Ultragenyx Begins Phase 2 Study Of KRN23 For Pediatric XLH In US, EU

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE), Tuesday said it has begun the Phase 2 trial of the human monoclonal anti-FGF23 antibody KRN23 in pediatric patients with X-linked hypophosphatemia or XLH, a type of metabolic bone disease.

The multi-center, randomized, open-label, dose-finding Phase 2 clinical study will assess the safety and efficacy of KRN23 in nearly 30 prepubertal pediatric XLH patients. The primary goals of the study are to identify a dose and dosing regimen and to establish the safety profile of treatment with KRN23 in pediatric XLH patients. The study has been evaluated and accepted for conduct by the United States Food and Drug Administration or FDA, the United Kingdom Medicinal and Health Regulatory Authority or MHRA, and the Dutch Medicines Evaluation Board or CBG-MEB.

Further, the company said, it plans to advance the clinical development in the adult population along with the pediatric program.