{"id":5101,"date":"2025-11-04T16:49:31","date_gmt":"2025-11-04T13:49:31","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5101"},"modified":"2025-11-04T17:01:51","modified_gmt":"2025-11-04T14:01:51","slug":"solid-biosciences-provides-update-on-inspire-duchenne-clinical-trial-progress-and-planned-regulatory-discussions","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/solid-biosciences-provides-update-on-inspire-duchenne-clinical-trial-progress-and-planned-regulatory-discussions\/","title":{"rendered":"Solid Biosciences, INSPIRE DUCHENNE Klinik Ara\u015ft\u0131rma \u0130lerlemesi ve Planlanan D\u00fczenleyici Tart\u0131\u015fmalar Hakk\u0131nda G\u00fcncelleme Sa\u011fl\u0131yor"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Solid Biosciences, Faz 1\/2 INSPIRE DUCHENNE klinik \u00e7al\u0131\u015fmas\u0131ndan olumlu yeni ara veriler duyurdu.<\/strong> \u015eirket ayr\u0131ca, SGT-003 i\u00e7in h\u0131zland\u0131r\u0131lm\u0131\u015f onay yollar\u0131 da dahil olmak \u00fczere potansiyel kay\u0131t yollar\u0131n\u0131 g\u00f6r\u00fc\u015fmek \u00fczere ABD G\u0131da ve \u0130la\u00e7 Dairesi (FDA) ile ger\u00e7ekle\u015ftirece\u011fi toplant\u0131n\u0131n planlanan zamanlamas\u0131 hakk\u0131nda da g\u00fcncelleme sa\u011flad\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Solid Biosciences Ba\u015fkan\u0131 ve CEO&#039;su Bo Cumbo, &quot;Bug\u00fcn bildirilen ge\u00e7ici INSPIRE DUCHENNE verileri, SGT-003&#039;\u00fcn tedavi potansiyeline olan g\u00fcvenimizi g\u00fc\u00e7lendiriyor. SGT-003 mikrodistrofin ekspresyon seviyelerinin distrofinle ili\u015fkili protein kompleksinin temel bile\u015fenlerinin do\u011fru lokalize restorasyonuyla g\u00fc\u00e7l\u00fc g\u00f6zlemlenen biyolojik korelasyonlar\u0131ndan, kardiyak fonksiyon normalle\u015fmesinin erken kan\u0131tlar\u0131na kadar, insan v\u00fccudunda tedaviye koordineli ve sistemik bir yan\u0131t oldu\u011funu g\u00f6steren net ve kademeli bir etki g\u00f6zlemliyoruz. Bu ge\u00e7ici verilerin, bug\u00fcne kadarki herhangi bir Duchenne gen terapisinin en kapsaml\u0131 erken analizlerinden birini temsil etti\u011fine inan\u0131yoruz. Bu ge\u00e7ici bulgular\u0131n kalitesi ve tutarl\u0131l\u0131\u011f\u0131, SGT-003&#039;\u00fcn molek\u00fcler etkiyi anlaml\u0131 klinik sonu\u00e7lara d\u00f6n\u00fc\u015ft\u00fcrme potansiyeline olan inanc\u0131m\u0131z\u0131 peki\u015ftiriyor.&quot; dedi.\u201c<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bay Cumbo s\u00f6zlerine \u015f\u00f6yle devam etti: &quot;Geli\u015fen d\u00fczenleyici ortam ve INSPIRE DUCHENNE&#039;e kay\u0131tlar\u0131n h\u0131zl\u0131 temposu \u0131\u015f\u0131\u011f\u0131nda, FDA ile planlanan toplant\u0131m\u0131z\u0131 2026&#039;n\u0131n ilk yar\u0131s\u0131na erteleme y\u00f6n\u00fcnde proaktif bir karar ald\u0131k. Bu ek s\u00fcre bize 1) FDA ile g\u00f6r\u00fc\u015fmek \u00fczere daha kapsaml\u0131 bir veri seti olu\u015fturma, 2) y\u00fcksek kaliteli, iyi e\u015fle\u015ftirilmi\u015f do\u011fal tarih verilerine dayal\u0131 kapsaml\u0131 bir harici kar\u015f\u0131la\u015ft\u0131r\u0131c\u0131 \u00fczerinde \u00e7al\u0131\u015fma, 3) potansiyel bir biyolojik lisans ba\u015fvurusu (BLA) sunumuna haz\u0131rl\u0131k olarak CDMO orta\u011f\u0131m\u0131zla s\u00fcre\u00e7 performans kalifikasyonu (PPQ) \u00fcretim \u00e7al\u0131\u015fmalar\u0131m\u0131za ba\u015flama ve 4) Amerika Birle\u015fik Devletleri d\u0131\u015f\u0131nda y\u00fcr\u00fct\u00fclecek olan Faz 3 randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc klinik \u00e7al\u0131\u015fmam\u0131z IMPACT DUCHENNE&#039;de dozlamay\u0131 ba\u015flatma olana\u011f\u0131 sa\u011flayacak. Bu faaliyetlerin, Solid&#039;i d\u00fczenleyicilere en ikna edici paketi sunmak i\u00e7in en iyi konuma getirdi\u011fine inan\u0131yoruz.