Ultragenyx Announced UX001 Phase 1 study for HIBM

Ultragenyx Pharmaceutical Inc., a biotechnology firm concentrated on improving therapies for uncommon and ultra-rare genetic problems, announced the Phase 1 evaluation of UX001 for hereditary inclusion body myopathy (HIBM) has been finished. UX001 is definitely an extended launch formulation of sialic acid planned being a substrate alternative therapy for HIBM, an acute, neuromuscular disorder caused by sialic acid shortfall. UX001 will be the first program coming from the company pipeline to input the clinic ever since its founding in 2010. The FDA Office of Orphan Products Development has approved orphan drug designation for UX001 for treatment of HIBM.

Emil D. Kakkis, MD, PhD, and Chief Executive Officer of Ultragenyx said, "Ultragenyx is continuing to make superb improvement in advancing and increasing our clinical progress pipeline together with the conclusion of our first clinical study." "We profoundly appreciate the involvement and enthusiastic help of the HIBM affected person community for our particular Phase 1 study. We look forward to releasing the outcomes out of this study over the following couple of months, and can look forward to presenting the results at a scientific encounter within the fall. We have plans to initiate a Phase 2 trial of SA-ER in patients by using HIBM inside the second quarter of the year."

The Phase 1 clinical study examined the pharmacokinetics (PK) and overall safety of UX001 in 28 HIBM affected individuals. The study examined five different single-dose stages in each group of six topics. Subjects in that case underwent repeat dosing at four dose level over seven days to set up the steady-state pharmacokinetics and safety of replicate doses of UX001. Ultragenyx anticipates files away from the Phase 1 survey in April 2012.