Avidity’s experimental therapy shows promise for form of muscular dystrophy 

12 Jun 2024
firstwordpharma.com
Clinical ResultOligonucleotideClinical Study
Avidity Biosciences said Wednesday that its experimental therapy delpacibart braxlosiran (del-brax) was able to successfully knock-out the gene that causes facioscapulohumeral muscular dystrophy (FSHD), setting up a registrational study of the antibody oligonucleotide conjugate. Shares in the company jumped as much as 30% on the news.
Del-brax, previously known as AOC 1020, consists of an antibody that binds to transferrin receptor 1 (TfR1) conjugated with an siRNA targeting DUX4 mRNA. FSHD is caused by abnormal expression of DUX4 protein, which leads to changes in gene expression in muscle cells.
Data from the Phase I/II FORTITUDE trial demonstrated that del-brax provided >50% mean reduction across multiple DUX4 regulated gene panels after four months. The efficacy results include 12 participants in the lower-dose cohort, all of whom experienced reductions of >20% in DUX4 regulated genes.
Study investigator Jeffrey Statland said, “It is very encouraging to see that the del-brax data demonstrate consistent reductions in DUX4 regulated genes and provided trends of functional improvement,” adding that the early findings “support the notion that del-brax has the potential to change the course of [the] disease.”
Avidity noted that the safety of del-brax across all 39 patients in the study was favourable, with adverse events being mild or moderate. The company plans to accelerate initiation of Phase III cohorts in the FORTITUDE trial, with a biomarker arm starting later this year – possibly supporting accelerated approval – and a functional cohort in the first half of 2025.
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