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# Ultragenyx’ Ace-ER Fails Phase 3 Study
- URL: https://www.fdaweb.com/ultragenyx-ace-er-fails-phase-3-study/
- Published: 2017-08-23T12:00:00.000Z
- Updated: 2026-09-14T22:42:56.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5139461

Ultragenyx says its aceneuramic acid extended release (Ace-ER) drug did not achieve its primary endpoint in a Phase 3 clinical trial in patients with GNE myopathy. The company says it is terminating its development program based on the Phase 3 results. The primary endpoint called for the therapy to demonstrate a statistically significant difference in an upper extremity muscle composite score compared to placebo. Officials say the study also did not meet its key secondary endpoints. Adverse events reportedly were generally balanced between Ace-ER and placebo, and safety was consistent with previously released Ace-ER data.