Ultragenyx, GeneTx: FDA Removes Hold Study of GTX-102 in Angelman Syndrome
September 27 2021 - 9:23AM
Dow Jones News
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
September 27, 2021 09:08 ET (13:08 GMT)
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