Biogen to Advance SMA Treatment Into Registrational Studies

Dow Jones

2025/06/25

By Colin Kellaher

Biogen plans to advance an experimental treatment for the neuromuscular disease spinal muscular atrophy, or SMA, into registrational studies following encouraging results from an early-stage trial.

Biogen on Wednesday said interim data from a Phase 1 study of salanersen show children with SMA previously treated with gene therapy experienced a substantial slowing of neurodegeneration and clinically meaningful improvements in motor function following initiation of the drug.

The Cambridge, Mass., biotechnology company said that based on the encouraging data, it is engaging with regulators to advance salanersen to registrational-stage studies.

Biogen said salanersen uses the same mechanism of action as its Spinraza drug for SMA but is designed to achieve greater potency, giving it the potential to achieve high efficacy and enable once-yearly dosing.

Biogen licensed the global development, manufacturing and commercialization rights for salanersen from Ionis Pharmaceuticals, which discovered the drug. Biogen and Ionis are also partners on Spinraza.

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

(END) Dow Jones Newswires

June 25, 2025 07:57 ET (11:57 GMT)

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Biogen and Ionis are also partners on Spinraza. \n</p>\n<pre>\n \n</pre>\n<p>\n  Write to Colin Kellaher at colin.kellaher@wsj.com \n</p>\n<pre>\n \n</pre>\n<p>\n  (END) Dow Jones Newswires\n</p>\n<p>\n  June 25, 2025 07:57 ET (11:57 GMT)\n</p>\n<p>\n  Copyright (c) 2025 Dow Jones &amp; Company, Inc.\n</p>\n</font>\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"IE00B19Z9Z06.USD","symbol_name":"Legg Mason ClearBridge - US Aggressive Growth A Acc USD","start_time":0,"source_url":"https://dowjonesnews.com/newdjn/logon.aspx?AL=N","article_id":"2546212044","we_media_id":"106","thumbnails":[],"rights":null,"url":"https://stock-news.laohu8.com/highlight/detail?id=2546212044","pubTimestamp":1750852620,"columns":[],"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":"Biogen plans to advance an experimental treatment for the neuromuscular disease spinal muscular atrophy, or SMA, into registrational studies following encouraging results from an early-stage trial.Biogen on Wednesday said interim data from a Phase 1 study of salanersen show children with SMA previously treated with gene therapy experienced a substantial slowing of neurodegeneration and clinically meaningful improvements in motor function following initiation of the drug.The Cambridge, Mass., biotechnology company said that based on the encouraging data, it is engaging with regulators to advance salanersen to registrational-stage studies.Biogen said salanersen uses the same mechanism of action as its Spinraza drug for SMA but is designed to achieve greater potency, giving it the potential to achieve high efficacy and enable once-yearly dosing.Biogen licensed the global development, manufacturing and commercialization rights for salanersen from Ionis Pharmaceuticals, which discovered the","collect":0,"end_time":0,"defaultTopTitle":"","property":[],"viewcount":null,"language":"en","relate_stocks":{"IE00B19Z9Z06.USD":"Legg Mason ClearBridge - US Aggressive Growth A Acc USD","LU0109394709.USD":"富兰克林生物科技新领域基金A (acc)","BK4585":"ETF&股票定投概念","BK4139":"生物科技","BK4532":"文艺复兴科技持仓","IE00B19Z9P08.USD":"LEGG MASON CLEARBRIDGE US AGGRESSIVE GROWTH \"A\" (USD) INC","LU0234570918.USD":"高盛全球核心股票组合Acc Close","BK4533":"AQR资本管理(全球第二大对冲基金)","IE00B894F039.SGD":"Legg Mason ClearBridge - US Aggressive Growth A Acc SGD-H","LU0889565916.HKD":"FRANKLIN BIOTECHNOLOGY DISCOVERY \"A\" (HKD) ACC","IONS":"Ionis Pharmaceuticals, Inc","LU0320765992.SGD":"FTIF - Franklin Biotechnology Discovery A Acc SGD","SMA":"Smartstop Self Storage REIT Inc","BK4588":"碎股","BIIB":"渤健公司"},"translate_title":"Biogen将SMA治疗推进注册研究","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"IONS":0.9,"SMA":1,"BIIB":1},"content_text":"By Colin Kellaher \n\n\n \n\n\n  Biogen plans to advance an experimental treatment for the neuromuscular disease spinal muscular atrophy, or SMA, into registrational studies following encouraging results from an early-stage trial. \n\n\n  Biogen on Wednesday said interim data from a Phase 1 study of salanersen show children with SMA previously treated with gene therapy experienced a substantial slowing of neurodegeneration and clinically meaningful improvements in motor function following initiation of the drug. \n\n\n  The Cambridge, Mass., biotechnology company said that based on the encouraging data, it is engaging with regulators to advance salanersen to registrational-stage studies. \n\n\n  Biogen said salanersen uses the same mechanism of action as its Spinraza drug for SMA but is designed to achieve greater potency, giving it the potential to achieve high efficacy and enable once-yearly dosing. \n\n\n  Biogen licensed the global development, manufacturing and commercialization rights for salanersen from Ionis Pharmaceuticals, which discovered the drug. 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