Ultragenyx receives FDA IND clearance for UX016 in GNE myopathy; Phase 1/2 to start H2 2026

Ultragenyx Pharmaceutical Inc.

FDA cleared IND for UX016, a prodrug of sialic acid for GNE myopathy, a rare inherited neuromuscular disorder.
UX016 is a small molecule prodrug designed to enhance sialic acid delivery to muscle cells.
Phase 1/2 study planned for H2 2026, enrolling ~24 adults aged 18–55 in the US; externally funded by a patient group.
Study will evaluate safety, pharmacokinetics, and efficacy including muscle strength and functional measures through 48 weeks.

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