PhIII bust forces Ultragenyx to scrap drug for rare muscle disease
Analysts had been speculating that a success in Ultragenyx’s Phase III study of Ace-ER for rare cases of GNE myopathy could trigger some near-term regulatory filings. Now, they’re being forced to write it off completely as investigators report that the drug failed the primary and secondary endpoints, spurring the biotech to write it off completely.
The primary goal for the Phase III study of Aceneuramic Acid Prolonged Release, which enrolled more than 80 patients, was a composite test for upper extremity muscles. The secondaries also focused on muscle strength as well. But after reporting what it terms encouraging mid-stage data, the therapy flunked the pivotal test.
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