NMD Pharma Receives IND Clearance from the FDA to initiate a Phase
2 Clinical Trial of NMD670 in Charcot-Marie-Tooth disease in the US
NMD Pharma Receives IND Clearance from
the FDA to initiate a Phase 2 Clinical Trial of NMD670 in
Charcot-Marie-Tooth disease in the US
Aarhus, Denmark, 18 June 2024 –
NMD Pharma A/S, a clinical-stage biotech company dedicated to
developing novel and improved treatments for patients living with
severe neuromuscular diseases, today announces that it has received
clearance from the US Food and Drug Administration (FDA) for its
Investigational New Drug (IND) application to initiate a Phase 2
clinical trial, named SYNAPSE-CMT, of NMD670 in patients living
with Charcot-Marie-Tooth (CMT) type 1 and type 2 disease. NMD670 is
a first-in-class, muscle-targeted small molecule inhibitor of the
skeletal muscle specific ClC-1 chloride ion channel.
The Phase 2 clinical trial is a randomized,
double-blind, placebo-controlled study that will evaluate the
efficacy, safety, and tolerability of twice daily orally
administered NMD670 over 21 days in approximately 80 adult patients
with any genetically confirmed CMT1 or CMT2 subtype. Endpoints of
the study include the 6-minute walk test, the 10-meter walk/run
test, and the timed-up-and-go test, among other endpoints. The
trial will take place at clinical sites in both the US and Europe,
and it is expected to start enrolling patients soon.
In June 2023, NMD Pharma announced the results
of ESTABLISH1, an international observational study of
neuromuscular junction function in CMT types 1 and 2, at the
Peripheral Nerve Society Annual Meeting. The results demonstrated
that dysfunction at the neuromuscular junction is an unappreciated
disease characteristic in patients with the inherited neurological
conditions CMT types 1 and 2. The study revealed that nerves in the
process of degeneration in patients with CMT transmit signals to
skeletal muscle with varying degrees of weakness and deficits. The
level of neuromuscular junction transmission deficit in CMT
patients correlated with disease severity and motor function
assessed through a range of clinical measurements of muscle
strength and function.
Thomas Holm Pedersen, Chief Executive
Officer of NMD Pharma, said:
“Charcot-Marie-Tooth disease is a highly debilitating condition.
There is no cure or approved medicines available to treat these
patients, so there is an urgent need to develop treatments. Results
from the ESTABLISH study highlight neuromuscular junction
transmission deficits as a previously unrecognized disease
characteristic in CMT. This gives us confidence that NMD670 has the
potential to work within the skeletal muscles to improve and
maintain clinically meaningful benefits for this underserved group
affected by the disease.”
Allison Moore, CEO of the Hereditary
Neuropathy Foundation (HNF) remarked:
“NMD Pharma’s focus on developing treatments that address the
symptoms of multiple subtypes of CMT represents a vital step toward
dramatically improving patient’s lives. By alleviating the
day-to-day challenges posed by this debilitating condition and
potentially restoring function, NMD Pharma is providing hope and
tangible benefits to those who need it most.”
“This is an exciting moment for the CMT
community,” says Cleary Simpson, CEO of
the CMT Research Foundation (CMTRF). “We are pleased to
see NMD Pharma moving to Phase 2 with NMD670, a treatment with the
potential for improving muscle function in people living with
multiple types of CMT.”
“CMTA is extremely proud to have NMD Pharma
as an Alliance Partner,” said Sue Bruhn,
PhD, Charcot-Marie-Tooth Association (CMTA) CEO. “Their
dedication to pioneering innovative treatments for CMT aligns
perfectly with our mission to bring treatments to those affected by
this devastating disease. We are thrilled with the FDA's IND
clearance for the NMD670 Phase 2 trial in CMT, this pivotal
milestone brings us closer to effective treatments.”
By the end of 2024, NMD Pharma will have three
ongoing Phase 2 trials using its skeletal muscle activation
enhancing therapy in rare neuromuscular diseases characterized by a
high degree of patient impact and unmet need. These include a Phase
2 study of NMD670 for adults living with spinal muscular atrophy
(SMA) type 3, a Phase 2b study of NMD670 in generalised myasthenia
gravis (gMG) both of which have already been initiated, and,
following today’s news, a Phase 2 study of NMD670 in patients with
CMT type 1 and type 2.
