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 having improved respiratory status or requiring no respiratory support at the final analysis. The data were presented in a late-breaking presentation at the 9
Joint Meeting of Paediatric Endocrinology in Milan, Italy.
“Hypophosphatasia is a genetic, very rare metabolic disease that can have devastating and life-threatening consequences. HPP is characterized by profound hypomineralization and a number of systemic effects, including respiratory, neurologic and renal complications. Infants who develop their first symptoms of HPP before 6 months of age have a very poor prognosis with an estimated 50% mortality rate,” said lead investigator Dr. Cheryl R. Greenberg, of the University of Manitoba in Winnipeg, Canada. “This study showed that treatment with asfotase alfa improved bone mineralization in infants and young children with HPP, and improved or preserved respiratory function, which is often a cause of death in these patients.”
“We are excited about these results as they are consistent with previously reported positive data now in a broader patient population of infants and children,” said Martin Mackay, Ph.D. Executive Vice President, Global Head of R&D at Alexion. “This further supports the potential of asfotase alfa as the first treatment for HPP, and we look forward to completing our registration program so that we may begin serving patients with this severe and often life-threatening disease.”