Archemix: Initiates Phase II ARC1779 Study

CLSDF continues to cover Cambridge-based Archemix; find out why (here) and (here). The aptamer company announced initiation of a Phase II study of ARC1779. The trial is designed to evaluate the safety and efficacy of ARC1779 as a first-in-class anti-platelet agent in patients suffering from a rare, life-threatening blood disorder known as thrombotic thrombocytopenic purpura (TTP).

TTP is a rare blood disorder that arises from uncontrolled activation of von Willebrand Factor (vWF), a protein which under normal conditions plays a key role in maintaining the normal balance between bleeding and clotting. In patients suffering from TTP, vWF binds excessively to platelets, causing excessive blood clots. These clots form throughout the circulation and can lead to serious medical consequences such as strokes, seizures, kidney failure and heart attack. Each year in the United States, between four and eleven new cases of TTP per million of the total population are diagnosed. There is no approved drug treatment for patients with TTP.

Aptamers are synthetically-derived oligonucleotides that bind to protein targets with high affinity and specificity and can be designed to have a specified duration of action. Aptamers represent an emerging class of potential therapeutic agents that Archemix believes may have broad application to treat a variety of human diseases. ARC1779, a pegylated aptamer consisting of 40 nucleotides, is Archemix’ lead product candidate for the treatment of TTP. ARC1779 is designed to inhibit the function of vWF, which is, when activated, responsible for the adhesion, activation and aggregation of platelets.

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