Ultragenyx Pharmaceutical Spikes 4% Following Release Of Positive Phase 2 Data

Shares of Ultragenyx Pharmaceutical Inc RARE gained more than 4 percent on Thursday after company reported positive interim data from a Phase 2 study.

Ultragenyx Pharmaceutical focuses on the development of novel products for rare and ultra-rare diseases. The company announced that initial data from the first eight patients in a Phase 2 study of KRN23 for the treatment of tumor-induced osteomalacia demonstrated an improvement in serum phosphorus levels and other bone metabolism measures.

The primary objectives of the study are to establish the dose, and assess the safety profile and efficacy of treatment with KRN23 in adults with TIO and ENS. Patients receive subcutaneous injections of KRN23 once every four weeks for 48 weeks.

The study is expected to expand to to 15 patients and enrollment is still ongoing.

"The data show that KRN23 can restore serum phosphorus levels to normal ranges and may have a meaningful effect on bone health in patients," said Sunil Agarwal, M.D., Chief Medical Officer of Ultragenyx. "We now have data in two diseases, TIO and XLH, suggesting that KRN23 binds to and inhibits the activity of excess fibroblast growth factor 23 (FGF23), the hormone that leads to phosphate-wasting in these patients."