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Enobia Pharma 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
MONTREAL, Canda | August 20, 2008 | Enobia Pharma, an emerging Canadian 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 is being evaluated in both adults and infants afflicted with hypophosphatasia in Canada and the USA.
“This is a significant step towards a treatment for hypophosphatasia, an under-recognized bone disease that can be fatal in infants and cause serious disability in older patients,” said Dr. Cheryl Greenberg, Medical Director, Child Health Program, Winnipeg Regional Health Authority, and Professor and Head, Department of Pediatrics & Child Health, University of Manitoba. “We are delighted to begin testing a potential treatment. I’m particularly grateful to our Institutional Review Board and Office of Research Services at the University of Manitoba, as well as the Research Impact Committees of Health Sciences Centre Winnipeg for their timely and constructive review of our protocols. This collaboration has allowed us to expeditiously enroll our group of patients in this clinical trial.”
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 metabolic bone disease. The term hypophosphatasia was coined in 1948 by Dr. John Rathburn, a Toronto pediatrician who was among the earliest clinicians to describe the disease. In 1955 Dr. Donald Fraser at the University of Toronto identified the first biomarker (PEA) helpful in diagnosing hypophosphatasia. In 1957 Dr. Fraser introduced the “infantile”, “childhood” and “adult”
classification of hypophosphatasia still in use today. Based on his experience over a 10 year period in Toronto, Dr. Fraser estimated the incidence of the severe forms of hypophosphatasia to be 1:100,000 in the general population. The highest known incidence of hypophosphatasia is among the Mennonite Community living in Manitoba where 1:2500 individuals are born with the disease.
In hypophosphatasia, 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 may stop ambulation.
About ENB-0040
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
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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. Enobia’s other investors include: Fonds de solidarité FTQ, Desjardins Capital régional et coopératif, Lothian Partners 27 (SARL) Sicar, T2C2/BIO 2000 s.e.c..
SOURCE: Enobia Pharma Inc.
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