Ultragenyx Shares Down 8.6% After Reporting Adverse Events in Genetic Therapy Study

Dow Jones04-15

By Zaeem Shoaib

Ultragenyx Pharmaceutical's shares dropped after the company reported observing side effects in some patients in a phase 1/2 study of GTX-102, a treatment for a genetic disorder that affects the nervous system.

Shares were down 8.6% to $42.53 in late morning trading on Monday. The stock is down 10% this year.

Ultragenyx said patients with Angelman syndrome in two cohorts treated with a set dose and regimen of GTX-102 showed rapid and clinically meaningful improvement across multiple domains consistent with or exceeding data from dose-escalation cohorts 4-7 at day 170.

The company said the treatment in dose-escalation cohorts 4-7 showed long-term increasing and sustained clinical benefit that exceeded natural history data at day 758.

There were no unexpected serious adverse events, the company said. However, three patients had serious adverse events of lower extremity weakness, which were assessed as related to study treatment.

Ultragenyx said the adverse events resolved rapidly without complications and patients remain in the study without ongoing safety concerns. The company said it notified the Food and Drug Administration and other regulatory agencies of all safety events and they raised no issues or required additional actions.

"Our next step is an end of Phase 2 meeting with the FDA and interactions with other health authorities to enable timely initiation of a Phase 3 pivotal study," Chief Medical Officer Eric Crombez said.

Write to Zaeem Shoaib at zaeem.shoaib@wsj.com

(END) Dow Jones Newswires

April 15, 2024 11:56 ET (15:56 GMT)

Copyright (c) 2024 Dow Jones & Company, Inc.

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