1 Observational Study of Neuromuscular Function
in CMT type 1&2 and health controls (ESTABLISH)
(NCT04980807)
END-
Contacts
NMD Pharma A/SThomas Holm
Pedersen, CEOE-mail: contact@nmdpharma.com
ICR ConsiliumMary-Jane Elliott
/ Ashley Tapp / Lindsey Neville E-mail:
NMDPharma@consilium-comms.com Tel: +44 (0)20 3709 5700
About NMD PharmaNMD Pharma A/S
is a clinical-stage biotech company developing a first-in-class
platform of small molecule therapies selectively and directly
targeting the skeletal muscle chloride ion channel (ClC-1) for the
treatment of severe neuromuscular disorders. The Company was
founded on more than 15 years of muscle physiology research with a
focus on regulation of skeletal muscle excitability under physical
activity. NMD Pharma has built a world-leading muscle
electrophysiology platform leveraging the in-depth know-how of
muscle physiology and muscular disorders, small molecule
modulators, enabling technologies and tools as well as in vivo
pharmacology models for discovering and developing proprietary
modulators of neuromuscular function. The Company has built
significant clinical and development expertise as its programmes
have progressed through the clinic. NMD Pharma has raised ~€155
million from investors including Novo Holdings, Lundbeckfonden
BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital.
Find out more about us online at http://www.nmdpharma.com.
About NMD670NMD670 is NMD
Pharma’s lead development program which is being developed in 3
Phase 2 clinical studies. It is a first-in-class small molecule
inhibitor of the skeletal muscle specific chloride ion channel
(CIC-1). NMD Pharma has demonstrated that CIC-1 inhibition enhances
weakened neuromuscular transmission and restores skeletal muscle
function, and this novel treatment approach has demonstrated
compelling preclinical and clinical data in animal models of
myasthenia gravis, spinal muscular atrophy and a range of other
neuromuscular disorders. NMD670 has also been granted orphan-drug
designation by the U.S. FDA for treatment of gMG.
About Charcot-Marie-Tooth disease
(CMT)CMT encompasses a group of hereditary sensory and
motor neuropathies that cause damage to peripheral nerves and their
neuromuscular junction. Damage caused by CMT worsens slowly over
time and can result in substantial reductions in mobility,
independence and quality of life due to muscle weakness, fatigue
and atrophy across skeletal muscle groups such as the legs, feet,
arms, hands and potentially leading to diaphragm weakness and
paralysis. CMT is often broadly grouped into demyelinating (CMT
type 1) and axonal (CMT type 2) based on nerve conduction studies,
but there are more than 160 subtypes of CMT based on their genetic
causes, clinical features and progression patterns. CMT affects
approximately 136,000 individuals in the United States and 3.2
million worldwide, with first symptoms typically appearing during
adolescence or early adulthood.
About The Charcot-Marie-Tooth
Association (CMTA)CMTA is a community-focused,
community-driven 501(c)(3) nonprofit organization with a mission to
support the development of new treatments for CMT, to improve the
quality of life for people with CMT, and, ultimately, to find a
cure. As the leading global philanthropic funder of CMT research,
CMTA unites the community with clinicians and industry experts to
accelerate the advancement of treatments, with investments of more
than $24 million since 2008. For more information,
visit https://www.cmtausa.org.
About CMT Research Foundation
(CMTRF)The CMT Research Foundation (CMTRF) is a
patient-led, non-profit focused on delivering treatments and cures
for CMT. The foundation identifies significant obstacles or
deficiencies impeding progress toward a cure and seeks out
collaborators to address these issues. To date, CMTRF has funded 24
projects, of which 8 are completed. Of those 6 completed projects,
5 have clinical candidates. CMTRF's mission to invest in promising
science with high potential of leading to treatments and cures was
proven effective and ground-breaking when DTx Pharma, with a
CMTRF-backed program as its lead candidate, was acquired by
Novartis for $1 billion. Founded by two patients who are driven to
expedite drug delivery to people who live with CMT, the 501(c)(3)
federal tax-exempt organization is supported by personal and
corporate financial gifts. For more information, visit
https://cmtrf.org.
About Hereditary Neuropathy Foundation
(HNF)
The Hereditary Neuropathy Foundation (HNF), a
patient advocacy and research non-profit with a mission to increase
awareness and accurate diagnosis of Charcot-Marie-Tooth (CMT),
support people living with CMT and their families with critical
information to improve quality of life, and fund research that will
lead to treatments and cures. HNF’s Therapeutic Research in
Accelerated Discovery (TRIAD) is a collaborative effort with
academia, government and industry to develop treatments for CMT. As
part of TRIAD’s research consortium, the Global Registry for
Inherited Neuropathies (GRIN) was established as a natural history
study to collect and analyze patient-reported data and clinical
scales, including the ONLS, CMT-FOM, CMTPedS, CMTInfS and the
collection and curation of genetic reports and biospecimens. Most
recently, GRIN’s natural history studies include digital health
technologies for the development of innovative biomarkers for all
CMT subtypes. For more information, visit
https://www.hnf-cure.org.