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Avidity Biosciences Completes Enrollment in Biomarker Cohort in Phase 1/2 FORTITUDE™ Trial for Delpacibart Braxlosiran (del-brax) in People Living with Facioscapulohumeral Muscular Dystrophy
Del-brax FORTITUDE biomarker cohort designed for potential accelerated approval; plan to share regulatory update in Q2 2025 Regulatory alignment on global
About this update from Atrium Therapeutics, Inc.
[{"type":"text","content":"Del-brax FORTITUDE biomarker cohort designed for potential accelerated approval; plan to share regulatory update in Q2 2025\nRegulatory alignment on global Phase 3 del-brax trial design and study initiation anticipated in Q2 2025\nPlan to present topline data from FORTITUDE dose escalation cohorts in Q2 2025\nOn track to be first globally approved drug for FSHD\nSAN DIEGO, March 31, 2025 /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 completion of enrollment in the biomarker cohort in the Phase 1/2 FORTITUDE™ clinical trial of delpacibart braxlosiran (del-brax) in people living with facioscapulohumeral muscular dystrophy (FSHD). A total of 51 participants were enrolled in the FORTITUDE biomarker cohort.\n\n \n \n \n \n \n \n\n \n\"Completing enrollment in the FORTITUDE biomarker cohort is an important milestone as we pursue a potential accelerated approval path in the U.S. for del-brax and work to bring the first approved drug to people living with this rare, devastating neuromuscular disease who have no treatment options as quickly as possible,\" said Steve Hughes, M.D., chief medical officer at Avidity. \"We are very encouraged by the del-brax 2 mg/kg data thus far, demonstrating unprecedented and consistent reductions in DUX4-regulated genes, significant decreases in novel circulating biomarker and creatine kinase, trends of functional improvement, and favorable safety and tolerability, and look forward to sharing additional data as well as other key milestones in the second quarter of this year.\"\nAvidity is on track to deliver multiple updates from the del-brax program in Q2 including:\nRegulatory alignment on a potential accelerated approval path in the U.S. for the ongoing FORTITUDE biomarker cohort;Regulatory alignment on the design of the global Phase 3 trial as well as initiation of the trial; andTopline data from the dose escalation cohorts in the FORTITUDE trial.Del-brax is the first investigational therapy designed to treat the underlying cause of FSHD by directly targeting the mRNA transcript of the disease-causing gene, double homeobox 4 (DUX4). Currently, there are no approved therapies for the treatment of FSHD, a rare, hereditary disorder marked b...