Enobia Pharma announced that it has successfully completed a Phase 2 study of ENB-0040 (asfotase alfa) for the treatment of juveniles with hypophosphatasia (HPP). HPP is a serious, rare metabolic bone disorder caused by a deficiency in the enzyme tissue non-specific alkaline phosphatase (TNSALP). This six-month, multinational, open-label investigation of the safety, tolerability, pharmacokinetics (PK), and efficacy of ENB-0040 treatment enrolled 13 children (5–12 years of age and Tanner Stage ≤2) with rickets and gross motor deficits from HPP. The juvenile study has met its primary endpoint, demonstrating a statistically significant improvement in rickets when compared with historical matched cohort controls (p=0.002). Nine of 13 children enrolled (69%) and nine of 12 children completing the study (75%) achieved a substantial improvement in rickets as assessed by skeletal radiographs of the wrists and knees, compared with two of 17 (12%) historical controls.
ENB-0040 is a subcutaneous enzyme replacement therapy of TNSALP fused to a bone targeting peptide. ENB-0040 is designed to directly target TNSALP to the bone in order to correct the enzyme deficiency, which could lead to restoration of normal bone mineralization.
For more information call (514) 596-2901 or visit www.enobia.com.