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Avidity Biosciences Pursues Potential Accelerated Approval Path With Initiation Of Biomarker Cohort In FORTITUDE Trial For Delpacibart Braxlosiran In People Living With Facioscapulohumeral Muscular Dystrophy

Avidity Biosciences Pursues Potential Accelerated Approval Path With Initiation Of Biomarker Cohort In FORTITUDE Trial For Delpacibart Braxlosiran In People Living With Facioscapulohumeral Muscular Dystrophy

Avidity Biosciences在FORTITUDE試驗中啓動生物標誌物隊列,探索延批利巴特布拉克索利蘭在患有顏面肩肱肌肉萎縮症的人群中的潛在加速批准途徑
Benzinga ·  10/30 08:11

Biomarker cohort for delpacibart braxlosiran (del-brax), the first potential therapy to target DUX4, is measuring changes in DUX4 regulated biomarkers; del-brax 2 mg/kg will be administered every six weeks

針對delpacibart braxlosiran(del-brax)的生物標誌物隊列,這是第一種針對DUX4的潛在療法,正在測量DUX4調控的生物標誌物的變化;del-brax 2毫克/公斤將每六週進行一次給藥。

Enrollment in the del-brax biomarker cohort expected to be completed in 1H 2025; on track to initiate del-brax functional cohort in 1H 2025

預計del-brax生物標誌物隊列的招募將在2025年上半年完成;計劃在2025年上半年啓動del-brax功能隊列。

In previously reported initial data, del-brax 2 mg/kg every six weeks showed unprecedented and consistent reductions of DUX4 regulated genes, significant decreases in novel circulating biomarker and creatine kinase, and trends of functional improvement at the four-month timepoint

在先前報道的初始數據中,每六週使用del-brax 2毫克/公斤顯示出DUX4調控基因的前所未有和一致的減少,新型循環生物標誌物和肌酸激酶的顯著降低,以及在四個月時間點上功能改善的趨勢。

SAN DIEGO, Oct. 30, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), today announced the initiation of a biomarker cohort in the Phase 1/2 FORTITUDE trial of delpacibart braxlosiran (del-brax/AOC 1020) in people living with facioscapulohumeral muscular dystrophy (FSHD). Avidity is pursuing a potential accelerated approval path for del-brax and expects enrollment in the biomarker cohort to be completed in the first half of 2025. Avidity also remains on track to initiate a functional cohort in the first half of 2025. In addition, enrollment of the FORTITUDE Open-label Extension study (OLE) is ongoing.

2024年10月30日,美國加利福尼亞州聖地亞哥 / PRNewswire / - Avidity Biosciences, Inc.(納斯達克:RNA),一家致力於提供一種名爲抗體寡核苷酸結合物(AOCs)的新型RNA治療的生物製藥公司,今天宣佈啓動了delpacibart braxlosiran(del-brax / AOC 1020)在患有面肩上臂肌肉萎縮症(FSHD)的患者中進行FORTITUDE第1/2期試驗的生物標誌物隊列招募。 Avidity正在尋求del-brax的潛在快速批准途徑,並預計生物標誌物隊列的招募將於2025年上半年完成。 Avidity還計劃在2025年上半年啓動功能隊列。此外,FORTITUDE開放標籤擴展研究(OLE)的招募仍在進行中。

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