Enobia selected Lonza for the production of its bone-targeted enzyme-replacement therapy ENB-0040, under investigation for the treatment of hypophosphatasia (HPP). Lonza will provide process validation and commercial manufacturing at its large-scale biologics site in Portsmouth, NH, with an option to extend production to a second Lonza site.
HPP is caused by a deficiency in the tissue nonspecific form of alkaline phosphatase (TNSALP), an enzyme that plays a key role in regulating bone mineralization. Without TNSALP, inorganic pyrophosphate accumulates extracellulary and inhibits skeletal mineralization, leading to rickets in pediatric patients or osteomalacia in adults. ENB-0040 is proposed to directly target functional alkaline phosphatase to the patient’s bones to help correct the enzyme deficiency.
Enobia recently completed a Phase II juvenile study. It met the primary endpoint, demonstrating a statistically significant improvement in rickets when compared with historical matched cohort controls. Rickets, a softening and weakening of the bones, is often seen in children with HPP and can lead to fractures, pain, and deformity.
The company previously reported the successful completion of a trial in infants. Participants reportedly continue to demonstrate functional and respiratory improvements in longer-term follow-up. A Phase II study in adolescents and adults with HPP is ongoing.