Complement Inhibition
The complement inhibition technology of Soliris is a groundbreaking therapeutic
approach that has the potential to treat patients with a variety of diseases that
involve complement activation. The inhibition of complement in animal models has
been shown to reduce laboratory measures of kidney disease,6 asthma,7
transplantation,8,9 multifocal motor neuropathy,10,11 myasthenia
gravis,12 lupus,6 and arthritis.13
The complement protein known as C5 acts at a relatively late stage in the complement
cascade and when activated, is directly involved in activating host cells, attracting
immune cells that can cause inflammation, and destroying cells by triggering pore
formation. If C5 activity is not regulated properly, the resulting complement activity
may lead to one of many human disorders.
From the Company's inception, researchers at Alexion have believed that the best
place to block the complement cascade is at complement protein C5, so that the normal
disease-preventing functions of complement largely remain intact, while the activity
of C5 that leads to inflammation and cell destruction is blocked. Alexion's fully
humanized monoclonal antibody, Soliris, is the first complement inhibitor approved
in the United States, European Union, Canada and Australia for the treatment of
patients with PNH. Soliris protects PNH red blood cells from destruction by complement.
Soliris has received two of the industry’s highest honors for innovation: the 2008
Prix Galien USA Award for Best Biotechnology Agent and the 2009 Prix Galien France
Award in the category of medicines for rare diseases.
The Company and independent researchers are investigating the potential role of
Soliris in the treatment of
other diseases where uncontrolled complement activity leads to poor outcomes
for patients.