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Dyne Therapeutics Announces Support for END-DM1 Natural History Study of Patients with Myotonic Dystrophy Type 1 (DM1)

June 17, 2019 Off By BusinessWire

Study sponsored by Myotonic Dystrophy Clinical Research Network
will advance understanding of disease progression and enable
development of clinical outcome assessments
Patients with DM1 aged 18-70 encouraged to enroll at study sites in
U.S. and Europe

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Dyne
Therapeutics, a biotechnology company pioneering targeted therapies
for patients with serious muscle diseases, today announced its support
for END-DM1, a natural history study 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
(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 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 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.

“At Dyne, we are striving to transform the lives of patients and
families affected by DM1 by developing the first disease-modifying
therapies,” said Romesh Subramanian, Ph.D., President and CEO of Dyne.
“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
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
Develop reliable biomarkers of disease severity and therapeutic
response using optimized biopsy collection and analysis among a subset
of patients
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,” said 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.”

For more information about END-DM1, including study requirements and a
list of participating sites, please visit the Myotonic
Dystrophy Foundation website.

About Dyne Therapeutics
Dyne Therapeutics is pioneering
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 (DM1) in addition to programs
for Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular
dystrophy (FSHD). Dyne launched in 2019 and is based in Cambridge, Mass.
For more information, please visit www.dyne-tx.com.