Enobia Pharma today announced positive results for its Phase II juvenile clinical trial of ENB-0040 (asfotase alfa), an experimental bone-targeted enzyme replacement therapy intended for the treatment of hypophosphatasia (HPP). Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease that affects individuals of all ages. Michael Whyte, MD, Medical/Scientific Director of the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospital for Children in St. Louis, Missouri, and a principal investigator of the Phase II study, presented the findings at the European Society for Paediatric Endocrinology (ESPE) Annual Meeting in Prague. Enobia's abstract describing these Phase II results was awarded the Henning Anderson Prize for best clinical abstract by ESPE.
In this trial, after six months of ENB-0040 treatment results include:
- Radiographic improvement of rickets
- Improvement in muscle strength and agility over baseline, including an average improvement of 125m on six-minute walk test
- Amelioration of pain in 6 of 7 patients experiencing pain at baseline
"We are encouraged by these preliminary results. Improvements were seen in objective measures of muscle strength, agility and pain. These were accompanied by histomorphometric, radiologic and biomarker improvements, suggesting an integrated improvement of this debilitating skeletal disorder," said Hal Landy, MD, Chief Medical Officer at Enobia. "With nearly 50 patients on ENB-0040 treatment, we are rapidly expanding our understanding of the potential safety and efficacy of ENB-0040."
Dr. Michael Whyte, Medical/Scientific Director of the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospital for Children in St. Louis and a Principal Investigators said, "The findings from this study represent an important advance in our quest to find a medical treatment for hypophosphatasia."