baseline of bad cholesterol greater than 116-miligrams. The subjects were enrolled into six sequential dose cohorts ranging from 0.015 to 0.400-mg in a 3:1 ratio to placebo.

Shares of the company rose nearly six percent reaching $10.60 apiece in trade on the Nasdaq on Friday afternoon.

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