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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

Gaithersburg, MD – August 15, 2018 – 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

Contact at Leadiant Biosciences, Inc.:
Joseph M. Wiley, MD, F.A.A.P.
Leadiant Biosciences, Inc.
Vice President, Medical Affairs, Drug Safety, and Pharmacovigilance
Phone: 301-670-2182

Alex Van Rees
SmithSolve LLC on behalf of Leadiant Biosciences, Inc.
Phone: 973-442-1555 ext. 111

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