UniQure Down in Premarket as FDA Says Huntington's Gene-Therapy Data Falls Short

Dow Jones

03/02

By Adriano Marchese

UniQure shares fell in premarket trading Monday after the Food and Drug Administration said the company's data isn't strong enough to show its Huntington's disease gene therapy is effective.

Shares traded 42% lower ahead of the morning bell at $9.

The gene-therapy company said Monday that the FDA stated that it can't agree that data from the Phase I/II studies, compared with an external control, provide enough primary evidence of effectiveness required to support a marketing application for the drug AMT-130.

Huntington's disease is an inherited and progressive neurodegenerative disorder that is caused by faulty genes triggering the breakdown of nerve cells in the brain.

UniQure said the regulator is urging the company to run a more rigorous trial that includes randomized, double-blind, sham surgery-controlled study.

The company said it intends to keep working with the FDA on what a Phase III program should look like and plans to request a type B meeting in the second quarter of 2026 to discuss possible study designs.

Write to Adriano Marchese at adriano.marchese@wsj.com

(END) Dow Jones Newswires

March 02, 2026 07:58 ET (12:58 GMT)

Copyright (c) 2026 Dow Jones & Company, Inc.

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Company, Inc.\n</p>\n</font>\n\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"LU0109394709.USD","symbol_name":"富兰克林生物科技新领域基金A (acc)","start_time":0,"source_url":"https://dowjonesnews.com/newdjn/logon.aspx?AL=N","article_id":"2616332185","we_media_id":"106","thumbnails":[],"rights":null,"url":"https://stock-news.laohu8.com/highlight/detail?id=2616332185","pubTimestamp":1772456280,"columns":[{"name":"行业数据","id":30,"label_key":"dataReport"}],"sourceInfo":null,"weMediaInfo":{"media_name":"Dow Jones","introduction":"Dow Jones publishes the world’s most trusted business news and financial information in a variety of media.","home_visible":0,"id":"106","head_image":"https://static.tigerbbs.com/150f88aa4d182df19190059f4a365e99"},"summary":"UniQure shares fell in premarket trading Monday after the Food and Drug Administration said the company's data isn't strong enough to show its Huntington's disease gene therapy is effective.Shares traded 42% lower ahead of the morning bell at $9.The gene-therapy company said Monday that the FDA stated that it can't agree that data from the Phase I/II studies, compared with an external control, provide enough primary evidence of effectiveness required to support a marketing application for the drug AMT-130.Huntington's disease is an inherited and progressive neurodegenerative disorder that is caused by faulty genes triggering the breakdown of nerve cells in the brain.UniQure said the regulator is urging the company to run a more rigorous trial that includes randomized, double-blind, sham surgery-controlled study.The company said it intends to keep working with the FDA on what a Phase III program should look like and plans to request a type B meeting in the second quarter of 2026 to disc","collect":0,"end_time":0,"defaultTopTitle":"","property":[],"viewcount":null,"language":"en","relate_stocks":{"LU0109394709.USD":"富兰克林生物科技新领域基金A (acc)","BK4139":"生物科技","LU0320765992.SGD":"FTIF - Franklin Biotechnology Discovery A Acc SGD","QURE":"uniQure N.V.","LU0889565916.HKD":"FRANKLIN BIOTECHNOLOGY DISCOVERY \"A\" (HKD) ACC"},"translate_title":"FDA称亨廷顿基因治疗数据不足，UniQure上市前下跌","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"QURE":0.9},"content_text":"By Adriano Marchese \n\n\n \n\n\n  UniQure shares fell in premarket trading Monday after the Food and Drug Administration said the company's data isn't strong enough to show its Huntington's disease gene therapy is effective. \n\n\n  Shares traded 42% lower ahead of the morning bell at $9. \n\n\n  The gene-therapy company said Monday that the FDA stated that it can't agree that data from the Phase I/II studies, compared with an external control, provide enough primary evidence of effectiveness required to support a marketing application for the drug AMT-130. \n\n\n  Huntington's disease is an inherited and progressive neurodegenerative disorder that is caused by faulty genes triggering the breakdown of nerve cells in the brain. \n\n\n  UniQure said the regulator is urging the company to run a more rigorous trial that includes randomized, double-blind, sham surgery-controlled study. \n\n\n  The company said it intends to keep working with the FDA on what a Phase III program should look like and plans to request a type B meeting in the second quarter of 2026 to discuss possible study designs. \n\n\n \n\n\n  Write to Adriano Marchese at adriano.marchese@wsj.com \n\n\n \n\n\n  (END) Dow Jones Newswires\n\n\n  March 02, 2026 07:58 ET (12:58 GMT)\n\n\n  Copyright (c) 2026 Dow Jones & Company, Inc.","kind":"highlight","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":true,"editions":null,"column":"","sentiment":"-1","news_tag":"dataReport","news_rank":0,"isVideo":false,"video":null,"symbols":[],"gpt_button":0,"need_auth":true,"need_login_tip":false,"code":"91000000","status":"200"}}}