Enobia Pharma today announced positive interim data from a clinical study of ENB-0040, a bone targeted enzyme replacement therapy, under investigation for the treatment of hypophosphatasia (HPP). After 12 weeks of treatment with ENB-0040, children with HPP showed marked improvements in bone mineralization and function including increases in strength, endurance and mobility and reduction in pain. These findings were presented by Dr. Michael Whyte at ENDO 2010, the 92nd Annual Meeting of The Endocrine Society.
There are currently no therapies approved for HPP, a rare, inherited, and sometimes fatal metabolic bone disease that affects individuals of all ages. HPP is caused by a deficiency in tissue non-specific alkaline phosphatase (TNSALP). This enzyme plays a key role in regulating skeletal mineralization. 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.
"As we have already seen in infants, we are encouraged to see that children with hypophosphatasia begin to show improvements within a few weeks after first receiving ENB-0040. We hope to confirm these early data once the trial is completed. We expect to present the full results from the study in children during the second half of 2010," stated Hal Landy, MD, Chief Medical Officer and Vice President, Medical Affairs at Enobia. "We continue to make strong progress with the ENB-0040 clinical program and are committed to bringing ENB-0040 to patients as quickly as possible."