Akp2-/- Mouse Model Mimics Human Infantile Hypophosphatasia
The knockout mouse model utilized in nonclinical studies of ENB-0040 was used for its similarities to disease manifestations in patients with the human infantile form of HPP.
| Akp2-/- mice |
Human Infantile HPP |
- Onset of skeletal disease at day 6 and
death generally by day 21
- Progressive rachitic changes
- Osteopenia and fractures
- Epileptic seizures and apnea
- Poor feeding/inadequate weight gain
|
- Onset before 6 months of age
and often death before 1 year
- Severe rickets
- Spontaneous fractures
- Occasional seizures and apnea
- Poor feeding/inadequate weight gain
|
Nonclinical research in the knockout mouse model of severe hypophosphatasia showed that daily subcutaneous administration of ENB-0040 has exhibited pre-clinical activity in the treatment of HPP.54, 55
54 Enobia Pharma. Enobia Pharma Presents Preclinical Data Showing ENB-0040 Significantly Imbroved Survival and Healed Skeletal Manifestations of Severe Hypophosphatasia in Mice. 15 Sept 2008 [cited 16 June 2009]. Available from: http://www.enobia.com/press_releases-4.html
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55 Millán J.L. , Narisawa S. , Lemire I., Loisel T.P, Boileau G., Leonard P., Gramatikova S., Terkeltaub R., Camacho N.P., McKee M.D. , Crine P., Whyte M.P. (2008) Enzyme Replacement Therapy for Murine Hypophosphatasia. Journal of Bone and Mineral Research,. 23:777-787.
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