Avidity Biosciences and FSHD Society Raise Awareness on World FSHD Day

Avidity Biosciences is collaborating with the FSHD Society to raise awareness about facioscapulohumeral muscular dystrophy (FSHD) in support of World FSHD Day. The partnership includes a webinar today at 1:00 p.m. ET to discuss promising data from Avidity's Phase 1/2 FORTITUDE™ trial. The trial, which focuses on FSHD, demonstrated significant results in reducing DUX4 regulated genes and showed trends of functional improvements along with favorable safety and tolerability.

FSHD is a rare, hereditary muscle disorder characterized by progressive muscle loss, significant pain, fatigue, and disability. The FORTITUDE™ trial is investigating del-brax, an experimental therapy aimed at addressing the underlying cause of FSHD. Avidity's President and CEO, Sarah Boyce, emphasized the company's commitment to developing a treatment for FSHD as quickly as possible, highlighting the vital contributions of patients and caregivers to the research.

The FSHD Society, led by President and CEO Mark Stone, has been dedicated to accelerating the delivery of treatments and a cure for FSHD. The organization is optimistic about the potential treatment options suggested by the successful data from Avidity's FORTITUDE study. World FSHD Day serves as an opportunity to acknowledge the collaborative efforts and dedication required to support families affected by FSHD and drive research forward.

The FORTITUDE trial involves 39 adult participants with FSHD and aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of del-brax administered intravenously. The primary goal is to evaluate the safety and tolerability of del-brax in FSHD patients. The trial also explores various biomarkers, including MRI measures of muscle volume and composition. Although not statistically powered to assess functional benefit, it looks into del-brax's clinical activity, including mobility, muscle strength, and quality of life measures.

Del-brax (AOC 1020) is designed to mitigate the abnormal expression of the DUX4 gene in muscle cells, a root cause of FSHD. It comprises a monoclonal antibody targeting the transferrin receptor 1 (TfR1) and a siRNA that targets DUX4 mRNA. Preclinical studies indicated that a single intravenous dose of del-brax could prevent muscle weakness in functional assays. Del-brax has received Orphan designation from the FDA and EMA and has been granted Fast Track designation by the FDA.

FSHD is a progressive genetic disorder that leads to skeletal muscle weakness, starting in the face, shoulders, arms, and trunk, and potentially progressing to the lower body. Many patients experience significant physical limitations and may rely on wheelchairs. Currently, no approved treatments exist for FSHD, making the advancements in the FORTITUDE™ trial particularly significant.

Avidity Biosciences is dedicated to revolutionizing RNA therapeutics with their Antibody Oligonucleotide Conjugates (AOCs™). The AOC platform combines the specificity of monoclonal antibodies with the precision of oligonucleotide therapies, targeting diseases previously beyond the reach of existing RNA therapies. Avidity is advancing clinical programs for three rare muscle diseases, including FSHD, and expanding their pipeline to address cardiology and immunology through internal efforts and key partnerships.

Today's webinar will feature insights from Dr. Jeffrey Statland, a trial investigator from the University of Kansas Medical Center, and will delve into the initial findings of the FORTITUDE trial. This event marks an important step in raising awareness and advancing research for FSHD, demonstrating the hopeful advancements in treatment possibilities for affected individuals and their families.