Ultragenyx falls after Phase III failure for rare disease candidate
Shares in Californian rare disease specialist Ultragenyx Pharmaceutical (Nasdaq: RARE) have dropped off nearly a fifth after the firm announced the failure of a Phase III study evaluating UX007 in patients with glucose transporter type-1 deficiency syndrome (Glut1 DS).
The firm said it would stop developing the candidate in this indication, after the trial failed to meet both primary and secondary endpoints.
Meanwhile a separate program evaluating UX007 in long-chain fatty oxidation disorders (LC-FAOD) will continue. The firm plans to file with the US regulator in this indication, based on existing data.
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