STRASBOURG, France and
PHILADELPHIA, July 30, 2020 /PRNewswire/ -- Dynacure, a
clinical stage drug development company focused on improving the
lives of patients with rare and orphan disorders, announced today
that the U.S. Food and Drug Administration (FDA) has granted rare
pediatric disease designation for DYN101, an investigational
antisense medicine designed to modulate the expression of dynamin 2
(DNM2), for the treatment of Myotubular and Centronuclear
Myopathies (CNM).
Under the FDA's rare pediatric disease designation
program, the FDA may grant a priority review voucher to a sponsor
who receives a product approval for a "rare pediatric disease,"
which is defined as a serious or life-threatening disease in which
the serious or life-threatening manifestations primarily affect
individuals aged from birth to 18 years and affects fewer than
200,000 people in the U.S. Subject to FDA approval of
DYN101 for the treatment of CNM, Dynacure would be eligible to
receive a voucher that may be redeemed to receive priority review
for a subsequent marketing application for a different product
candidate or which could be sold or transferred.
"The rare pediatric designation of DYN101 for CNM demonstrates
the FDA's recognition of Myotubular and Centronuclear Myopathies as
serious, life-threatening disorders that affect skeletal muscles
from birth and progress over time," said Leen Thielemans (Ir, PhD, MSc), Chief
Development Officer of Dynacure.
"Our clinical development strategy is to provide benefit to
a wide range of CNM patients, including children affected by this
progressive disease, by modulating DNM2 with DYN101," said
Chris Freitag (M.D.), Chief Medical
Officer of Dynacure. "We are very excited about this new
designation and we expect to open a pediatric study end of 2021 in
both the US and EU."
About Myotubular and Centronuclear Myopathies
Myotubular and Centronuclear Myopathies (CNM) are serious, rare,
life-threatening disorders that affect skeletal muscles from
birth. CNMs derive their name based on the central location
of the muscle fiber nucleus, which is an abnormal finding observed
in muscle biopsies. The disease is driven by mutations in
multiple genes including MTM1, DNM2 and BIN1 and Dynacure
scientists have discovered the link between an increase in DNM2 and
the direct cause of the disease (Cowling et al 2014 JCI).
There are many genetic forms of CNM including X-linked recessive
(XLCNM/ Myotubular Myopathy), autosomal dominant (ADCNM), and
autosomal recessive (ARCNM), which are all associated with poor
prognosis. Centronuclear Myopathies affect between 4,000 and 5, 000
patients in the EU, US, Japan and
Australia1.
About DYN101
DYN101, an investigational antisense oligonucleotide using
Ionis' proprietary antisense technology, is designed to
modulate the expression of dynamin 2 (DNM2) for the treatment of
Myotubular and Centronuclear Myopathies (CNM). Preclinical studies
have demonstrated that DYN101 has the potential to be disease
modifying in CNM, with compelling preclinical efficacy in treating
animal models of XLCNM and ADCNM2,3. Prevention and
reversion of the disease was observed with a clear dose-dependent
improvement in whole body strength and mice survival.
The development plan for DYN101 was designed to be very broad
and it is the only known program being investigated for most CNM
populations, XLCNM and ADCNM. DYN101 has been granted Orphan Drug
designations by the US FDA and EMA.
About the Phase 1 / 2 Study 'Unite-CNM'
(DYN101-C101)
'Unite-CNM' (DYN101-C101) study is a multicenter, ascending dose
study to evaluate the safety, tolerability, pharmacokinetics and
preliminary efficacy of DYN101 in approximately 18 patients greater
than 16 years of age with X-linked (XLCNM) or autosomal dominant
CNM (ADCNM). Enrolled patients will have a run-in period or be
rolled over from an ongoing natural history study, sponsored by the
Institute of Myology in France,
which includes 60 subjects that have XLCNM or ADCNM. While the
Phase 1 / 2 study will primarily focus on finding an optimal dose
of the drug via safety, tolerability and after 12 weeks of
treatment, multiple domains of efficacy will also be assessed in an
exploratory analysis, which include muscular function, respiratory
function and muscle strength. After completing the Unite-CNM study,
Dynacure expects to investigate a potentially registration-directed
Phase 2 / 3 study (all age groups) that would include US and
European sites.
More information can be found at
https://clinicaltrials.gov/ct2/show/NCT04033159?term=dynacure&rank=1.
About Dynacure
Dynacure is a clinical-stage drug development company focused on
improving the lives of patients with rare and orphan diseases. The
Dynacure team leverages its proven track record in rare disease
drug development to build a pipeline of novel drugs. Dynacure
is developing DYN101, an investigational antisense medicine
designed to modulate the expression of dynamin 2 for the treatment
of Myotubular and Centronuclear Myopathies, with Ionis
Pharmaceuticals. The company maintains its headquarters in
Strasbourg, France and a corporate
office in Philadelphia, PA,
USA. Dynacure's investors are Andera Partners, Bpifrance
Large Venture, Bpifrance through its FABS and Fonds Biothérapies
Innovantes et Maladies Rares funds, Idinvest, Ionis
Pharmaceuticals, Kurma Partners, Perceptive Advisors, Pontifax and
funds managed by Tekla Capital Management LLC.
For more information, please visit www.dynacure.com
1 Neuromuscul Disord. 2018 Sep;28(9):766-777. doi:
10.1016/j.nmd.2018.06.012. Epub 2018 Jul
2 Nat Commun. 2017
Jun 7;8:15661. doi:
10.1038/ncomms15661.
3 Proc Natl Acad Sci U S A. 2018 Oct
23;115(43):11066-11071. doi: 10.1073/pnas.1808170115. Epub 2018
Oct 5.
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SOURCE Dynacure