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201c&quot;\u015eimdiki \u00f6nceli\u011fimiz, SGT-003 i\u00e7in potansiyel h\u0131zland\u0131r\u0131lm\u0131\u015f bir d\u00fczenleyici s\u00fcreci destekleyecek g\u00fc\u00e7l\u00fc bir veri setini h\u0131zla olu\u015fturmak ve SGT-003&#039;\u00fcn potansiyelini m\u00fcmk\u00fcn olan en k\u0131sa s\u00fcrede hayata ge\u00e7irmek i\u00e7in acilen \u00e7al\u0131\u015fmaya kararl\u0131y\u0131z. SGT-003&#039;\u00fcn \u00f6tesinde, \u00f6nc\u00fc boru hatt\u0131 programlar\u0131m\u0131z ilerlemeye devam ediyor.&quot; diye s\u00f6zlerini tamamlad\u0131 Cumbo.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">SGT-003 \u2013 INSPIRE DUCHENNE Klinik Ara\u015ft\u0131rmas\u0131<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>31 Ekim 2025 itibar\u0131yla INSPIRE DUCHENNE denemesinde 23 kat\u0131l\u0131mc\u0131ya doz uyguland\u0131; Solid, 2026&#039;n\u0131n ba\u015flar\u0131na kadar toplam 30 kat\u0131l\u0131mc\u0131ya doz uygulamay\u0131 bekliyor ve ard\u0131ndan 2026&#039;n\u0131n ilk yar\u0131s\u0131nda potansiyel kay\u0131t yollar\u0131n\u0131 g\u00f6r\u00fc\u015fmek \u00fczere FDA ile bir araya gelmeyi planl\u0131yor.<\/li>\n\n\n\n<li>SGT-003, yaln\u0131zca steroid i\u00e7eren profilaktik imm\u00fcnomod\u00fclasyon rejimi kullan\u0131larak genel olarak iyi tolere edilmi\u015ftir; kardiyak g\u00fcvenlik izlemesi, kardiyak hasarda azalma ve kardiyak fonksiyon normalle\u015fmesinin erken sinyallerini g\u00f6stermeye devam etmi\u015ftir.<\/li>\n\n\n\n<li>10 tedavi edilen kat\u0131l\u0131mc\u0131dan (ya\u015flar\u0131 5-10 aras\u0131nda) al\u0131nan 90. g\u00fcn biyopsi verileri, t\u00fcm kat\u0131l\u0131mc\u0131lar\u0131n western blot ile 58%&#039;nin ortalama mikrodistrofin ekspresyonu, k\u00fctle spektrometrisi ile 58%, imm\u00fcnofloresan ile 51%&#039;nin ortalama mikrodistrofin pozitif lifleri ve imm\u00fcnofloresan ile 50%&#039;nin ortalama beta-sarkoglikan pozitif lifleri ile tedaviye yan\u0131t verdi\u011fini g\u00f6sterdi.<\/li>\n\n\n\n<li>90. g\u00fcnde mikrodistrofin ekspresyon seviyeleri ile distrofinle ili\u015fkili protein kompleksinin (DAPC) temel bile\u015fenleri olan beta-sarkoglikan (r = 0,95) ve nNOS (r = 0,95) aras\u0131nda g\u00f6zlemlenen g\u00fc\u00e7l\u00fc istatistiksel korelasyonlar, SGT-003 arac\u0131l\u0131 DAPC restorasyonuna ve CK seviyelerindeki azalmalar yoluyla kas korumas\u0131n\u0131n uyumlu sinyallerine dair kan\u0131t g\u00f6stermi\u015ftir (r = -0,78) \u2013<\/li>\n\n\n\n<li>Solid, ilk klinik deneme merkezini aktif hale getirdi ve \u015fu anda SGT-003&#039;\u00fcn ABD d\u0131\u015f\u0131nda y\u00fcr\u00fct\u00fclen, Faz 3 randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc klinik denemesi olan IMPACT DUCHENNE i\u00e7in kat\u0131l\u0131mc\u0131lar\u0131 tar\u0131yor.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Hem 90. g\u00fcnde hem de 360. g\u00fcnde kas yaralanmas\u0131 ve y\u0131k\u0131m\u0131na ili\u015fkin \u00e7e\u015fitli biyobelirte\u00e7lerde olumlu azalmalar g\u00f6zlemlendi:<\/p>\n\n\n\n<figure class=\"wp-block-table\"><table class=\"has-fixed-layout\"><tbody><tr><td><strong>Serum Biyobelirte\u00e7leri<\/strong><\/td><td class=\"has-text-align-center\" data-align=\"center\"><strong>90. G\u00fcn Ortalama Azalmalar<\/strong><br><strong>(Aksi belirtilmedi\u011fi s\u00fcrece N=14)<\/strong><\/td><td class=\"has-text-align-center\" data-align=\"center\"><strong>360. G\u00fcn Ortalama Azalmalar<\/strong><br><strong>(Aksi belirtilmedi\u011fi s\u00fcrece N=3)<\/strong><\/td><\/tr><tr><td>Serum kreatin kinaz (CK)<\/td><td class=\"has-text-align-center\" data-align=\"center\">34%<\/td><td class=\"has-text-align-center\" data-align=\"center\">42%<\/td><\/tr><tr><td>Serum alanin transaminaz (ALT)<\/td><td class=\"has-text-align-center\" data-align=\"center\">41%<\/td><td class=\"has-text-align-center\" data-align=\"center\">29%<\/td><\/tr><tr><td>Serum aspartat aminotransferaz (AST)<\/td><td