=Ultragenyx (RARE) : muscle-disease drug study fails

Ultragenyx Pharma announces its Phase 3 study evaluating aceneuramic acid extended release in patients with GNE Myopathy did not achieve its primary endpoint; co will discontinue further development --halted--(58.85 +1.94)
The Phase 3 study evaluating aceneuramic acid extended release (Ace-ER) in patients with GNE Myopathy (GNEM) did not achieve its primary endpoint of demonstrating a statistically significant difference in the upper extremity muscle strength composite score compared to placebo. The study also did not meet its key secondary endpoints. Adverse events were generally balanced between Ace-ER and placebo and safety was consistent with previously released Ace-ER data. Ultragenyx plans to discontinue further clinical development of Ace-ER.