Due to the negative results of the Phase III trial of sialic acid extended-release in GNE myopathy, Ultragenyx will halt the development of this molecule.
For several years now, Ultragenyx has been working on the development of a sialic acid extended-release tablets for the treatment of GNE myopathy, distal myopathies related to abnormalities of an enzyme involved in the synthesis of sialic acid. The long-term goal of this formulation is to raise the level of sialic acid, which is deficient in patients with GNE myopathy.
Despite positive results of sialic acid extended-release obtained in preclinical and Phase II studies, the results of the Phase III trial of sialic acid extended-release in patients with GNE myopathy (HIBM -Phase III UX001-CL301) were negative: the primary endpoint evaluating upper extremity muscle strength was not achieved and the three key secondary endpoints (lower extremity muscle strength, physical functioning, muscle strength in knee extensors) were not met. This international trial (including France) which enrolled 89 adults with GNE myopathy who could walk at least 200 meters, will be stopped over the coming months. For each participant, the trial will be stopped at the end of a final study visit.
However, Ultragenyx is continuing studies on GNE myopathy. The two natural history studies in progress, one international that includes France, and the other, a complementary study taking place only in France, ClinBio-GNE, will continue as planned.
> Further information on this Phase III trial may be available at the next World Muscle Society conference held in Saint Malo from October 3rd to 7th (a poster presentation about this trial is planned).
> If you have any questions, please contact the principal investigators of the trial in France, Dr A. Behin (a.behin@institut-myologie.org) or Dr T. Gidaro (t.gidaro@institut-myologie.org).
> Read the press release from Unltragenyx “Ultragenyx Announces Top-Line Results from Phase 3 Study of Ace-ER in GNE Myopathy”
