{"id":4004,"date":"2025-06-26T19:44:06","date_gmt":"2025-06-26T16:44:06","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4004"},"modified":"2025-06-26T19:56:53","modified_gmt":"2025-06-26T16:56:53","slug":"edgewise-therapeutics-reports-positive-results-on-sevasemten-program-for-becker-and-duchenne-muscular-dystrophies","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/edgewise-therapeutics-reports-positive-results-on-sevasemten-program-for-becker-and-duchenne-muscular-dystrophies\/","title":{"rendered":"Edgewise Therapeutics, Becker ve Duchenne Musk\u00fcler Distrofileri \u0130\u00e7in Sevasemten Program\u0131nda Olumlu Sonu\u00e7lar Bildirdi"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Kas hastal\u0131klar\u0131 biyofarmas\u00f6tik alan\u0131nda \u00f6nde gelen \u015firketlerden Edgewise Therapeutics, bug\u00fcn Becker ve Duchenne kas distrofisi i\u00e7in uygulad\u0131\u011f\u0131 tedavi program\u0131nda elde etti\u011fi olumlu sonu\u00e7lar\u0131 duyurdu.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0130\u00e7indekiler<\/h2><nav><ul><li><a href=\"#what-is-sevasemten\">Sevasemten Nedir?<\/a><\/li><li><a href=\"#sevasemten\">Sevasemten: NSAA Puanlar\u0131<\/a><\/li><li><a href=\"#phase-2-duchenne-trials\">Faz 2 Duchenne Denemeleri<\/a><\/li><li><a href=\"#sevasemten-1\">Sevasemten Klinik \u00c7al\u0131\u015fmalar\u0131na Kat\u0131lan \u00c7ocuklar\u0131n Ya\u015flar\u0131<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-sevasemten\">Sevasemten Nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Sevasemten, Becker ve Duchenne gibi kas distrofilerinde kas\u0131lmaya ba\u011fl\u0131 kas hasar\u0131na kar\u015f\u0131 koruma sa\u011flamak \u00fczere tasarlanm\u0131\u015f, oral yoldan uygulanan, s\u0131n\u0131f\u0131nda birinci s\u0131n\u0131f h\u0131zl\u0131 iskelet miyozin inhibit\u00f6r\u00fcd\u00fcr.<\/strong> Sevasemten, fonksiyonel distrofinin yoklu\u011fu veya kayb\u0131 nedeniyle olu\u015fan abart\u0131l\u0131 kas hasar\u0131n\u0131 se\u00e7ici olarak s\u0131n\u0131rlamak i\u00e7in tasarlanm\u0131\u015f yeni bir etki mekanizmas\u0131 sunar. Benzersiz etki mekanizmas\u0131, Sevasemten'i distrofinopatilerde temel bir tedavi olarak, tek ba\u015f\u0131na bir tedavi olarak veya mevcut tedaviler ve geli\u015ftirilmekte olanlarla kombinasyon halinde kurma potansiyeli sa\u011flar.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"sevasemten\">Sevasemten: NSAA Puanlar\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">MESA verileri, \u00f6nceki ARCH ve CANYON bulgular\u0131n\u0131 g\u00fc\u00e7lendirerek, s\u00fcrd\u00fcr\u00fclebilir hastal\u0131k stabilizasyonunu g\u00f6sterdi. Daha da \u00f6nemlisi, MESA&#039;ya ge\u00e7en CANYON kat\u0131l\u0131mc\u0131lar\u0131, 18 ay boyunca Kuzey Y\u0131ld\u0131z\u0131 Ambulatuvar De\u011ferlendirmesi (NSAA) puanlar\u0131nda art\u0131\u015f g\u00f6sterdi (ba\u015flang\u0131\u00e7tan itibaren 0,8 puan iyile\u015fme), sevasemten&#039;e ge\u00e7en plasebo kat\u0131l\u0131mc\u0131lar\u0131nda iyile\u015fme e\u011filimi vard\u0131 (sevasemten ba\u015flang\u0131c\u0131ndan bu yana 0,2 puan iyile\u015fme). Sevasemten tedavisinin 18 ay\u0131 boyunca, kat\u0131l\u0131mc\u0131lar\u0131n NSAA puanlar\u0131, birden fazla Becker do\u011fal tarih \u00e7al\u0131\u015fmas\u0131nda g\u00f6r\u00fclen beklenen i\u015flevsel d\u00fc\u015f\u00fc\u015flere