Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, Kyowa Hakko Kirin Co., Ltd. (Kyowa Hakko Kirin) and Kyowa Kirin International PLC announced positive 48-week data from the randomized, double-blind, placebo-controlled Phase 3 study of burosumab (KRN23) in adults with Xlinked hypophosphatemia (XLH).
Treatment with burosumab for 48 weeks showed sustained maintenance of normal serum phosphorus levels and further improvement in stiffness, physical function and pain. Patients who crossed over from placebo to burosumab after 24 weeks showed normalization of serum phosphorus and improvement in stiffness, pain and physical functioning.
An increased rate of fracture healing, in favor of burosumab treated patients, was observed during the first 24 weeks of burosumab treatment and this increased up to 48 weeks of treatment. Placebo patients who crossed over to burosumab showed a similar increased rate of fracture healing. The safety profile was consistent with what has been previously observed in this study and in other open label studies of burosumab in adults and children. Ultragenyx is conducting the study under a collaboration and license agreement with Kyowa Hakko Kirin. Burosumab is being developed by Ultragenyx, Kyowa Hakko Kirin and Kyowa Kirin International.
“This longer term data on symptom improvement and fracture healing support burosumab’s potential value in treating serious disease symptoms and promoting bone healing in adult patients with XLH,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “The continued clinical improvements in patients and the new data demonstrating a significant decrease in pain medication use after treatment with burosumab provide further support for the potential value in the treatment of adults with XLH.”
“This study provides valuable additional long term data for adult patients with XLH,” said Mitsuo Satoh, Executive Officer, Vice President, Head of Research and Development Division of Kyowa Hakko Kirin. “I believe burosumab has the potential to be an effective treatment option for patients with XLH.”
“The longer term data from this adult Phase 3 study demonstrates the potential of burosumab to positively impact the lives of patients with XLH and we look forward to progressing our discussions with the regulatory bodies in Europe and the US,” said Dr. Tom Stratford, President and CEO of Kyowa Kirin International.
About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com
About Kyowa Kirin Kyowa Hakko Kirin Co., Ltd. is a research-based life sciences company, with special strengths in biotechnologies. In the core therapeutic areas of oncology, nephrology and immunology/allergy, Kyowa Hakko Kirin leverages leading-edge biotechnologies centered on antibody technologies, to continually discover innovative new drugs and to develop and market those drugs world-wide. In this way, the company is working to realize its vision of becoming a Japan-based global specialty pharmaceutical company that contributes to the health and wellbeing of people around the world. Kyowa Kirin International PLC is a wholly owned subsidiary of Kyowa Hakko Kirin and is a rapidly growing specialty pharmaceutical company engaged in the development and commercialization of prescription medicines for the treatment of unmet therapeutic needs in Europe and the United States. Kyowa Kirin International is headquartered in Scotland. You can learn more about the business at: www.kyowa-kirin.com