New Zealand Pharmaceuticals collaborating with the National Institutes of Health on the ManNAc trial - Dex-M74

There has been  quite alot of news on research progress this week with HIBM.  I feel absolutely forntunate to  have met Dr. Gahl and the exceptional staff/researchers at NIH and to see the progress with this study.  For those who are/were involved with the Natural History Study of HIBM, well here is good news. As always, I have attached the link at the bottom of this posting.

New Drug Development Partnership

National Human Genome Research Institute (NHGRI) (Bethesda, MD; www.genome.gov) and New Zealand Pharmaceuticals Ltd (NZP) (Palmerston North, New Zealand; www.nzp.co.nz) have partnered on a drug development project. The goal is to reduce or halt the progression of a rare disorder, Hereditary Inclusion Body Myopathy (HIBM) by treating affected patients with a small molecule therapeutic drug. HIBM primarily affects distal muscle tissue, and due to dramatically decreased muscle strength, most HIBM patients must use a wheelchair by the time they are in their 30s. NZP has licensed NHGRI’s patent portfolio related to treating HIBM and other muscle wasting diseases, as well as kidney diseases related to hyposialylation, with a monosaccharide N-Acetyl-D-mannosamine (ManNAc), also known as DEX-M74. DEX-M74 is one of the first molecules to enter development in the Therapeutics for Rare and Neglected Diseases (TRND) program (http://nctt.nih.gov/trnd) at the National Institutes of Health (NIH). NZP and TRND are currently collaborating to complete needed pre-clinical studies for treating HIBM with DEX-M74 - these studies will be the basis of an investigational new drug application (IND) to be submitted to the Food and Drug Administration (FDA). Once the IND goes into effect, a phase I/II clinical trial is scheduled to begin at the NIH Clinical Center. That study will be led by HIBM expert and NHGRI Clinical Director, Dr. William Gahl, M.D., Ph.D.

http://www.nzp.co.nz/index.php/news/7-news/30-new-drug-development-partnership.html