class=\"has-text-align-center\" data-align=\"center\">25%<\/td><td class=\"has-text-align-center\" data-align=\"center\">40%<\/td><\/tr><tr><td>Serum laktat dehidrogenaz (LDH)*<\/td><td class=\"has-text-align-center\" data-align=\"center\">42%<\/td><td class=\"has-text-align-center\" data-align=\"center\">46%<\/td><\/tr><tr><td>Serum titin**<\/td><td class=\"has-text-align-center\" data-align=\"center\">22%<\/td><td class=\"has-text-align-center\" data-align=\"center\">25%<\/td><\/tr><tr><td colspan=\"3\"><em>*LDH i\u00e7in 90. g\u00fcnde N=12 kat\u0131l\u0131mc\u0131 \u00f6rne\u011fi mevcuttu (iki \u00f6rnek hemolize edildi); **Titin i\u00e7in 90. g\u00fcnde N=11 kat\u0131l\u0131mc\u0131 \u00f6rne\u011fi ve 360. g\u00fcnde N=2 \u00f6rnek mevcuttu; bu \u00f6rnekler daha erken bir kesme tarihinde toplu olarak analiz edildi.<\/em><\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\">IMPACT DUCHENNE Faz 3<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Ekim 2025&#039;te Solid, ilk klinik ara\u015ft\u0131rma merkezini faaliyete ge\u00e7irdi ve SGT-003&#039;\u00fc de\u011ferlendiren Faz 3 randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc bir klinik ara\u015ft\u0131rma olan IMPACT DUCHENNE i\u00e7in kat\u0131l\u0131mc\u0131 taramas\u0131na ba\u015flad\u0131. IMPACT DUCHENNE, Amerika Birle\u015fik Devletleri (ABD) d\u0131\u015f\u0131ndaki pediatrik kat\u0131l\u0131mc\u0131larda y\u00fcr\u00fct\u00fclecek ve olas\u0131 ABD d\u0131\u015f\u0131 d\u00fczenleyici onaylar\u0131 desteklemek \u00fczere tasarlanm\u0131\u015ft\u0131r. IMPACT DUCHENNE&#039;i hem Kanada hem de Avustralya&#039;da y\u00fcr\u00fctmek i\u00e7in d\u00fczenleyici onaylar ald\u0131k ve d\u00fczenleyici onaylar\u0131n al\u0131nmas\u0131na ba\u011fl\u0131 olarak ara\u015ft\u0131rmay\u0131 ba\u015fka \u00fclkelere de geni\u015fletmeyi planl\u0131yoruz. <a href=\"https:\/\/dmdwarrior.com\/tr\/solid-biosciences-sgt-003-gene-therapy-phase-3-clinical-trial-nct07160634\/\" target=\"_blank\" rel=\"noreferrer noopener\">(IMPACT DUCHENNE)<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu Sayfay\u0131 Takip Edin<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne i\u00e7in T\u00fcm Klinik Ara\u015ft\u0131rmalar<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Solid Biosciences announced positive new interim data from the Phase 1\/2 INSPIRE DUCHENNE clinical trial. The Company also provided an update to the planned timing of its meeting with the U.S. Food and Drug Administration (FDA) to discuss potential registrational pathways, including accelerated approval pathways, for SGT-003. Bo Cumbo, President and CEO of Solid Biosciences [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5102,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[61,110,170,109],"class_list":["post-5101","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-gene-therapy","tag-sgt-003","tag-sgt-003-gene-therapy","tag-solid-biosciences"],"subtitle":"SGT-003 mikrodistrofin tedavisi ile serum kreatin kinaz (CK), laktat dehidrogenaz (LDH), aspartat aminotransferaz (AST) ve embriyonik miyozin a\u011f\u0131r zinciri (eMHC) dahil olmak \u00fczere kas b\u00fct\u00fcnl\u00fc\u011f\u00fcn\u00fcn \u00e7e\u015fitli biyobelirte\u00e7lerindeki iyile\u015fmeler aras\u0131nda da g\u00fc\u00e7l\u00fc korelasyonlar g\u00f6zlemlendi; bu da SGT-003 tedavisinin koordineli bir a\u015fa\u011f\u0131 ak\u0131\u015f etkisine i\u015faret ediyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5101","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=5101"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5101\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5102"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5101"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5101"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5101"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}