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ULTRAGENYX PHARMACEUTICAL INC.

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Ultragenyx, GeneTx : FDA Removes Hold Study of GTX-102 in Angelman Syndrome

09/27/2021 | 07:08am EDT

By Colin Kellaher

Ultragenyx Pharmaceutical Inc. and GeneTx Biotherapeutics LLC on Monday said the U.S. Food and Drug Administration has removed the clinical hold on a study of GTX-102 for the treatment of the genetic disorder Angelman syndrome.

The companies said GeneTx may now begin dosing naive patients in a phase 1/2 open-label, multiple-dose study evaluating the safety, tolerability, and plasma and cerebrospinal fluid concentrations of GTX-102 in pediatric patients.

The FDA late last year put the study on hold after all five patients in the program developed lower extremity weakness at the highest doses.

The adverse events were assessed as mild or moderate in severity, and all fully resolved, and the companies later filed an amended dosing and administration plan with the FDA.

Ultragenyx, a Novato, Calif., biopharmaceutical company, formed a partnership with startup biotechnology company GeneTx in August 2019 to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

09-27-21 0908ET

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Financials (USD)
Sales 2021 354 M - -
Net income 2021 -455 M - -
Net cash 2021 606 M - -
P/E ratio 2021 -11,7x
Yield 2021 -
Capitalization 5 318 M 5 318 M -
EV / Sales 2021 13,3x
EV / Sales 2022 12,2x
Nbr of Employees 893
Free-Float 95,5%
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Emil D. Kakkis President, Chief Executive Officer & Director
Mardi C. Dier Chief Financial Officer & Executive Vice President
Daniel G. Welch Non-Executive Chairman
Dennis Karl Huang Chief Technical Operations Officer & Senior VP
Camille L. Bedrosian Chief Medical Officer & Executive Vice President
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