Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said it will discontinue development of UX007 to treat glucose transporter type-1 deficiency syndrome (Glut1 DS; De Vivo disease) after the therapy missed the primary and key secondary endpoints in a Phase III trial. Ultragenyx lost $10.06 (17%) to $49.30 in morning trading Friday.
The trial's primary endpoint measured the change from baseline in the frequency of disabling paroxysmal movement disorder events at week 22. Secondary endpoints included the duration of those disabling events; walking capacity and endurance; and patient-reported quality of life assessments. The double-blind, crossover trial enrolled 44 patients to receive UX007 followed by placebo, or placebo followed by UX007.
UX007 is a formulation of triheptanoin, a synthetic compound providing medium-length, odd-chain fatty acids that the body metabolizes to replace intermediates in fatty acid oxidation and in the tricarboxylic acid (TCA) cycle. There are no approved drugs for Glut1 DS, which the company estimates has a prevalence of 12,000-28,000 patients worldwide.
Ultragenyx said a separate program evaluating UX007 to treat long-chain fatty oxidation disorders (LC-FAOD) is unaffected by the failure in Glut1 DS. The company expects to hold a pre-NDA meeting with FDA by year end, and anticipates submitting an NDA next year based on existing data (see "FDA Clarifies Path to Submission for Ultragenyx's Rare Disease Therapy").
The company has been endeavoring to develop a series of drugs for ultra-rare disorders (see "Making of Mepsevii").
With Friday morning's stock move, Ultragenyx's market cap dipped below $2.5 billion. The company's shares are off 46% from their 52-week high of $90.98, reached Sept. 25 intraday.
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