Leadiant Biosciences, Inc. 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.”
(Source: Leadiant Biosciences, Inc.)