Boston, MA 06/19/2014 (wallstreetpr) – Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) is developing a treatment for X-linked Hypophosphatemia (XLH) through its drug candidate known as KRN23. If successful, the drug will play an important role in the treatment of XLH because there is currently no existing approved therapy for the medical problem.
The available remedy involves oral care where patients are subjected to vitamin-D therapy. However, the therapy not only has lower efficacy, but also comes with numerous side effects.
The company has announced the initiation of a Phase II clinical trial of XLH, which will be conducted on children of ages between 5 and 12 years. The study will last 64 weeks, and the treatment will be administered once after every four weeks for the trial duration. The study will capture 30 patients, and the primary goal of the study is to establish the efficacy and safety of the treatment. Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) also intends to monitor any side effects associated with the investigational drug.
Shares of the company jumped more than 14 percent on Wednesday on the news of Phase II study initiation. The stock is up 15.2 percent since the beginning of 2014.
Presenting study results
As Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) enters Phase II study of KRN23 on children, the company will next week between Jun 23 and June 24, the present result of Phase 1/2 study of the drug on adults. The presentation will take place at the ICE/ENDO meeting.
Ultragenyx is developing the treatment in collaboration with Kyowa Hakko Kirin Pharma, Inc., which spearheaded the Phase 1/2 study
In addition to the presentation of the KRN23 study results at ICE/ENDO, the company said its CEO Emil D. Kakkis will present at the JMP Securities Healthcare Conference. The JMP event will takes place on June 25, at the Westin New York Grand Central. The event may offer greater insight into the company’s pipeline and growth plans. At least it is already known that the drug (KRN23) has a big market potential if successful because of the existing unmet medical need.