Avidity Biosciences has successfully finished enrolling participants for the biomarker cohort in its Phase 1/2 FORTITUDE™ trial, targeting facioscapulohumeral muscular dystrophy (FSHD). This milestone marks a significant step towards potentially accelerating the approval process for delpacibart braxlosiran (del-brax), the company’s innovative RNA therapeutic.
Advancing Treatment for a Rare Disease
The FORTITUDE trial enrolled 51 individuals suffering from FSHD, a debilitating neuromuscular disorder with no current FDA-approved treatments. Del-brax aims to address the root cause of FSHD by targeting the DUX4 gene, which is responsible for muscle degeneration in affected patients. Early data from the 2 mg/kg dosage group revealed substantial reductions in disease markers and promising safety profiles, encouraging further development.
Strategic Steps Towards Regulatory Approval
Looking ahead, Avidity plans to seek regulatory alignment for a global Phase 3 trial alongside initiating study protocols by the second quarter of 2025. The company anticipates sharing comprehensive regulatory updates and top-line data from dose escalation cohorts within the same timeframe, positioning del-brax as a frontrunner for FDA approval.
– Del-brax demonstrated over 50% reduction in DUX4-regulated genes
– Significant decreases observed in circulating biomarkers and creatine kinase
– Trend indicators show potential functional improvements in patients
– Favorable safety and tolerability profiles noted in trial participants
The completion of the biomarker cohort not only validates the trial’s design but also sets the stage for del-brax to potentially become the first approved therapy for FSHD globally. Participants now have the option to continue their treatment in the open-label extension study, ensuring long-term data collection and safety monitoring.
Avidity’s innovative Antibody Oligonucleotide Conjugates (AOCs™) platform underscores the company’s commitment to pioneering RNA-based treatments. By combining monoclonal antibodies with precision oligonucleotide therapies, Avidity targets diseases that were previously challenging to address with existing RNA technologies.
Del-brax’s progress exemplifies Avidity’s mission to transform the treatment landscape for rare and debilitating conditions. As the company moves closer to potential regulatory approval, patients and stakeholders remain hopeful for a viable treatment option that promises to improve quality of life for those affected by FSHD.
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