Lead molecule ENB-0040 has completed mid-stage studies in infants and juveniles.
Enobia Pharma raised $40 million in a private placement to fund further development of its Phase II-stage enzyme replacement therapy (ERT) ENB-0040 for the genetic bone disorder hypophosphatasia. The disease is caused by deficiency in a tissue-nonspecific form of alkaline phosphatase (TNSALP), and causes inorganic pyrophosphate to accumulate in the extracellular space and inhibit skeletal mineralization, leading to rickets in pediatric patients and osteomalacia in adults.
Lead hypophosphatasia candidate ENB-0040 is designed to directly target functional TNSALP to the patient’s bone to help correct enzyme deficiency. The ERT has already completed two clinical trials in younger hypophosphatasia patients: a Phase I/II study in infants and young children, and a Phase II study in children aged 5–12 years. Enrollment into a Phase II trial evaluating ENB-0040 in adolescent and adult patients was completed in February.
Data from the study in juveniles was reported in January, and confirmed therapy using ENB-0040 led to significant improvements in rickets in treated patients. Participants in both the juvenile and infant trials are continuing to receive therapy under extension protocols, and new patients under the age of five years are still being enrolled.
Enobia was in addition recently awarded a $1.2 million, three-year FDA orphan grant to support development of ENB-0040 for the long-term treatment of infants and young children with hypophosphatasia.