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
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
针对delpacibart braxlosiran(del-brax)的生物标志物队列,这是第一种针对DUX4的潜在疗法,正在测量DUX4调控的生物标志物的变化;del-brax 2毫克/公斤将每六周进行一次给药。
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
预计del-brax生物标志物队列的招募将在2025年上半年完成;计划在2025年上半年启动del-brax功能队列。
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
在先前报道的初始数据中,每六周使用del-brax 2毫克/公斤显示出DUX4调控基因的前所未有和一致的减少,新型循环生物标志物和肌酸激酶的显著降低,以及在四个月时间点上功能改善的趋势。
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,美国加利福尼亚州圣地亚哥 / PRNewswire / - Avidity Biosciences, Inc.(纳斯达克:RNA),一家致力于提供一种名为抗体寡核苷酸结合物(AOCs)的新型RNA治疗的生物制药公司,今天宣布启动了delpacibart braxlosiran(del-brax / AOC 1020)在患有面肩上臂肌肉萎缩症(FSHD)的患者中进行FORTITUDE第1/2期试验的生物标志物队列招募。 Avidity正在寻求del-brax的潜在快速批准途径,并预计生物标志物队列的招募将于2025年上半年完成。 Avidity还计划在2025年上半年启动功能队列。此外,FORTITUDE开放标签扩展研究(OLE)的招募仍在进行中。