Enobia Completes Target Enrollment in Clinical Trial for Infantile Hypophosphatasia
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
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
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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.
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.
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