Ultragenyx Pharmaceutical Inc. RARE announced positive data from an ongoing pediatric phase II study on KRN23 for the treatment of X-linked hypophosphatemia ("XLH").
The randomized, open-label, dose-finding study is evaluating safety and efficacy of KRN23 in 36 pediatric XLH patients aged between 5 and 12 years. The study will be conducted for a period of 64 weeks in three cohorts.
Ultragenyx reported 16-week data from the study that showed an increase in serum phosphorus levels from baseline in all the patients. In the monthly dosing group, 71% patients attained the normal serum phosphorus range with a mean dose of 0.84 mg/kg per treatment, while in the biweekly dosing group, 50% patients attained the normal serum phosphorus range with a mean dose of 0.48 mg/kg per treatment.
Meanwhile, Ultragenyx provided an update on its development plans for KRN23. The company is gearing up for an expansion of its phase II pediatric study, and simultaneously, planning a phase III study in adult XLH patients. The company’s shares gained 6.8% reflecting positive reaction to the news by investors.
Based on a feedback from the European Medicines Agency and data from the ongoing phase II study, Ultragenyx intends to expand the phase II pediatric study by up to 16 patients to generate additional safety and efficacy data. The company will determine its future steps regarding the submission of a marketing application for conditional approval of the candidate in the EU after 40-week data from the phase II study becomes available (expected in late 2015 or early 2016). If necessary, Ultragenyx is prepared to reconsider its course of action upon the availability of data from the fully-expanded study (expected by mid-2016).
Meanwhile, the company is planning a randomized, double-blind, placebo-controlled phase III study in adults suffering from XLH, with the planned primary endpoint of assessing serum phosphorus levels at week 24. In addition, Ultragenyx intends to initiate a 48-week open-label bone quality study in adult XLH patients to evaluate the impact of KRN23 on underlying osteomalacia via bone biopsy. The company expects to start both studies in the second half of 2015.
We note that Ultragenyx has a collaboration and license agreement with Japan-based Kyowa Hakko Kirin for the development and commercialization of KRN23. Upon approval, KRN23 will be commercialized by Kyowa Hakko Kirin in Europe and Ultragenyx will be entitled to receive a royalty.
Ultragenyx carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector are Actelion Ltd. ALIOF, Gilead Sciences Inc. GILD and Acorda Therapeutics, Inc. ACOR. While Actelion and Gilead sport a Zacks Rank #1 (Strong Buy), Acorda carries a Zacks Rank #2 (Buy).
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