NHGRI Seeking Partner for Phase II Trial for Kidney and Diabetes Therapeutic

ManNAc rescues glomerular hyposialylation and proteinuria in rodents with glomerulopathy. Glomerular hyposialylation occurs in 25-60% of human glomerulopathies. ManNAc could be therapeutic for human glomerular disease.

Inventors at the National Human Genome Research Institute (NHGRI) discovered that N-acetylmannosamine (ManNAc) can be used for therapeutic purposes. ManNAc therapy given orally shows long-term safety and biochemical efficacy.

ManNAc is a small uncharged physiological molecule that crosses membranes readily. It is an intracellular precursor of sialic acid synthesis. NHGRI discovered that it can be used to treat certain kidney diseases (such as those involving proteinuria and hematuria) that are a result of a lack of sialic acid.

This therapeutic could be used to treat kidney disorders due to sialic acid deficiency, including but not limited to minimal change disease glomerulopathy, focal segmental glomerulosclerosis, and membranous nephropathy in humans. ManNAc therapy might also be used to treat diabetic nephropathy or diabetes.

NHGRI is seeking a partner for a Phase II clinical trial of ManNAc therapy. Previous clinical research and the Phase I clinical trial found that:

  • ManNAc is easy to administer to patients.
  • Long-term administration has been shown to be safe and well-tolerated in humans.

Other advantages of this technology include that there are many issued patents in the U.S., Canada, Europe, Japan, and Israel for this indication. There are also extensive published and unpublished preclinical data for the kidney indication available.

NHGRI is looking for both a licensee and a clinical Cooperative Research and Developments Agreement (CRADA) collaborator for this therapeutic. The Phase II study is currently being developed and will be held at NIH. NHGRI will provide their expertise, recruit patients for the study, and is open to transferring their Investigational New Drug (IND) filing.

If you are interested in partnering with NHGRI for a Phase II Clinical Trial of ManNAc therapy, please see this abstract for further information and the licensing contact: Treating Kidney Disorders and Diabetic Nephropathy with N-acetyl mannosamine (ManNAc).