{"id":35861,"date":"2026-03-20T16:48:09","date_gmt":"2026-03-20T16:48:09","guid":{"rendered":"https:\/\/www.europesays.com\/ch\/35861\/"},"modified":"2026-03-20T16:48:09","modified_gmt":"2026-03-20T16:48:09","slug":"roche-stops-work-on-experimental-sma-drug","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/ch\/35861\/","title":{"rendered":"Roche stops work on experimental SMA drug"},"content":{"rendered":"\n

This story was originally published on BioPharma Dive<\/a>. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter<\/a>. <\/p>\n

Roche, citing discouraging clinical trial results, has chosen to stop developing an experimental antibody drug as a potential treatment for two rare, muscle-wasting diseases.<\/p>\n

Known as emugrobart, the drug is meant to lower levels of a protein, \u201cmyostatin,\u201d that both breaks down muscle fibers and restricts new muscle from building. Chugai Pharmaceutical, a Japan-based Roche subsidiary, designed it to bind to, and thereby keep inactive, a precursor protein to myostatin. Researchers have been testing emugrobart against spinal muscular atrophy and facioscapulohumeral muscular dystrophy, and as a way to retain lean muscle mass in people with obesity who are taking a GLP-1 medication.<\/p>\n

In SMA, Roche has been sponsoring a two-part trial to evaluate whether it\u2019s worth adding emugrobart on top of the company\u2019s already approved therapy Evrysdi<\/a>. The experiment aimed to recruit around 250 patients, and focused on safety as well as biological markers of muscle health.<\/p>\n

But, after a \u201ccomprehensive assessment\u201d of all the evidence gathered in the first part of the experiment, the Swiss pharmaceutical giant determined its drug \u201cdid not consistently deliver\u201d enough improvements in muscle growth and motor function to warrant further study.<\/p>\n

\u201cWe recognise this news will be disappointing to the SMA community,\u201d wrote Louisa Townson, Roche\u2019s patient partnership director for rare diseases, in a Thursday letter<\/a> to a European patient advocacy group, explaining the company\u2019s decision.<\/p>\n

Townson added that Roche \u201cremains committed to advancing quality care for people living with SMA,\u201d and plans to share data from the trial at an upcoming medical conference \u201cso that this research can also help to inform the development of future treatments.\u201d She penned a similar letter<\/a> to the facioscapulohumeral muscular dystrophy community, referring to a separate mid-stage trial<\/a> that also generated less-than-ideal results.<\/p>\n

While estimates vary, the number of people living with SMA in the U.S. is generally thought to be in the range of 10,000<\/a> to around 30,000<\/a>. It mostly affects young children, and is caused by alterations in a gene responsible for making \u201csurvival motor neuron,\u201d a protein crucial for signaling between muscles and the nervous system.<\/p>\n

While historically fatal, the prognosis for this disease changed drastically over the past decade as three medicines \u2014 Biogen\u2019s antisense drug Spinraza, Novartis\u2019 gene therapy Zolgensma and Evrysdi \u2014 became available to patients. Each of these works by boosting survival motor neuron levels.<\/p>\n

Last year, sales of Evrysdi, which is taken orally, totaled 1.76 billion Swiss francs<\/a>, or roughly $2.1 billion. Roche had been hoping to expand the business with emugrobart, which, by targeting myostatin, offered a different route to addressing SMA. It\u2019s a strategy adopted by other developers as well, including Biohaven<\/a> and Massachusetts-based Scholar Rock<\/a>.<\/p>\n

Eric Schmidt, an analyst at Cantor Fitzgerald who covers Scholar Rock, highlighted how its drug apitegromab has been ahead of Roche\u2019s for several years, and is now being developed as both an under-the-skin shot along with a monthly intravenous infusion. Roche, therefore, \u201coffered little in the way of differentiation,\u201d according to Schmidt.<\/p>\n

\u201cWe now expect apitegromab to have near complete market share as the lone muscle-building therapy for SMA patients,\u201d the analyst wrote in a Thursday note to clients, adding that his team sees an approval from the Food and Drug Administration this year as a “when not if” event. The Cantor team expects the therapy, which the FDA rejected last year due to manufacturing concerns<\/a>,\u00a0to have multibillion-dollar potential.<\/p>\n

Roche\u2019s update is a \u201cclear win\u201d for Scholar Rock ahead of a \u201clikely FDA approval,\u201d wrote Raymond James analyst Martin Auster in his own note to clients.<\/p>\n

Shares of Scholar Rock were up 1.5%, to a little over $46 apiece, in late morning trading Friday. Biohaven shares were up 2.5%.<\/p>\n

Recommended Reading <\/p>\n","protected":false},"excerpt":{"rendered":"This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free…\n","protected":false},"author":2,"featured_media":35862,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":"","_share_on_mastodon":"0"},"categories":[124],"tags":[21230,2421,134,21232,21231,9913],"class_list":["post-35861","post","type-post","status-publish","format-standard","has-post-thumbnail","category-roche","tag-muscular-dystrophy","tag-rare-diseases","tag-roche","tag-scholar-rock","tag-sma-patients","tag-spinal-muscular-atrophy"],"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@ch\/116262528786532406","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts\/35861","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/comments?post=35861"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts\/35861\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/media\/35862"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/media?parent=35861"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/categories?post=35861"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/tags?post=35861"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}