Enobia Pharma today announced positive data from a clinical study of ENB-0040, a bone targeted enzyme replacement therapy, under investigation for the treatment of hypophosphatasia (HPP). After six months of treatment with ENB-0040, four of five severely affected patients showed marked improvements in bone mineralization, correction of skeletal defects, better respiratory function, including weaning from assisted ventilation, and cognitive and motor development. These findings were presented by Dr. Michael Whyte at the 31(st) Annual Meeting of the American Society for Bone and Mineral Research in Denver, CO.
HPP is a genetic disease characterized primarily by defective bone mineralization and is caused by a deficiency in the enzyme tissue non-specific alkaline phosphatase (TNSALP). This enzyme plays a key role in regulating skeletal mineralization. There are currently no therapies approved for HPP. As an enzyme replacement therapy designed to specifically target TNSALP to the bones, ENB-0040 may help correct the enzyme deficiency and restore bone mineralization.
"Poor bone formation in infants with severe hypophosphatasia has devastating consequences, including fractures, respiratory failure and death in approximately half the infants," stated Michael P. Whyte, M.D., Medical/Scientific Director of the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospitals for Children in St. Louis and an investigator for the study. "The findings from this study are very encouraging, as they demonstrate correction of the primary skeletal disturbance after treatment with ENB-0040."
"The results from this study in infants and young children mark an important milestone for our program," said Hal Landy, M.D., Chief Medical Officer and Vice President, Medical Affairs of Enobia. "We are pleased that all five patients who completed the study have opted to continue treatment under a long-term extension protocol, and we have amended the initial study to include an additional five patients. We will also begin enrollment of a Phase 2 study in children aged five to 12 years in the weeks ahead."