Enobia Pharma initiates clinical testing of Enzyme Replacement Therapy to treat hypophosphatasia

22-Aug-2008 - Canada

Enobia Pharma announced that the first patient in its clinical program for hypophosphatasia has been dosed. Enobia is investigating Enzyme Replacement Therapy (ERT) with ENB-0040 for the treatment of this genetic bone disorder for which there is no approved treatment. Under two separate protocols, ENB-0040 is being evaluated in both adults and infants afflicted with hypophosphatasia in Canada and the USA.

Under the first protocol, safety, tolerability and pharmacokinetics of ENB-0040 will be evaluated for one month in an open-label, dose escalation Phase I study of ENB-0040 delivered intravenously and subcutaneously to six adults with hypophosphatasia at three North American sites.

Under the second protocol, safety, tolerability, pharmacokinetics, and efficacy of ENB-0040 will also be evaluated in a six-month open label study of up to six infants with particularly severe hypophosphatasia. Key efficacy outcomes include assessment of skeletal and respiratory manifestations of the disease.

ENB-0040 is a fusion protein that includes the catalytic domain of human tissue non-specific alkaline phosphatase (TNSALP), and a patented peptide used to target the enzyme to bone. The preclinical studies of ENB-0040 in the "knockout" mouse model of severe hypophosphatasia were recently published in the Journal of Bone and Mineral Research (June 2008:23:777-787) and showed that subcutaneous administration of ENB-0040 significantly improved survival and prevented the skeletal and dental manifestations of the disease. In addition to the ongoing trials, pediatric studies are also being planned.

https://www.bionity.com/en/news/86031/enobia-pharma-initiates-clinical-testing-of-enzyme-replacement-therapy-to-treat-hypophosphatasia.html