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A possible milestone in the treatment of hypophosphatasia, a rare and often crippling genetic bone disorder, has been announced by emerging biotech company Enobia Pharma.
It is researching enzyme replacement therapy (ERT) with ENB-0040 to tackle the condition, for which there is as yet no approved treatment.
Under two separate protocols, ENB-0040 will be evaluated in both adults and infants afflicted with hypophosphatasia in Canada and the USA.
“This may well be a milestone in the treatment of hypophosphatasia,” said Michael Whyte, professor of medicine at Washington University School of Medicine. “Studies in a model for hypophosphatasia show ENB-0040 has the potential to greatly enhance the quality of life for patients.”
Under the first protocol, safety, tolerability and pharmacokinetics 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.
Under the second protocol, similar tests will be evaluated in a six-month open-label study of up to six infants with particularly severe hypophosphatasia.
Enobia boss Robert Heft said: “The initiation of clinical studies brings us closer to the goal of providing drug therapy for hypophosphatasia patients where none currently exists.”
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