| HOME >> MEDICINE >> TECHNOLOGY |
MONTREAL, Aug. 20 /PRNewswire/ -- Enobia Pharma, an emerging biotech company focused on developing novel therapeutics for serious bone disorders, today 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 rare and often crippling 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 USA. "This may well be a milestone in the treatment of hypophosphatasia," said Michael Whyte, MD, Professor of Medicine at Washington University School of Medicine, St. Louis, MO, and consultant to Enobia Pharma. "Studies in a mouse 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 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.
"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," stated Robert Heft, PhD, Chief Executive Officer of Enobia.
About Hypophosphatasia
Hypophosphatasia is a rare, inherited, and sometimes fatal metabo
'/>"/>
| SOURCE Enobia Pharma Copyright©2008 PR Newswire. All rights reserved |