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news.Enobia Pharma, a biotechnology company, has completed target enrollment in its pilot clinical trial in infants suffering from a severe form of hypophosphatasia.
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Enobia is investigating enzyme replacement therapy (ERT) with ENB-0040 for the treatment of this rare and often crippling genetic bone disorder for which there is no approved treatment.
ENB-002-08 is an open-label, six-month study evaluating safety, efficacy and pharmacokinetics of subcutaneous ENB-0040 in hypophosphatasia infants with severe rickets and respiratory compromise.
Approximately six severely affected infants from the EU, US and the Middle East will be treated at approved sites in the US, Canada, UK and the United Arab Emirates. Key efficacy outcomes include improvement in rickets and clinical manifestations of the disease. Results from the trial will be reported in June 2009.
A Phase I study, which evaluated safety, tolerability and pharmacokinetics of ENB-0040 in adults with hypophosphatasia was completed in December 2008. Results of that study will be announced in March 2009.
Robert Heft, CEO of Enobia, said: “Everyone involved in the clinical program is extremely pleased with our progress. Since filing an investigational new drug only eight months ago, we have completed a Phase I study and enrolled this Phase I/II study in infants. We believe ENB-0040 will be shown to significantly improve the devastating skeletal manifestations of hypophosphatasia and may potentially be life-saving therapy for those suffering from the lethal form of the disease.”