delpacibart braxlosiran (AOC-1020) / Avidity Biosci - LARVOL DELTA

FORTITUDE: Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (clinicaltrials.gov) - P1/2 | N=90 | Active, not recruiting | Sponsor: Avidity Biosciences, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Muscular Atrophy • Muscular Dystrophy

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 (PRNewswire) - "Avidity Biosciences, Inc...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...Avidity is on track to deliver multiple updates from the del-brax program in Q2 including: Regulatory 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; and Topline data from the dose escalation cohorts in the FORTITUDE trial."

Enrollment closed • New P3 trial • P3 data: top line • Muscular Dystrophy

Avidity Biosciences Reports Fourth Quarter 2024 Financial Results and Recent Highlights (PRNewswire) - "Delpacibart zotadirsen (del-zota) for the treatment of DMD44: Planned BLA submission year end 2025....Presentation of topline data from the EXPLORE44 trial (Q1). Presentation of topline data from the ongoing EXPLORE44-OLE trial (Q4). Delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1)...Publication of data analyses from the completed Phase 1/2 MARINA trial (2025)....Research and Development (R&D) Expenses: These expenses were $95.6 million for the fourth quarter of 2024 compared with $52.8 million for the fourth quarter of 2023, and $303.6 million for the year ended 2024 compared with $191.0 million for the year ended 2023. The increases were primarily driven by the advancement of del-desiran, del-brax and del-zota, as well as internal and external costs related to the expansion of the company's overall research capabilities."

Commercial • FDA filing • P1/2 data • P2 data • Duchenne Muscular Dystrophy • Muscular Dystrophy • Myotonic Dystrophy

2025 Upcoming Clinical and Regulatory Highlights (PRNewswire) - "Delpacibart braxlosiran (del-brax) for the treatment of FSHD: Regulatory alignment on a global Phase 3 trial design (Q2); Alignment on a potential accelerated approval path for the ongoing FORTITUDE biomarker cohort (Q2); Completion of enrollment of the FORTITUDE biomarker cohort (Q2); Presentation of topline data from the FORTITUDE trial (Q2); Initiation of a global, potentially registrational trial in FSHD (Q2)"

New trial • Trial status • Muscular Dystrophy

FORTITUDE: Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (clinicaltrials.gov) - P1/2 | N=100 | Recruiting | Sponsor: Avidity Biosciences, Inc. | N=72 ➔ 100 | Trial completion date: Sep 2025 ➔ Apr 2027 | Trial primary completion date: Sep 2025 ➔ Oct 2026

Enrollment change • Trial completion date • Trial primary completion date • Muscular Atrophy • Muscular Dystrophy • DUX4

FORTITUDE-OLE: Phase 2 Open-label Extension Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (clinicaltrials.gov) - P2 | N=72 | Enrolling by invitation | Sponsor: Avidity Biosciences, Inc.

New P2 trial • Muscular Atrophy • Muscular Dystrophy

Targeting DUX4 for silencing with AOC for the Treatment of FSHD (MDA 2024) - "Transcriptomic analysis revealed the dysregulation of many genes and pathways upon induction of DUX4 expression in muscle of this mouse model, which was largely prevented with DUX4 AOC treatment. These data support the evaluation of DUX4-targeting AOC 1020 in the ongoing Phase 1/2 FORTITUDE trial in adults with FSHD."

Muscular Dystrophy • ACTA1 • DUX4 • TFRC

FORTITUDE: Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (clinicaltrials.gov) - P1/2 | N=72 | Recruiting | Sponsor: Avidity Biosciences, Inc.

Trial completion date • Trial primary completion date • Muscular Atrophy • Muscular Dystrophy

Phase 1/2 study to evaluate AOC 1020 for adult patients with Facioscapulohumeral muscular dystrophy: FORTITUDE trial design (WMS 2023) - No abstract available

Clinical • P1/2 data • Muscular Dystrophy

Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of AOC 1020 Administered Intravenously to Adult Patients with Facioscapulohumeral Muscular Dystrophy (FORTITUDE) Trial Design (AAN 2023) - "Strategies targeting DUX4 expression in skeletal muscle of individuals with FSHD via oligonucleotides are promising therapeutic approaches.AOC 1020 is an antibody-oligonucleotide conjugate (AOCTM) comprised of a DUX4-targeting siRNA conjugated to a humanized antibody targeting transferrin receptor 1 (TfR1) to facilitate delivery to muscle. Exploratory measures of efficacy include pharmacodynamics, PROs, and measures of muscle function, strength, and composition by MRI. Results N/A Conclusions N/A"

Clinical • P1/2 data • PK/PD data • Muscular Dystrophy • DUX4 • TFRC

FORTITUDE: Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (clinicaltrials.gov) - P1/2 | N=72 | Recruiting | Sponsor: Avidity Biosciences, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Muscular Atrophy • Muscular Dystrophy

FORTITUDE: Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (clinicaltrials.gov) - P1/2 | N=72 | Not yet recruiting | Sponsor: Avidity Biosciences, Inc.

New P1/2 trial • Muscular Atrophy • Muscular Dystrophy

AOC 1020: An Antibody Oligonucleotide Conjugate (AOC) in Development for the Treatment of FSHD (WMS 2022) - "Single treatment also prevented FSHD-related muscle phenotype in this mouse model. Data presented provide support for AOC 1020 to enter the clinic for the treatment of FSHD by end of 2022."

Late-breaking abstract • ACTA1 • TFRC