Alexion could shell out another $470 million in development milestones.

Alexion Pharmaceuticals is buying Enobia Pharma for an initial $610 million in cash so it can acquire the latter’s lead recombinant human alkaline phosphatase enzyme replacement therapy, ENB-0040 (asfotase alfa). The candidate is in Phase II development for treating hypophosphatasia (HPP).

The all-cash transaction could see Alexion shell out another $470 million in regulatory and sales-related milestones. The firm says it plans to finance the acquisition through existing cash reserves and $300 million in committed bank debt.

HPP is a rare and potentially fatal inherited metabolic bone disease characterized by poor bone minerailization and profound skeletal defects. The disease is caused by a deficiency in tissue nonspecific alkaline phosphatase, which causes abnormalities in the metabolism of calcium and phosphate. Asfotase alfa has been developed to replace the missing enzyme in relevant tissues. The enzyme replacement therapy has received orphan drug designation in the EU and U.S. and been granted fast track status in the U.S.

“The acquisition of Enobia is very well aligned with Alexion’s objective to develop and deliver life-transforming therapies for patients suffering with ultrarare, severe, and life-threatening disorders,” comments Leonard Bell, M.D., Alexion CEO. “Asfotase alfa has shown very compelling Phase II clinical data in infants and juveniles with hypophsphatasia.”

Alexion is focused on the development and commercialization of treatments for severe and ultrarare disorders. The firm’s marketed product, Soliris® is a terminal complement inhibitor developed for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).  Soliris is currently approved in more than 35 countries for the PNH indication. In September and November the drug was granted FDA and EU approval, respectively, for the treatment of aHUS.

Alexion is separately evaluating other potential indications for Soliris and has leveraged its complement inhibition and antibody platforms to generate a clinical development-stage pipeline spanning the fields of hematologic, kidney, and neurologic diseases, transplant rejection, cancer and autoimmune disorders.

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