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Re: DewDiligence post# 210039

Tuesday, 08/22/2017 4:24:30 PM

Tuesday, August 22, 2017 4:24:30 PM

Post# of 251620
RARE terminates GNE-myopathy program following phase-3 failure:

https://globenewswire.com/news-release/2017/08/22/1091258/0/en/Ultragenyx-Announces-Top-Line-Results-from-Phase-3-Study-of-Ace-ER-in-GNE-Myopathy.html

Ultragenyx Pharmaceutical…today announced that a 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… Ultragenyx plans to discontinue further clinical development of Ace-ER.

The stock is currently halted. No CC.

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