Ultragenyx stock drops 14% on late-stage trial failure

Ultragenyx Pharmaceutical Inc. [s:RARE] shares dropped 14% in Friday premarket trade on news that its therapy UX007 failed to meet the primary endpoint in a phase 3 clinical trial. UX007 was being tested in patients with a rare disease called glucose transporter type-1 deficiency syndrome, but did not show a statistically significant reduction in the number of disabling movement disorders relative to the placebo, the phase 3 trial’s primary endpoint, the company said. The trial, which enrolled 44 children and adults with the disease, also did not meet key secondary endpoints. Ultragenyx plans to discontinue development of UX007 for the condition, though it will continue to develop the drug for another disease, long-chain fatty oxidation disorders. Ultragenyx, founded in 2010, has two approved medications on the market, but UX007 is a key component of its pipeline. The company is also testing its medication Crysvita in tumor-induced osteomalacia, and it has two gene therapy programs in phase 1 development, according to its pipeline. Ultragenyx shares have dropped 26.1% over the last three months to $59.36, compared with a 4.7% decline in the S&P 500 SPX, +1.86% and a 2.1% drop in the Dow Jones Industrial Average DJIA, +1.63%

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