Alexion to pay as much as $1 billion for Enobia

CHESHIRE, Conn. & MONTREAL, CANADA—Alexion PharmaceuticalsInc. and Enobia Pharma Corp. have announced that they have signed a definitive agreement under which Alexion willacquire 100 percent of the capital stock of Enobia, a privatebiopharmaceutical company based in Montreal, Canada and Cambridge,Mass.

Enobia is focused on the development of therapies to treatpatients with ultra-rare and life-threatening genetic metabolicdisorders, and its lead product candidate ENB-0040 (asfotase alfa), is a humanrecombinant targeted alkaline phosphatase enzyme-replacement therapy forpatients suffering with hypophosphatasia (HPP), an ultra-rare,life-threatening, genetic metabolic disease for which there are noapproved treatment options. Alexion will acquire full worldwidedevelopment and commercial rights to asfotase alfa. Asfotase alfa wasawarded orphan drug designation in the United States and European Union in 2008 and fast track status in the United States in 2009, and is currently in Phase II clinicaldevelopment.

"Hypophosphatasia is an ultra-rare and life-threatening disease, andthose patients who survive live with debilitating morbidities includingskeletal deformity, severe muscle weakness, and progressive damage tovital organs," said Dr. Leonard Bell, CEO ofAlexion. "Asfotase alfa has shown very compelling Phase II clinical datain infants and juveniles with hypophosphatasia. The acquisition ofEnobia is very well aligned with Alexion's objective to develop anddeliver life-transforming therapies for patients suffering withultra-rare, severe, and life-threatening disorders."

"Alexion has proven expertise in developing and commercializingtherapies to transform the lives of patients with severe and ultra-raredisorders, making them the ideal partner to advance the work of theEnobia team and bring asfotase alfa to HPP patients around the world,"said Jonathan Silverstein, Enobia's chairman as well as a general partner of OrbiMed, which is a controlling shareholder in Enobia.

"Enobia and our scientific collaborators have developed an elegantcompound showing very promising clinical results to date," said Dr.Robert Heft, president and CEO of Enobia. "Togetherwith Alexion, we share a sharp focus on transforming the lives ofpatients with severe and ultra-rare disorders. The hypophosphatasiapatient community will be well served by the experience andinternational scope of Alexion."

HPP is characterized by defective bone mineralization and impaired phosphateand calcium regulation leading to progressive damage to multiple vitalorgans including destruction and deformity of bones, profound muscleweakness, seizures, impaired renal function, and respiratoryfailure. The severe manifestations of the genetic deficiency inHPP affect people of all ages, and approximately 50 percent of infantswith the disease do not survive past one year of age. HPP is caused by a genetic deficiency of an enzyme known as tissuenon-specific alkaline phosphatase (TNSALP), which causes life-longabnormalities in metabolism of the two vital minerals calcium andphosphate, leading directly to the debilitating morbidities andpremature mortality of the disease. T

Asfotase alfa is an investigational, first-in-classrecombinant protein that addresses the underlying cause of HPP bytargeting replacement of the missing enzyme to the necessary bodytissues. Asfotase alfa is designed to normalize the geneticallydefective metabolic process and prevent or reverse the severe andlife-threatening complications of life-long dysregulated mineralmetabolism in patients with HPP.

SOURCE: Alexion and Enobia news release