Enobia Pharma initiates dosing in hypophosphatasia study

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 will be evaluated in both adults and infants afflicted with hypophosphatasia in Canada and the US.

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.

Robert Heft, CEO of Enobia, said: “In preclinical studies using a mouse model, ENB-0040 consistently improved survival as well as bone and dental manifestations of hypophosphatasia. The initiation of clinical studies brings us closer to the goal of providing drug therapy for hypophosphatasia patients where none currently exists.”