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ALNY > Here’s why data this weekend from Alnylam's blood disease drugs could be a big deal

http://www.bizjournals.com/boston/blog/bioflash/2015/12/here-s-why-data-this-weekend-from-alnylams-blood.html

The trial data from Alnylam’s hemophilia drug will update early results (presented in June) from the same Phase 1 trial from the first 12 patients. While the company isn’t saying how many patients will be included in the updated data, it will clearly be more, potentially validating the results seen in June.

According to Vaishnaw, there will be three main measurements from the ALN-AT3 trial presented mid-day on Monday, Dec. 7: the ability of the drug to reduce levels of anti-thrombin, the resulting increase in levels of thrombin, and the frequency of severe bleeding episodes. The data in June showed that ALN-AT3 reduced anti-thrombin by “up to 86 percent,” with effects lasting more than two months. There was also an average increase of “up to 350 percent” in thrombin generation, and evidence of reduced bleeding frequency.

As for ALN-CC5, the drug to treat so-called complement-mediated diseases, Alnylam’s Phase 1 trial is in healthy volunteers. The drug is intended to reduce levels of a protein called C5 which is made in the liver and is a key component in the chain of events in which red blood cells self-destruct in patients with such diseases. Here the company is measuring three things: reduction in C5, the amount of residual C5 and how that reduction inhibits activity that leads to hemolysis (red blood cell destruction).

Trial data on ALN-CC5 will be revealed Sunday evening, Dec. 6, and will update date from the same trial presented in June based on the first 12 patients. That study showed a single dose of ALN-CC5 reduced serum C5 up to 96 percent, and blocked so-called “serum complement activity” by to 92 percent.