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Alnylam Files to Begin Human Trial of Complement-mediated Disease Drug

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NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals announced that it has filed an application with UK regulators to begin a Phase I/II trial of its complement-mediated disease treatment ALN-CC5.

The complement system is involved in immunity as a protective mechanism for host defense, and dysregulation of the system can create serious complications in a variety of diseases, according to the company.

ALN-CC5 is designed to silence complement component C5, which is primarily expressed in liver cells and whose loss is associated with an attenuated immune response against certain infections. It uses Alnylam's second-generation GalNAc delivery conjugate technology, which enables subcutaneous administration.

Alnylam's planned Phase I/II study would begin by testing the drug in up to 60 healthy volunteers, then in up to eight patients with paroxysmal nocturnal hemoglobinuria. The first part of the study is designed to evaluate safety and tolerability of single and multiple doses of ALN-CC5. The second part will assess, safety, tolerability, pharmacokinetics and pharmacodynamics, and clinical activity, as well as the exploratory evaluation of the drug's effect on lactate dehydrogenase, a measure of red blood cell hemolysis.

Pending approval, the trial is expected to begin in mid-2015.

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