Avidity Biosciences(RNA) Prnewswire·2025-03-31 13:00Core Insights - Avidity Biosciences has completed enrollment in the FORTITUDE biomarker cohort for delpacibart braxlosiran (del-brax), targeting accelerated approval for treating facioscapulohumeral muscular dystrophy (FSHD) [1][2] - The company plans to present topline data from the FORTITUDE trial in Q2 2025, with regulatory alignment anticipated for a global Phase 3 trial [1][6] - Del-brax aims to be the first approved therapy for FSHD, a rare disease affecting approximately 45,000 to 87,000 individuals in the U.S. and EU [2][10] Company Overview - Avidity Biosciences focuses on RNA therapeutics, specifically Antibody Oligonucleotide Conjugates (AOCs™), to address rare neuromuscular diseases [11] - The company is advancing clinical programs for multiple conditions, including myotonic dystrophy type 1 and Duchenne muscular dystrophy, alongside FSHD [11] Clinical Trial Details - The FORTITUDE trial is a Phase 1/2 randomized, placebo-controlled study evaluating del-brax in 90 participants with FSHD [3] - The trial assesses safety, tolerability, pharmacokinetics, and pharmacodynamics, using biomarkers like DUX4-regulated genes and MRI measures [3][4] - The ongoing biomarker cohort evaluates del-brax at a dose of 2 mg/kg every six weeks for 12 months, focusing on changes in DUX4-regulated gene expression [5] Efficacy and Safety Data - Initial data from the 2 mg/kg dose of del-brax show over 50% reductions in DUX4-regulated genes, alongside trends of functional improvement and favorable safety [2][9] - The trial's design includes three dose cohorts, with the 2 mg/kg every six weeks identified for future studies [4] Future Plans - Avidity aims to share additional data and key milestones from the del-brax program in Q2 2025, including regulatory updates and topline results [2][6]