Ultragenyx Pharmaceutical (RARE) announced that the FDA has cleared the Investigational New Drug application for UX016, an investigational small molecule prodrug of sialic acid being evaluated as a substrate replacement therapy for GNE myopathy. The planned first-in-human, Phase 1/2 study will enroll approximately 24 adults ages 18 to 55 years with GNEM. The study, expected to begin in the second half of 2026, will evaluate the safety and efficacy of UX016.
The UX016 program is externally funded by a patient group through clinical proof-of-concept, including a Phase 1/2 study expected to begin in the second half of 2026.
In pre-market trading on NasdaqGS, Ultragenyx shares are up 0.26 percent to $19.43.
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Business News
May 15, 2026 15:25 ET Apart from the confirmation of Kevin Warsh as the next Fed chair, the main news on the economics front this week included key price data from the U.S. and the first quarter economic growth figures from major economies. Both consumer prices and producer costs have started to reflect the effect of supply shocks due to the Middle East conflict. In Europe, GDP data was in focus, while inflation data from China dominated the news flow in Asia.