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Ultragenyx Halts Ace-ER Development After Phase III Failure

05:40 EDT 23 Aug 2017 | Genetic Engineering News

Ultragenyx plans to halt development of aceneuramic acid (sialic acid) extended release (Ace-ER) after the GNE Myopathy (GNEM) candidate failed a Phase III trial. According to topline results disclosed by Ultragenyx yesterday, Ace-ER did not achieve its primary endpoint of demonstrating a statistically significant difference in the upper extremity muscle strength composite score (UEC) compared to placebo in adults with GNEM following 48 weeks of treatment. Without disclosing details, Ultragenyx added that the Phase III study also did not meet its three key secondary endpoints—lower extremity muscle strength composite score as measured by hand-held dynamometry (HHD); physical functioning using the Mobility domain of the GNE Myopathy-functional activity scale (GNEM-FAS); and muscle strength in knee extensors. The Phase III Ace-ER study enrolled 89 adults with GNEM who were able to walk 200 or more meters in the six-minute walk test. Patients were randomized 1:1 to Ace-ER at ...

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