Dyne Therapeutics Supporting FSHD Natural History Study Underway in US, Europe

Dyne Therapeutics announced that it has joined in supporting an ongoing global and observational natural history study to better inform the development of treatments for facioscapulohumeral muscular dystrophy (FSHD).

The company’s support — the amount was not disclosed — will help to bring new sites across Europe into the study, and to finance a muscle biopsy sub-study to advance biomarker development and understanding of muscle changes and disease progression, a Dyne spokesman told Muscular Dystrophy News.

Called Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD (ReSolve) (NCT03458832), the 18-month longitudinal study is run by the FSHD Clinical Trial Research Network (CTRN), a University of Kansas Medical Center-led group of medical centers across the United States and Europe that seek to validate new outcome evaluations for patients and improve FSHD clinical trial planning.

“We are proud to contribute to initiatives like the ReSolve study that seek to accelerate the development of new therapeutic options,” Romesh Subramanian, PhD, Dyne president and CEO, said in a news release. Using its muscle-targeted FORCE platform technology, which can deliver oligonucleotides (lab-made pieces of DNA or RNA) and other molecules to skeletal, cardiac and smooth muscle, Dyne is developing therapies for muscle disorders such as FSHD.

To support the design and implementation of clinical studies, ReSolve is specifically constructed to validate new clinical outcome assessments and assess biomarkers. It’s also aimed at gaining a better understanding of how genetic and demographic factors relate to disease progression.

The study is currently enrolling about 160 FSHD patients, ages 18 to 75, at eight U.S. sites, plus 60 patients across three sites in Europe. So far, roughly 140 patients have been enrolled. The U.S. locations are in California, Kansas, Maryland, New York, Ohio, Utah, Virginia, and Washington; contact information is available here.

Sponsored by the University of Kansas Medical Center in collaboration with the National Institute of Neurological Disorders and Stroke, the study hopes to develop and validate new tools for future FSHD trials, including two clinical outcome assessments: a composite functional outcome measure (FSHD-COM) and skeletal muscle biomarker, and an electrical impedance myography (EIM).

The FSHD-COM is composed of disease-relevant motor tests involving balance, the trunk, and arms, hands, legs and shoulders.  EIM is a painless and non-invasive technique for collecting information on changes in a patient’s muscle structure. It uses a small electrical current to measure the health of underlying muscle.

“With the advancement of targeted treatments for FSHD, it is now more critical than ever to develop reliable clinical outcome assessments and methodologies,” said Jeffrey Statland, MD, the ReSolve study’s co-principal investigator. “We are grateful for Dyne’s support and look forward to working with them to advance this important work.”

Visit this site to learn more about the ReSolve study, including enrollment eligibility and study locations.

The Muscular Dystrophy Association (MDA), the FSHD Society, Fulcrum Therapeutics and Friends of FSH Research are among those also supporting the ReSolve study.

FSHD is marked by a progressive weakening of muscles, beginning in the face, shoulders and upper arms. According to the National Institutes of Health (NIH), the disorder has an estimated prevalence of 1 in every 20,000 individuals.