Community Communications
August 2024 – Delpacibart zotadirsen (AOC 1044) EXPLORE44™ Phase 1/2 Trial Data
June 2024 – Delpacibart braxlosiran (AOC 1020) FORTITUDE™ Phase 1/2 Initial Data
May 2024 – FDA Breakthrough Therapy Designation for Delpacibart Etedesiran (AOC 1001)
March 2024 – Delpacibart Etedesiran (AOC 1001) MARINA-OLE™ Long-term Data and Phase 3 HARBOR Study
December 2023 – AOC 1044 Healthy Volunteer Data Update
October 2023 – AOC 1001 Data Update
Clinical Studies
The global Phase 3 HARBOR™ trial is a randomized, placebo-controlled, double-blind pivotal study designed to evaluate del-desiran in approximately 150 people (age 16 and older) living with DM1. The trial will be conducted at approximately 40 sites globally. For more information about the HARBOR trial, visit the HARBOR study website or visit http://www.clinicaltrials.gov and search for NCT06411288.
MARINA-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of del-desiran in participants with DM1 who were previously enrolled in the MARINA® Phase 1/2 trial. For more information on this study click here or visit http://www.clinicaltrials.gov and search for NCT05479981.
The FORTITUDE™ trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial designed to evaluate single and multiple doses of del-brax (AOC 1020) in approximately 100 participants with facioscapulohumeral muscular dystrophy (FSHD). In October 2024, Avidity announced the initiation of the biomarker cohort in the FORTITUDE study. Avidity is pursuing a potential accelerated approval path for del-brax and enrollment in the biomarker cohort is expected to be completed in the first half of 2025. Avidity also remains on track to initiate the functional cohort for del-brax in the first of half of 2025. For more information about the FORTITUDE trial, visit the FORTITUDE study website or visit http://www.clinicaltrials.gov and search for NCT05747924.
The EXPLORE44™ trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial designed to evaluate del-zota in healthy volunteers and participants with DMD mutations amenable to exon 44 skipping (DMD44). The EXPLORE44 trial is now fully enrolled and ongoing. For more information about the EXPLORE44 trial, visit the EXPLORE44 study website or http://www.clinicaltrials.gov and search for NCT05670730.