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Alexion Presentation Shows Phase 2 Study of Asfotase Alfa in Infants and Young Children with HPP Met Primary Endpoint


Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) today announced that
researchers have presented data from an ongoing multinational Phase 2
study of asfotase alfa in infants and young children with
hypophosphatasia (HPP), an inherited, ultra-rare metabolic disorder that
in this patient population leads to progressive damage to multiple vital
organs, destruction and deformity of bones, and death. The
study met its primary endpoint: infants and young children with HPP
treated with asfotase alfa had significant improvement in skeletal
mineralization from baseline as assessed radiographically after 24 weeks
of treatment (p=0.001). This response was observed as early as 12 weeks
and improvement continued at 48 weeks. Ninety three percent of the
patients survived the first 48 weeks of treatment with 80% of patients

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