News da: Biz.Yahoo.com 01/12/2008
Enobia initiates infantile Enzyme Replacement Trial for rare bone disease
Enobia initiates infantile Enzyme Replacement Trial for rare bone disease
Monday December 1, 12:42 pm ET
Baby Girl with Hypophosphatasia Treated With ENB-0040 at Children's Hospital, Health Sciences Centre in Winnipeg
MONTREAL, Dec. 1 /CNW Telbec/ - Enobia Pharma, an emerging Canadian biotech company focused on developing novel therapeutics for serious bone disorders, today announced that the first infant patient in its clinical program for hypophosphatasia was 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.
"Our enzyme replacement therapy program for hypophosphatasia has progressed well," said Robert Heft, CEO of Enobia. "Based upon the excellent preclinical data we are hopeful that hypophosphatasia patients will benefit from treatment with ENB-0040. We're grateful for everyone involved in expediting the treatment of this patient."
Under two separate protocols, ENB-0040 will be evaluated in both adults and infants afflicted with hypophosphatasia at sites in Canada and the United States. In August, physicians at the University of Manitoba dosed the first adult patient as part of that cohort to evaluate the safety, tolerability and pharmacokinetics of ENB-0040.
Enobia Pharma is pleased to reproduce below today's announcement made by Children's Hospital, Health Sciences Centre Winnipeg regarding the dosing of the first infant patient in the early ENB-0040 studies.
CHILD RECEIVING POTENTIALLY LIFE-SAVING TREATMENT AT HSC AS PART OF
INTERNATIONAL DRUG TRIAL
Infant Girl From Belfast Flown To Winnipeg
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 last month 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 I study 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.
Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease. Patients have low levels of the tissue non-specific form of alkaline phosphatase, an important regulator of bone mineralization, leading to rickets in infants and children and osteomalacia ("soft bones" resulting from poor mineralization) in adults. Disease severity is inversely proportional to the age at symptom onset, but morbidity can be cumulative and worsen with age. Clinical severity ranges from the severe perinatal or infantile form, with profound skeletal hypomineralization and respiratory compromise often causing death, to a more slowly progressive and debilitating osteomalacia in adults.
In the infantile form, infants may appear normal at birth but develop serious symptoms in the first six months of life. These can include failure to thrive, respiratory failure, fractures, and seizures. Radiographic findings include generalized hypomineralization and rickets. Mortality in these patients may be as high as 50%. In the childhood form, patients have varying degrees of hypomineralization, frank rickets, short stature, bone pain, muscle weakness, delayed motor milestones, early loss of deciduous teeth, and may experience frequent, poorly-healing fractures. In the adult form, the underlying osteomalacia causes bone pain due to overt or poorly-healing stress fractures that in some cases stops ambulation.
ENB-0040 is a fusion protein that includes the catalytic domain of human tissue non-specific alkaline phosphatase (TNSALP), and a patented peptide used to target the enzyme to bone. The preclinical studies of ENB-0040 in the "knockout" mouse model of severe hypophosphatasia were recently published in the Journal of Bone and Mineral Research (June 2008:23:777-787) and showed that subcutaneous administration of ENB-0040 significantly improved survival and prevented the skeletal and dental manifestations of the disease. In addition to the ongoing trials, pediatric studies are also being planned. If interested in learning more, contact Hypophosphatasia.Clinical.Trials(at)enobia.com.
About Enobia Pharma Inc.
Enobia Pharma Inc., is a private, Montreal based company focused on the development of therapeutics to treat serious bone disorders for which there is no currently approved drug therapy. Enzyme Replacement Therapy for the treatment of hypophosphatasia is the Company's lead program. In 2007 Enobia completed a $40M Series B financing lead by OrbiMed Advisors and CTI Life Sciences.
For further information
Heidi Graham, Winnipeg Regional Health Authority, (204) 926-7178, Cell: (204)
Durhane Wong-Rieger, PhD, Canadian Organization for Rare Disorders, (416) 969-7435
Julie Anne Smith, Enobia Pharma, (516) 596-2701 x214
Source: ENOBIA PHARMA