Dyne helps expand muscular dystrophy studies

WALTHAM, Mass.—Dyne Therapeutics, a biotechnology company pioneering targeted therapies for patients with serious muscle diseases, has announced its support for the ReSolve (Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD) study, an ongoing natural history study designed to inform the development of therapies for facioscapulohumeral muscular dystrophy (FSHD).

The ReSolve study is an observational study run by the FSHD Clinical Trial Research Network (CTRN), a network of medical centers across the U.S. and Europe that aims to validate new clinical outcome assessments and refine trial planning strategies in FSHD.

“Dyne is working to deliver life-changing therapies to patients with serious muscle diseases, including FSHD, by leveraging our muscle-targeted FORCE platform technology,” said Romesh Subramanian, Ph.D., president and chief executive officer of Dyne. “We are proud to contribute to initiatives like the ReSolve study that seek to accelerate the development of new therapeutic options.”

The 18-month longitudinal ReSolve study is enrolling approximately 160 patients with FSHD across eight sites in the U.S., plus an additional 60 patients across three sites in Europe. The specific goals of the study are to validate new clinical outcome assessments and evaluate physiological biomarkers to support the design and implementation of future clinical trials. Around 140 patients have been enrolled in the study so far.

Funding from Dyne will contribute to an expansion of the number of sites and participants in Europe, as well as a muscle biopsy study to advance biomarker development. In addition to Dyne, supporters of the ReSolve study and the CTRN include the National Institutes of Health, Muscular Dystrophy Association, FSHD Society, AFM, Fulcrum Therapeutics and Friends of FSH Research.

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

Back in June Dyne also announced support for END-DM1 (Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1), a natural history study designed to advance the understanding of disease progression in patients with myotonic dystrophy type 1 (DM1) and enable the development of clinical outcome assessments.

END-DM1 is a non-interventional study sponsored by the Myotonic Dystrophy Clinical Research Network (DMCRN), a network of medical centers that aims to support future clinical trials of potential therapies for DM1 through the standardization of testing methods. The END-DM1 study will enroll approximately 650 people with DM1 aged 18-70 across nine DMCRN study sites in the U.S. and seven sites in Europe.

Financial support from Dyne has enabled the expansion of the study to Europe. In addition to Dyne, supporters of END-DM1 include the U.S. Food & Drug Administration (FDA), the Myotonic Dystrophy Foundation, the Wyck Foundation and the Muscular Dystrophy Association.

“At Dyne, we are striving to transform the lives of patients and families affected by DM1 by developing the first disease-modifying therapies,” noted Subramanian in a press release. “As we rapidly advance our development programs, we are proud to support this critical initiative that facilitates a global DM1 patient community and provides the foundation for clinical trial readiness.”

END-DM1 will be the third and largest natural history study sponsored by DMCRN since the network was established in 2012. Building on current knowledge of DM1 pathogenesis and therapeutic targets, the END-DM1 study aims to enhance understanding of patient heterogeneity by characterizing baseline status and disease progression of a larger cohort through selected functional tests and patient-reported outcomes; develop reliable biomarkers of disease severity and therapeutic response using optimized biopsy collection and analysis among a subset of patients; and identify possible genetic modifiers of disease severity through genome-wide association analysis.

“This is an exciting time for the development of targeted RNA-based therapies in DM1, but our understanding of this disease is still growing,” added Nicholas Johnson, M.D., a co-principal investigator for END-DM1 at VCU Health in Richmond, Virginia. “Information collected during this study will allow improved assessment of the efficacy of potential new treatment options, and we encourage patients and caregivers to consider participating.”