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Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX016, a Sialic Acid Prodrug for the Treatment of GNE Myopathy

RARE
  • The U.S. FDA cleared the Investigational New Drug application for UX016, an investigational prodrug of sialic acid developed by Ultragenyx Pharmaceutical Inc.
  • UX016 is being evaluated as a substrate replacement therapy for GNE myopathy (GNEM), a rare, debilitating neuromuscular disorder caused by GNE gene mutations.
  • The UX016 program is externally funded by a patient group, enabling a Phase 1/2 clinical study expected to begin in the second half of 2026.
  • UX016 consists of sialic acid linked to a hydrophobic C16 fatty acid tail to enhance delivery to muscle tissue & improve uptake compared to naturally occurring sialic acid.
  • Preclinical data in a GNEM mouse model showed that UX016 increased sialic acid concentrations across multiple skeletal muscles & restored levels toward normal.
  • The planned first-in-human Phase 1/2 study will enroll approximately 24 adults aged 18 to 55 years with GNEM in the United States.
  • The study will evaluate safety, efficacy, pharmacokinetics, & muscle delivery at two doses relative to placebo over 12 weeks, followed by assessments of muscle strength & functional measures through Week 48.
  • GNEM affects approximately 10,000 people in commercially accessible geographies & currently has no approved therapies in the United States.
  • Ultragenyx is committed to developing novel therapies for rare genetic diseases using a time- & cost-efficient development strategy.