Following Disappointing Phase 3 Results, Ultragenyx Discontinues Ace-ER Program for GNEM/HIBM

Ultragenyx Pharmaceutical has announced that a phase 3 study to evaluate aceneuramic acid extended release (brand name Ace-ER), also known as sialic acid extended release, or SA-ER, for the treatment of GNE myopathy (GNEM), also known as hereditary inclusion-body myopathy (HIBM) and Nonaka myopathy, failed to meet its primary and key secondary endpoints.

The study was designed to collect information on the safety and efficacy of Ace-ER in people with GNEM. Results showed no significant difference in the upper extremity muscle strength of those treated with Ace-ER as compared with those who received a placebo.

Based on these results, Ultragenyx has said it will not file with the U.S. Food and Drug Administration for approval of Ace-ER and will discontinue further clinical development of the drug.

“We are disappointed by these results, as we had hoped that Ace-ER would offer a new option for GNEM patients,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We would like to thank the patients, caregivers, and investigators involved in the Ace-ER development program.”

The company reported it will work with investigators and patient groups to make available the valuable natural history data and development tools associated with the trial for the development of other therapies.

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Ultragenyx has said it will consult with investigators and patients to determine a reasonable transition plan for those still taking Ace-ER.

If you participated in the Ace-ER trial, be sure to speak with your physician about any questions you may have regarding your trial experience and treatment plan going forward.