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MarketScreener Homepage  >  Equities  >  Nasdaq  >  Ultragenyx Pharmaceutical Inc    RARE

ULTRAGENYX PHARMACEUTICAL INC (RARE)
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Ultragenyx Pharmaceutical : UX007 fails Phase III in glucose transporter disorder

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10/29/2018 | 09:33am CET

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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(C) 2018 M2 COMMUNICATIONS, source M2 PressWIRE

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Financials ($)
Sales 2018 49,7 M
EBIT 2018 -376 M
Net income 2018 -202 M
Finance 2018 291 M
Yield 2018 -
P/E ratio 2018 -
P/E ratio 2019
EV / Sales 2018 43,4x
EV / Sales 2019 21,5x
Capitalization 2 447 M
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Mean consensus OUTPERFORM
Number of Analysts 20
Average target price 70,5 $
Spread / Average Target 46%
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NameTitle
Emil D. Kakkis President, Chief Executive Officer & Director
Daniel G. Welch Non-Executive Chairman
Wladimir Hogenhuis Chief Operating Officer
Shalini Sharp Chief Financial Officer & Executive Vice President
Dennis Karl Huang Chief Technical Operations Officer & Senior VP
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