{"id":82235,"date":"2026-06-12T09:10:10","date_gmt":"2026-06-12T09:10:10","guid":{"rendered":"https:\/\/www.europesays.com\/ch\/82235\/"},"modified":"2026-06-12T09:10:10","modified_gmt":"2026-06-12T09:10:10","slug":"novartis-dystrophy-data-tee-up-regulatory-talks-on-avidity-drug","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/ch\/82235\/","title":{"rendered":"Novartis dystrophy data tee up regulatory talks on Avidity drug"},"content":{"rendered":"<p dir=\"ltr\">Novartis has\u00a0<a href=\"https:\/\/www.novartis.com\/news\/media-releases\/novartis-delpacibart-braxlosiran-del-brax-phase-iii-study-facioscapulohumeral-muscular-dystrophy-fshd-meets-primary-biomarker-endpoint\" rel=\"nofollow noopener\" target=\"_blank\">reported<\/a> a phase 1\/2 win for one of the jewels of its $12 billion Avidity Biosciences buyout, setting the Swiss drugmaker up to talk to global regulatory agencies about the next steps for its dystrophy prospect.<\/p>\n<p dir=\"ltr\">Back in April, Novartis CEO Vas Narasimhan, M.D.,\u00a0<a href=\"https:\/\/edge.media-server.com\/mmc\/p\/652bb6yc\/\" rel=\"nofollow noopener\" target=\"_blank\">told<\/a> investors that the company planned to talk to the FDA about seeking accelerated approval based on the phase 1\/2 data. The pharma has already started a phase 3 trial of the candidate, the antibody-oligonucleotide conjugate (AOC) delpacibart braxlosiran (del-brax), but has spied a chance to come to market before that trial wraps up.<\/p>\n<p dir=\"ltr\">Novartis\u2019 topline phase 1\/2 data take del-brax a step toward commercialization in facioscapulohumeral muscular dystrophy (FSHD). The 51-subject biomarker cohort hit its primary endpoint, which looked at change in the plasma concentration of KHDC1.<\/p>\n<p dir=\"ltr\">KHDC1 is a circulating biomarker regulated by DUX4. In FSHD, abnormal expression of DUX4 leads to changes in gene expression associated with the lifelong, progressive loss of muscle function. Del-brax combines an siRNA targeting DUX4 mRNA with an anti-TfR1 antibody. The antibody delivers the payload to muscle cells to reduce DUX4 mRNA in target tissues, addressing the underlying cause of FSHD.<\/p>\n<p dir=\"ltr\">Novartis\u2019 phase 3 study, which\u00a0<a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07038200\" rel=\"nofollow noopener\" target=\"_blank\">started<\/a> last year, is evaluating whether the mechanism improves muscle strength and other clinical outcomes through Week 78. That study will be the acid test of del-brax. But the phase 1\/2 data suggest the AOC has the intended biological effects. Novartis reported the drop in KHDC1 alongside a fall in creatine kinase, suggesting target engagement and reduced muscle damage.<\/p>\n<p dir=\"ltr\">It remains to be seen whether the FDA is open to granting accelerated approval based on the phase 1\/2 data. Either way, Narasimhan expects del-brax to be the first drug to market in FSHD, reflecting Novartis\u2019 position at the front of a pack of companies that includes Sarepta Therapeutics.<\/p>\n<p dir=\"ltr\">Sarepta\u00a0<a href=\"https:\/\/www.fiercebiotech.com\/biotech\/sareptas-big-bet-arrowheads-sirna-assets-yields-early-data\" rel=\"nofollow noopener\" target=\"_blank\">posted<\/a> initial clinical results on its FSHD candidate, SRP-1001, in March. The data on the siRNA drug, which Sarepta\u00a0<a href=\"https:\/\/www.fiercebiotech.com\/biotech\/sarepta-pays-500m-cash-multiple-potential-blockbusters-arrowhead\" rel=\"nofollow noopener\" target=\"_blank\">picked up<\/a> from Arrowhead Pharmaceuticals in a deal worth $500 million upfront, impressed TD Cowen analysts. Writing in a note to investors in March, the analysts said the data support SRP-1001\u2019s \u201cpotentially superior biomarker efficacy\u201d compared to del-brax.\u00a0<\/p>\n","protected":false},"excerpt":{"rendered":"Novartis has\u00a0reported a phase 1\/2 win for one of the jewels of its $12 billion Avidity Biosciences buyout,&hellip;\n","protected":false},"author":2,"featured_media":82236,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":"","_share_on_mastodon":"0"},"categories":[127],"tags":[1115,16594,9275,21230,206,2421,7091],"class_list":["post-82235","post","type-post","status-publish","format-standard","has-post-thumbnail","category-novartis","tag-biotech","tag-clinical-data","tag-fierce-biotech-homepage","tag-muscular-dystrophy","tag-novartis","tag-rare-diseases","tag-sirna"],"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@ch\/116736362118587097","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts\/82235","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=82235"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/posts\/82235\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/media\/82236"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/media?parent=82235"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/categories?post=82235"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/ch\/wp-json\/wp\/v2\/tags?post=82235"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}