MONTREAL, March 3 /PRNewswire/ -- Enobia Pharma, an emerging Canadian biotech company focused on developing novel therapeutics for serious bone disorders, today announced completion of target enrollment in its pilot clinical trial in infants suffering from a severe form of hypophosphatasia. 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. 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 presented at the Endocrine Society meeting in June.
A Phase I study, which evaluated safety, tolerability and pharmacokinetics of ENB-0040 in adults with hypophosphatasia was completed in December of 2008. Results of that study will be presented at the American College of Medical Genetics in March.
"Everyone involved in the clinical program is extremely pleased with our progress," said Robert Heft, CEO of Enobia. "Since filing an IND 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."
Additional trials are planned. If interested in learning more, contact hypophosphatasia.clinical.trials@enobia.com.
About Hypophosphatasia
Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease. Affected patients have low levels of the tissue non-specific form of alkaline phosphatase, an essential 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 is cumulative and worsens with age. Clinical severity ranges from the severe perinatal or infantile forms, 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 pathological fractures that in some cases stops ambulation.
About ENB-0040
ENB-0040 is a subcutaneous enzyme replacement therapy of tissue non-specific alkaline phosphatase (TNSALP) fused to a patented bone targeting peptide. Preclinical studies in the "knockout" mouse model of severe hypophosphatasia showed that subcutaneous administration of ENB-0040 significantly improved survival, prevented the skeletal and dental manifestations of the disease and corrected skeletal defects in mice with established disease. ENB-0040, awarded orphan designation in the US and EU in 2008, is currently in phase II testing.
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.
Enobia Contact:
Julie Anne Smith, (514) 596-2901, extension 214
CONTACT: Julie Anne Smith, +1-514-596-2901, extension 214