g\u00f6re farkl\u0131la\u015fmaya devam etti. Dahas\u0131, MESA&#039;ya ge\u00e7en ARCH kat\u0131l\u0131mc\u0131lar\u0131n\u0131n NSAA puanlar\u0131, \u00fc\u00e7 y\u0131ll\u0131k tedaviden sonra stabil kald\u0131. Sevasemten, \u00fc\u00e7 y\u0131la kadar tedaviden sonra olumlu bir g\u00fcvenlik profili g\u00f6stermeye devam ediyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Edgewise yak\u0131n zamanda ABD G\u0131da ve \u0130la\u00e7 Dairesi (FDA) ile ba\u015far\u0131l\u0131 bir Tip C toplant\u0131s\u0131n\u0131 tamamlad\u0131 ve bu toplant\u0131 Becker i\u00e7in ilk tedavi olarak sevasemtenin kayd\u0131na giden net bir yol sa\u011flad\u0131. FDA, CANYON verilerinin tek ba\u015f\u0131na h\u0131zland\u0131r\u0131lm\u0131\u015f bir onay i\u00e7in yeterli olmad\u0131\u011f\u0131n\u0131 d\u00fc\u015f\u00fcnse de, Ajans NSAA&#039;n\u0131n geleneksel onay i\u00e7in klinik olarak anlaml\u0131 bir son nokta oldu\u011funu yineledi. FDA, Edgewise&#039;\u0131 GRAND CANYON tamamlanmadan \u00f6nce MESA verilerini ve do\u011fal tarih prospektif modellemesini payla\u015fmaya devam etmeye te\u015fvik etti. Ayr\u0131ca, FDA, devam eden k\u00fcresel pivot plasebo kontroll\u00fc kohort olan GRAND CANYON&#039;a ve kayd\u0131 desteklemek i\u00e7in yeterli ve iyi kontroll\u00fc tek bir \u00e7al\u0131\u015fma olarak potansiyeline olan desteklerini vurgulad\u0131. GRAND CANYON, 18 ay boyunca NSAA ile plasebo aras\u0131nda istatistiksel olarak anlaml\u0131 bir fark g\u00f6stermek i\u00e7in olduk\u00e7a g\u00fc\u00e7l\u00fcd\u00fcr ve 2026&#039;n\u0131n d\u00f6rd\u00fcnc\u00fc \u00e7eyre\u011finde en \u00fcst d\u00fczey veriler i\u00e7in yoldad\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"phase-2-duchenne-trials\">Faz 2 Duchenne Denemeleri<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket ayr\u0131ca Faz 2 Duchenne denemeleri LYNX ve FOX&#039;tan cesaret verici \u00fcst d\u00fczey verileri duyurdu. Ama\u00e7lar, g\u00fcvenli\u011fi de\u011ferlendirmek ve Faz 3 i\u00e7in potansiyel olarak faydal\u0131 bir doz belirlemek i\u00e7in bir dizi dozu ke\u015ffetmekti. Denemenin doz art\u0131rma paradigmas\u0131, doz se\u00e7imi i\u00e7in biyobelirte\u00e7leri de\u011ferlendirmek \u00fczere \u00fc\u00e7 ayl\u0131k plasebo kontroll\u00fc bir d\u00f6nem sa\u011flad\u0131 ve bunu a\u00e7\u0131k etiketli bir d\u00f6nem izledi. Her iki \u00e7al\u0131\u015fmada da, hedef dozlarda, sevasemten iyi tolere edildi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">LYNX, Duchenne&#039;li d\u00f6rt ila dokuz ya\u015f\u0131ndaki kat\u0131l\u0131mc\u0131larda sevasemtenin g\u00fcvenlik, kas hasar\u0131 biyobelirte\u00e7leri ve i\u015flevi \u00fczerindeki etkisini de\u011ferlendirmek i\u00e7in devam eden \u00e7ok merkezli, plasebo kontroll\u00fc, doz bulma Faz 2 \u00e7al\u0131\u015fmas\u0131d\u0131r ve \u00fc\u00e7 ayl\u0131k plasebo kontroll\u00fc doz aral\u0131\u011f\u0131 \u00e7al\u0131\u015fmas\u0131nda sevasemten ile tedavi edilmi\u015ftir. Stride Velocity 95th Centile (SV95C), NSAA ve 4 merdiven \u00e7\u0131kma dahil olmak \u00fczere i\u015flevsel \u00f6l\u00e7\u00fcmlerdeki tutarl\u0131 g\u00f6zlemler, Faz 3&#039;te de\u011ferlendirilmek \u00fczere 10 mg&#039;l\u0131k bir doz belirlemi\u015ftir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"sevasemten-1\">Sevasemten Klinik \u00c7al\u0131\u015fmalar\u0131na Kat\u0131lan \u00c7ocuklar\u0131n Ya\u015flar\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">LYNX tasar\u0131m\u0131na benzer \u015fekilde, FOX, daha \u00f6nce gen terapisi g\u00f6rm\u00fc\u015f Duchenne&#039;li alt\u0131 ila 14 ya\u015f\u0131ndaki kat\u0131l\u0131mc\u0131larda sevasemten&#039;in g\u00fcvenlik, kas hasar\u0131 biyobelirte\u00e7leri ve i\u015flevi \u00fczerindeki etkisini de\u011ferlendirmek i\u00e7in devam eden \u00e7ok merkezli, plasebo kontroll\u00fc bir Faz 2 \u00e7al\u0131\u015fmas\u0131d\u0131r. FOX kat\u0131l\u0131mc\u0131lar\u0131 ortalama olarak 10 ya\u015f\u0131n \u00fczerindedir ve gen terapisi almalar\u0131ndan d\u00f6rt y\u0131l ge\u00e7mi\u015ftir. Duchenne gen terapisiyle tedavi edilen erkek \u00e7ocuklarda kapsaml\u0131 bir do\u011fal ge\u00e7mi\u015f olmamas\u0131na ra\u011fmen, FOX \u00e7al\u0131\u015fmas\u0131ndan elde edilen ilk sonu\u00e7lar, sevasemten 10 mg&#039;\u0131n i\u015flevsel gerileme oran\u0131n\u0131 azaltma potansiyeline sahip oldu\u011funu g\u00f6stermektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cDuchenne&#039;de sevasemten i\u00e7in \u00f6nemli bir Faz 2 ara\u015ft\u0131rmas\u0131 y\u00fcr\u00fctt\u00fck,\u201d dedi Dr. Kevin Koch, Ba\u015fkan ve \u0130cra Kurulu Ba\u015fkan\u0131. \u201c10 mg dozunda g\u00f6r\u00fclen i\u015flevsel yan\u0131ttan cesaret al\u0131yoruz ve bu y\u0131l\u0131n ilerleyen zamanlar\u0131nda Ajans ile g\u00f6r\u00fc\u015fmelerimizi sab\u0131rs\u0131zl\u0131kla bekliyoruz.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket, 2025&#039;in d\u00f6rd\u00fcnc\u00fc \u00e7eyre\u011finde FDA ile hasta pop\u00fclasyonu ve son noktalara ili\u015fkin girdiler de dahil olmak \u00fczere bir Faz 3 tasar\u0131m\u0131 g\u00f6r\u00fc\u015fmek \u00fczere bir araya gelmeyi ve 2026&#039;da pivot \u00e7al\u0131\u015fmay\u0131 ba\u015flatmay\u0131 planl\u0131yor. Ayr\u0131ca \u015eirket, deneme kat\u0131l\u0131mc\u0131lar\u0131na ilaca daha fazla eri\u015fim sa\u011flayacak olan daha uzun vadeli a\u00e7\u0131k etiketli uzatma verilerini toplamaya devam etmeyi planl\u0131yor.<\/p>","protected":false},"excerpt":{"rendered":"<p>Edgewise Therapeutics, a leading muscle disease biopharmaceutical company, today unveiled positive results in its sevasemten program for Becker and Duchenne muscular dystrophies. What is Sevasemten? Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect against contraction-induced muscle damage in muscular dystrophies including Becker and Duchenne. Sevasemten presents a novel mechanism of [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4005,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[67,137,242,66,151,152,150],"class_list":["post-4004","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-becker-muscular-dystrophy","tag-clinical-trial","tag-clinical-trials","tag-duchenne-muscular-dystrophy","tag-edgewise","tag-edgewise-therapeutics","tag-sevasemten"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4004","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=4004"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4004\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4005"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4004"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4004"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4004"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}