Enobia Pharma - Nonclinical Studies

Akp2^-/- Mouse Model Mimics Human Infantile Hypophosphatasia

The knockout mouse model utilized in nonclinical studies of A subcutaneous enzyme replacement therapy with affinity for bone that is currently in clinical trials.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 A softening of bones in children leading to fractures and deformity, which is predominantly caused by a vitamin D deficiency.rickets Spontaneous fractures Occasional seizures and apnea Poor feeding/inadequate weight gain

Nonclinical research in the knockout mouse model of severe A rare, inherited, and sometimes fatal metabolic bone disease characterized by poor bone mineralization and profound skeletal defects.hypophosphatasia showed that daily subcutaneous administration of ENB-0040 has exhibited pre-clinical activity in the treatment of HPP.⁵⁴^,⁵⁵

Nonclinical Studies