Breakthrough designation for Alexion's ALXN1101

Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) said FDA granted breakthrough therapy designation for ALXN1101 to treat molybdenum cofactor deficiency (MoCD) Type A, a rare genetic disorder characterized by severe brain damage and rapid death in newborns. Alexion is conducting a natural history study in patients and has completed dosing in a Phase I trial of ALXN1101 in healthy volunteers. The company acquired the IV Escherichia coli-derived cyclic pyranopterin monophosphate (cPMP) replacement therapy from Orphatec Pharmaceutical GmbH (Niederkassel, Germany) in 2011 (see BioCentury, Feb. 14, 2011).

Separately, Alexion reported 3Q13 earnings that beat the Street. The company reported diluted non-GAAP EPS of $0.83, beating the Street's $0.79 estimate and up from $0.60 in 3Q12. Third quarter sales of Soliris eculizumab, which accounts for all of Alexion's revenues, rose 36% to $400.4 million. The Street was expecting $395.3 million. Alexion also raised its 2013 guidances and now expects $1.535-$1.54 billion in revenues and non-GAAP EPS of $3.02-$3.04. The company previously expected revenues of $1.52-$1.53 billion and EPS of $2.97-$3.02. The Street was expecting EPS of $3.03 on full-year revenues of $1.53 billion. Alexion markets Soliris for rare blood disorders paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). ...