Community Communications

March 2025 – Phase 1/2 FORTITUDE Trial Biomarker Cohort Enrollment Completion

March 2025 – Topline Del-zota Data from Phase 1/2 EXPLORE44® trial in people living with DMD44

October 2024 – Initiation of Biomarker Cohort in FORTITUDE™ Trial for Delpacibart Braxlosiran (del-brax/AOC 1020) in People Living with Facioscapulohumeral Muscular Dystrophy

August 2024 – Delpacibart zotadirsen (AOC 1044) EXPLORE44™ Phase 1/2 Trial Data

June 2024 – First Patient Enrolled in Global Phase 3 HARBOR™ Clinical Trial for the Treatment of Myotonic Dystrophy Type 1

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 in 90 participants with facioscapulohumeral muscular dystrophy (FSHD). For more information about the FORTITUDE trial, visit the FORTITUDE study website or visit http://www.clinicaltrials.gov and search for NCT05747924.

FORTITUDE-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of delpacibart braxlosiran or del-brax in participants with facioscapulohumeral muscular dystrophy (FSHD) who were previously enrolled in the Phase 1/2 FORTITUDE™ trial. The total duration of active treatment with del-brax in the FORTITUDE-OLE is approximately 24 months. Avidity may extend active treatment beyond 24 months at a future timepoint. For more information on the FORTITUDE-OLE study click here or visit http://www.clinicaltrials.gov and search for NCT06547216.

The EXPLORE44® trial is now complete. The study was 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). For more information about the EXPLORE44 trial, visit Avidity’s website publications page, the EXPLORE44 study website or http://www.clinicaltrials.gov and search for NCT05670730.

The Phase 2 EXPLORE44-OLE™ trial is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of del-zota in participants living with DMD44. Participants in the EXPLORE44® trial had the option to enroll in the EXPLORE44-OLE study for del-zota. In addition, Avidity enrolled additional participants in the EXPLORE44-OLE study to support Avidity’s first BLA submission at year end 2025. The EXPLORE44-OLE study completed enrollment in February 2025 with a total of 39 participants enrolled. This trial will evaluate the safety, tolerability, PK, PD, and efficacy of del-zota. For more information on this study click here or visit http://www.clinicaltrials.gov and search for NCT06244082.