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biocqr

05/09/15 5:17 PM

#191070 RE: DewDiligence #190929

ALNY, GEVA, ALXN >Fear of a 'Soliris Killer' Might Have Driven Alexion to Overpay for Synageva

http://www.thestreet.com/story/13142079/1/fear-of-a-soliris-killer-might-have-driven-alexion-to-overpay-for-synageva.html

Company executives are worried about a third company and a competitive threat to its Soliris ultra-orphan blockbuster franchise. Investors see Alexion's willingness to write a huge check for Synageva as a tacit acknowledgment that Soliris' long-term future might be more at risk than the company lets on.

That third company is Alnylam Pharmaceuticals (ALNY - Get Report), the maker of drugs based on gene silencing technology known as RNA interference. Alnylam is developing a drug which if successful would compete directly against Soliris as a treatment for rare, inherited blood disorders.

PNH and related diseases are caused by an over-activation of complement, a protein involved with the body's immune system. Soliris is a monoclonal antibody which targets and stops a specific type of complement known as complement component C5 from damaging blood cells. In order to have a therapeutic effect, large amounts of Soliris must be given to patients via an intravenous infusion every two weeks.

Alnylam's ALN-CC5 is an RNAi molecule designed to turn off the gene responsible for making disease-causing complement component C5. ALN-CC5 is administered by an injection just under the skin, and while dosing is still being worked out, Alnylam hopes the RNAi therapeutic can be given every two weeks or perhaps even once per month. Promising results from a primate study presented in 2013 demonstrated ALN-CC5 was capable of decreasing levels of complement component C5.

anotherbiotechguy

05/11/15 10:21 PM

#191135 RE: DewDiligence #190929

This makes me sick for some very personal reasons. Coulda woulda shoulda from a past life.