Ultragenyx Pharmaceutical Inc. announced that the FDA has cleared its Investigational New Drug application for UX016, a small molecule prodrug of sialic acid being developed as a substrate replacement therapy for GNE myopathy, a rare inherited neuromuscular disorder.
The UX016 program is externally funded by a patient group through clinical proof-of-concept, including a planned first-in-human Phase 1/2 study in the United States. This trial, expected to start in the second half of 2026, will enroll about 24 adults with GNE myopathy to evaluate safety, efficacy, pharmacokinetics, and muscle delivery over up to 48 weeks of treatment.
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