20 June 2019 - - US-based biotechnology company Dyne Therapeutics is supporting END-DM1, a natural history study to advance the understanding of disease progression in patients with myotonic dystrophy type 1 and enable the development of clinical outcome assessments, the company said.
END-DM1 (Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1) 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 standardisation of testing methods.
The END-DM1 study will enroll approximately 650 people with DM1 aged 18-70 across nine DMCRN study sites in the US and seven sites in Europe. Financial support from Dyne Therapeutics enables the expansion of the study to Europe.
In addition to Dyne, supporters of END-DM1 include the FDA, the Myotonic Dystrophy Foundation, the Wyck Foundation and the Muscular Dystrophy Association.
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 will aim 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
It will also seek to develop reliable biomarkers of disease severity and therapeutic response using optimized biopsy collection and analysis among a subset of patients.
Finally, the study will dentify possible genetic modifiers of disease severity through genome-wide association analysis.
Dyne Therapeutics is developing therapies that target muscle tissue with unprecedented precision to restore muscle health. The company's FORCE platform delivers oligonucleotides and other molecules to skeletal, cardiac and smooth muscle to treat a range of serious muscle diseases.
Dyne is advancing a treatment for myotonic dystrophy type 1 in addition to programs for Duchenne muscular dystrophy and facioscapulohumeral muscular dystrophy. Dyne launched in 2019 and is based in Cambridge, Mass.