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December 1, 2008

Child Receiving Potentially Life-Saving Treatment at HSC as Part of International Drug Trial

Child Receiving Potentially Life-Saving Treatment at HSC as Part of International Drug Trial

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Infant Girl From Belfast Flown to Winnipeg

A 9-month-old baby girl from Belfast is receiving potentially life-saving experimental treatment at Children's Hospital, Health Sciences Centre Winnipeg as part of an international drug trial.

The child has a bone disorder known as infantile hypophosphatasia, an often-fatal condition that affects only one in every 100,000 children born. She was flown to Winnipeg in late October with her parents and two-year old sister after physicians in Northern Ireland learned of the drug trial and contacted the principal investigator in Winnipeg.

Dr. Cheryl Rockman-Greenberg, Medical Director of the Winnipeg Regional Health Authority's Child Health Program, said the baby girl has been receiving "enzyme replacement" treatment by injection under the skin (subcutaneous) 3 times weekly for the past 4 weeks and to date has not shown any side-effects.

"We are very encouraged so far," Dr. Rockman-Greenberg said. "But we are still in the early stages, and it will be several more weeks, possibly months before we know whether or not the drug has worked as we hope it will."

This rare disease leads to severe rickets in infants and children and debilitating osteomalacia - 'soft bones' - in adults. The earlier the symptoms appear in the patient, the more severe they will be. When symptoms appear in infancy, up to fifty percent of hypophosphatasia patients die. Montreal-based biotechnology company Enobia recently initiated the clinical testing of the drug called ENB-0040 here in Winnipeg after receiving the appropriate approval from Health Canada for a Phase 1 clinical trial in adults. In addition, Enobia has regulatory approval to enroll several additional severely affected infants who meet study criteria in a parallel study. There is no approved treatment for the disease. To date Winnipeg is the only approved study site for the infantile protocol.

The infant girl will remain in Winnipeg for at least two, and possibly five, more months in order to complete her treatment. She, her parents and sister were flown to the city from Belfast with the assistance of the Canadian Organization for Rare Disorders (CORD).

"CORD is delighted to have played a role in helping to bring this infant to Canada to receive potentially life-saving therapy," CORD President Dr. Durhane Wong-Rieger said. "It is not often that Canada gets to be a leader in rare disorders research and this clinical trial is an affirmation of Canada's potential. We are grateful to Health Canada, the WRHA, the University of Manitoba and Enobia for helping to make this happen."

"These efforts underscore the potential severity of hypophosphatasia and the hope that ENB-0040 will be a safe and effective treatment for this disease," said Hal Landy, MD, VP Medical Affairs & Chief Medical Officer at Enobia Pharma. "This family has gone to extraordinary lengths to have their daughter participate in this study. We're extremely pleased to hear that she appears to be accepting the treatment well and look forward to following her progress. We continue to enroll patients in our adult Phase 1 and will be treating other pediatric patients from North American and around the world in the coming months."

The adult male who was initially enrolled in Phase 1 of the trial - designed to demonstrate the drug's safety - as well as two others who entered afterwards are all doing well.

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