Ultragenyx Announces the Presentation of Data from a Single Dose Phase 1 Study, Conducted by Kyowa Hakko Kirin Co. Ltd. (KHK), of KRN23 in X-linked Hypophosphatemia (XLH) in Adults

News | 10. 06. 2013

Ultragenyx Pharmaceutical

KRN23 increased phosphate levels and was safe and well tolerated
NOVATO, CA – October 6, 2013— Ultragenyx Pharmaceutical Inc., a biotechnology company, announced the release of data from a first-in-human, randomized, double-blind, placebo-controlled, single dose study of a human monoclonal anti-FGF23 antibody (KRN23) in X-linked hypophosphatemia in adults. X-linked hypophosphatemia (XLH) is an inherited metabolic bone disease characterized by short stature and skeletal deformities. A Phase I study (US-02) was conducted by KHK with KRN23 to assess its safety and tolerability and to measure changes in biochemical markers in adult patients with XLH. Thirty-eight adults with XLH were randomized to receive single doses of KRN23 or placebo via intravenous (IV) (0.003 to 0.3 mg/kg) or subcutaneous (SC) (0.1 to 1.0 mg/kg) routes.