Alexion Pharmaceuticals (ALXN) recently presented new data on patients with atypical hemolytic uremic syndrome (aHUS).

The retrospective analysis conducted on 30 patients showed that most patients suffered severe complications of the disease across multiple organs in spite of getting supportive care including plasma infusion. The data laid emphasis on the risk of sudden and potentially fatal systemic complications associated with aHUS.

Another analysis, conducted with 19 pediatric subjects with aHUS, revealed that patients treated with Soliris experienced a reduction in thrombotic microangiopathy (TMA). This resulted in an improvement in kidney function thereby reducing the use of dialysis in patients who required it earlier.

Alexion also presented data indicating the need for testing patients at high risk for paroxysmal nocturnal hemoglobinuria (PNH). Alexion presented these data at the European Hematology Association (EHA).

Soliris, Alexion’s only marketed product, is available for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare genetic blood disorder and atypical hemolytic uremic syndrome (aHUS), an ultra-rare genetic disorder. The company intends to start launching Soliris in European countries for the aHUS indication this year. Alexion reported Soliris revenues of $244.7 million at the first quarter earnings of 2012, an increase of 47% from the prior year quarter.

Alexion is also working on several other indications of the drug including acute humoral rejection (AHR). Alexion is studying Soliris in patients undergoing kidney transplantation, having a high risk of organ rejection. The company also expects to commence a deceased donor study this summer.

Alexion is also running a phase II trial to evaluate patients with Shiga-toxin producing E. coli hemolytic uremic syndrome (STEC-HUS) in Germany. The company expects results from this study by the end of 2012.

Soliris is also being studied in investigator-initiated studies for dry form of age related macular degeneration (AMD) and neuromyelitis optica (NMO). Results from phase II investigator-initiated trials of NMO are expected to be presented by the second half of this year at a scientific meeting.

Currently, we have a Neutral recommendation on Alexion. The stock carries a Zacks #3 Rank (Hold rating) in the short run.

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