Market Overview

Leadiant Biosciences to Collaborate with National Institutes of Health on Therapy in Late-Stage Development for GNE Myopathy


Multi-Center Collaborative Clinical Trial Planned to Test Efficacy of
Oral-N-acetyl-D-Mannosamine (ManNAc)

Company Applies its Drug Development, Clinical and Regulatory
Capabilities in Rare Diseases

Leadiant Biosciences, Inc. today announced it has entered into a license
agreement and cooperative research and development agreement (CRADA)
with the National Institutes of Health (NIH) to develop oral
N-acetyl-D-mannosamine (ManNAc) for the potential treatment of
individuals with GNE myopathy. GNE myopathy is a rare genetic disorder
that causes progressive skeletal muscle atrophy, with an onset in young
adulthood, leading to severe disability.

Leadiant Biosciences will partner with investigators at the National
Human Genome Research Institute (NHGRI), one of 27 institutes that make
up NIH, and various clinical sites around the U.S., including Brigham
and Women's Hospital in Boston, MA, to plan and carry out a multi-center
clinical trial of ManNAc for the treatment of individuals with GNE
myopathy. The Company is working with the co-principal investigator from
NHGRI, Dr. Nuria Carrillo, and the co-principal investigator from
Brigham and Women's Hospital, Dr. Anthony Amato. These clinical sites
are all part of the National Institute of Neurological Disorders and
Stroke (NINDS) Network for Excellence in Neuroscience Clinical Trials
(NeuroNEXT). The primary objective of this trial is to evaluate the
efficacy of ManNAc in individuals with GNE myopathy as measured by the
ability of the investigational therapy to slow the progression of muscle
strength decline, compared with placebo. The study is sponsored by NHGRI
and funded by cooperative
agreement U01AR070498
from the National Institute of Arthritis and
Musculoskeletal and Skin Diseases (NIAMS) and NINDS. The trial will be
conducted at the NIH Clinical Center in Bethesda, MD and various
NeuroNEXT sites, in partnership with Leadiant Biosciences.

"We are excited to collaborate with NIH in the development of ManNAc,
with the aim of serving individuals with GNE myopathy, a severe and
debilitating disease," said Michael Minarich, chief executive officer of
Leadiant Biosciences, Inc. "By leveraging the clinical and scientific
expertise of NIH and the drug development capabilities of Leadiant
Biosciences, we are hopeful that this collaboration will bring us a step
closer to delivering a much-needed therapy to individuals living with
GNE myopathy."

ManNAc is believed to work by restoring production of sialic acid and
increasing protein sialylation in skeletal muscle cells, thereby
addressing a deficiency in GNE myopathy. ManNAc has been evaluated in
two early phase NIH trials in individuals with GNE myopathy. Data
gathered from these trials, together with information from an ongoing
natural history study of individuals with GNE myopathy at NIH, provided
the basis for this clinical trial design.

"Currently there are no treatments available for GNE myopathy, a
progressive genetic disease that weakens muscles and reduces mobility,"
commented William Gahl, M.D., Ph.D., clinical director of the NHGRI.
"Based on the preclinical and clinical data gathered to date, ManNAc may
be a solution for this underserved patient community. In partnership
with our colleagues in the NIH NeuroNEXT network and our collaborators
at Leadiant Biosciences, a company with a long history of collaborating
with government agencies and academic institutions to develop therapies
for people with rare diseases, we aim to advance this program for the
GNE myopathy community."

About GNE Myopathy

GNE myopathy is a rare genetic (inherited; autosomal recessive) disorder
that causes progressive skeletal muscle atrophy and weakness. The
disorder has an estimated prevalence of 1-10 per one million people, but
it is more common in certain ethnic groups. It results from mutations in
a gene called GNE, which produces an enzyme that is responsible
for a key step in the production of a monosaccharide called sialic acid.
Symptoms of GNE myopathy usually appear between the ages of 20-40 years,
and include foot drop and difficulty walking, followed by progressive
skeletal muscle weakness. Eventually the disease progresses to involve
all skeletal muscles, leading to the use of a wheelchair and, in some
cases, dependence on a caregiver. DNA sequencing for mutations in the GNE
gene confirms the diagnosis of GNE myopathy, which was previously also
known as hereditary inclusion body myopathy (HIBM), inclusion body
myopathy type 2 (IBM2), distal myopathy with rimmed vacuoles (DMRV), or
Nonaka myopathy.

About ManNAc

ManNAc (N-acetyl-D-mannosamine) is an uncharged monosaccharide and a
precursor molecule in the production of sialic acid within cells.
Individuals with GNE myopathy have decreased GNE enzyme activity, and do
not produce enough ManNAc and, subsequently, sialic acid. This
deficiency results in decreased sialic acid bound to membrane proteins
on the surface of skeletal muscle cells; this is thought to be the cause
of the muscle wasting that characterizes GNE myopathy.

About NeuroNEXT

NINDS's Network for Excellence in Neuroscience Clinical Trials, or
NeuroNEXT, was created to conduct studies of treatments for neurological
diseases through partnerships with academia, private foundations, and
industry. NeuroNEXT sites include many of the leading medical centers in
the U.S. (
The goals of NeuroNEXT include testing of promising neurological
therapies in Phase 2 clinical trials, optimizing drug development time
and cost components through an established clinical trials
infrastructure, and the coordination of public/private sector efforts by
leveraging NINDS's existing relationships with academic investigators
and patient advocacy groups. A clinical coordinating center for
NeuroNEXT is led by Dr. Merit Cudkowicz and is based at Massachusetts
General Hospital and the data coordinating center is led by Dr. Chris
Coffey at the University of Iowa.

About Leadiant Biosciences, Inc.

Leadiant Biosciences is a research-based pharmaceutical company that
dedicates considerable scientific and financial resources to the
research, development, and distribution of novel and effective therapies
for patients affected by rare disease. At Leadiant Biosciences, our
legacy of serving patients is as strong as the future we envision. For
more information, visit

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