Ultragenyx Reports Positive Phase 2 Study Data

Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) revealed positive interim data from the second-stage study of KRN23 for tumor-induced osteomalacia (TIO) treatment. The company indicated its interim data at 24 weeks from the first eight patients showed KRN23 improved serum phosphorus levels and bone metabolism measures. This included a patient with epidermal nevus syndrome or ENS.

The company is conducting the second-stage study through a collaboration and license agreement with Kyowa Hakko Kirin. The company said these data were presented at the American Society for Bone and Mineral Research (ASBMR) 2016 Annual Meeting on Monday.

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Ultragenyx CEO and President Emil Kakkis said, "Patients with TIO have substantial hypophosphatemia, osteomalacia and fractures. These data support that KRN23 could potentially reverse some of these symptoms and improve bone health in patients."

The company disclosed that overall the study demonstrated improvement in serum phosphorus and other bone mineral metabolism measures that is in line with what was observed in trials of KRN23 in pediatric and adult patients with X-linked hypophosphatemia (XLH).

Following the news, the stock advanced $1.38, or 1.79 percent, to $78.50 in the pre-market trading on Monday. At time of publication, the stock was up 3.12 percent at $79.53.

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