Ultragenyx Pharma (RARE) Commences KRN23 Phase 3 in XLH

October 26, 2016 8:31 AM EDT

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Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE) announced the initiation of the Phase 3 study of KRN23 for the treatment of pediatric patients with X-linked hypophosphatemia (XLH). Kyowa Hakko Kirin and Ultragenyx entered into a collaboration and license agreement in August 2013 to develop and commercialize KRN23.

“The initiation of the Phase 3 study of KRN23 in pediatric XLH patients is a significant step forward in our overall development plan for this debilitating bone disease,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “Preliminary data from studies in children and adults with XLH indicate that KRN23 can increase serum phosphorus and reduce bone disease. This study will allow us to directly assess whether KRN23’s potential impact on rickets, phosphorus and other endpoints is superior to that of conventional oral phosphate and vitamin D therapy for children with XLH.”

The Phase 3 study is a randomized, open-label clinical study comparing the efficacy and safety of KRN23 to oral phosphate and active vitamin D therapy. The study will enroll approximately 60 patients ages one through 12 in the US, EU, Canada, Japan, and Korea. The primary endpoint is the change in rickets at 40 weeks, assessed by the radiographic global impression of change (RGI-C) scale. Secondary endpoints include additional rickets assessments using the RGI-C scale and the Thacher Rickets Severity Scoring (RSS) system, changes in growth velocity and height, pharmacodynamic assessments, walking ability, patient reported outcomes assessing pain, fatigue and physical function, and safety. All patients were previously treated with oral phosphate and active vitamin D therapy, and go through a 7-day washout period prior to randomization. Patients in the KRN23 treatment group receive a starting dose of 0.8 mg/kg administered biweekly, and the dose may be increased up to 1.2 mg/kg.



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