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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