{"id":100972,"date":"2026-07-10T09:02:28","date_gmt":"2026-07-10T09:02:28","guid":{"rendered":"https:\/\/www.europesays.com\/ch\/100972\/"},"modified":"2026-07-10T09:02:28","modified_gmt":"2026-07-10T09:02:28","slug":"roche-drops-two-huntingtons-programmes-from-ionis","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/ch\/100972\/","title":{"rendered":"Roche drops two Huntington\u2019s programmes from Ionis"},"content":{"rendered":"

Why it matters: The decisions eliminate two of Roche\u2019s three clinical approaches to lowering the toxic huntingtin protein that drives the neurodegenerative disease, for which no disease-modifying treatment is currently approved. Roche communicated the news in a letter to the patient community<\/a>.<\/p>\n

Zoom in: Roche\u2019s phase 2 GENERATION HD2 trial found that tominersen significantly reduced mutant huntingtin and neurofilament light chain, a marker associated with neuronal damage.<\/p>\n

Yes, but: The treatment had no meaningful effect on clinical outcomes compared with placebo after 16 months, prompting Roche to end the programme.<\/p>\n

Backstory: Roche licensed tominersen from US-based Ionis Pharmaceuticals in 2017. The antisense oligonucleotide had already failed a phase 3 trial in 2021<\/a>, after patients receiving the more frequent dosing regimen performed worse than those receiving placebo.<\/p>\n

GENERATION HD2 was designed to test whether lower and less frequent doses could benefit younger patients at an earlier stage of the disease.<\/p>\n

Meanwhile: Roche has also stopped the phase 1 POINT-HD trial of RG6496, another Ionis-partnered drug, after a longer-term animal study indicated that the antisense drug could not be administered chronically through repeated dosing.<\/p>\n

Only three participants had entered the first-in-human study. Roche said there were no safety concerns associated with the single doses they received.<\/p>\n

What remains: Roche is continuing a phase 1\/2 trial of RG6662, an experimental gene therapy previously known as SPK-10001.<\/p>\n

The programme came from Spark Therapeutics, the US gene therapy company Roche acquired for $4.3bn in 2019. RG6662 uses an adeno-associated virus vector to deliver genetic material intended to reduce production of the mutant huntingtin protein.<\/p>\n

The big picture: The Spark-derived asset now represents Roche\u2019s only remaining clinical Huntington\u2019s programme.<\/p>\n

Competition is also advancing. Dutch biotech uniQure is preparing a US regulatory filing<\/a> for its Huntington\u2019s gene therapy AMT-130 in the third quarter of 2026.
\nUK-based biotech LoQus23 Therapeutics is advancing an oral small molecule targeting MutS\u03b2<\/a>, a protein complex that has emerged as a central driver of disease progression in Huntington\u2019s disease. The company is aiming to file regulatory submissions to initiate clinical testing later this year.<\/p>\n

Reality check: It\u2019s not a good week for Ionis, as its AstraZeneca-partnered drug eplontersen failed to hit the primary endpoint<\/a> in a phase 3 trial in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).<\/p>\n","protected":false},"excerpt":{"rendered":"Why it matters: The decisions eliminate two of Roche\u2019s three clinical approaches to lowering the toxic huntingtin protein…\n","protected":false},"author":2,"featured_media":100973,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":"","_share_on_mastodon":"0"},"categories":[124],"tags":[30409,14275,134],"class_list":["post-100972","post","type-post","status-publish","format-standard","has-post-thumbnail","category-roche","tag-huntingtons-disease","tag-partnering","tag-roche"],"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@ch\/116894875390794021","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts\/100972","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=100972"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts\/100972\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/media\/100973"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/media?parent=100972"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/categories?post=100972"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/tags?post=100972"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}