Enobia Pharma initiates study of bone-targeted enzyme replacement therapy for hypophosphatasia

Nov 4 2009

Enobia Pharma announced today the initiation of a Phase 2 clinical study of ENB-0040, a bone-targeted enzyme replacement therapy, under investigation for the treatment of hypophosphatasia (HPP). The six-month study will assess the safety, efficacy and pharmacokinetics of ENB-0040 in up to 12 children with HPP. This study follows the recent presentation of positive Phase 2 data in infants, which showed that, in the majority of patients, six months of treatment with ENB-0040 led to marked improvements in bone mineralization, correction of skeletal defects, better respiratory function, and improvements in cognitive and motor development.

Enobia also announced today the launch of the Hypophosphatasia Impact Patient Survey (HIPS), an online questionnaire intended to better understand the burden of HPP on patients and their families. The survey is designed to document the disease history, medical history, functional disability, and quality of life in patients with HPP.

There are currently no therapies approved for HPP, a rare genetic disease characterized primarily by defective bone mineralization caused by a deficiency in the enzyme 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.

"We continue to make strong progress with ENB-0040, and we are pleased to expand our clinical program with the start of the Phase 2 study in children and the launch of the HPP Impact Patient Survey," said Robert Heft, Ph.D., President and Chief Executive Officer of Enobia. "The expansion of our clinical program and insights gained from our online questionnaire will add to the collective body of knowledge about HPP and support our efforts to bring this promising therapy to patients as quickly as possible."

SOURCE Enobia Pharma Inc.