Altamira Bio has acquired from New Zealand Pharmaceuticals (NZP) an NIH license and cooperative research and development agreements (CRADAs) for development of oral N-acetyl-D-mannosamine (ManNAc).
The value of the license was not disclosed by Altamira parent Fortress Biotech, which announced the deal today.
ManNAc, the precursor compound for the biosynthesis of the sialic acids, will be developed for the treatment of hyposialylation disorders that include hereditary inclusion body myopathy (HIBM or GNE myopathy), a rare genetic disease that causes progressive muscle-wasting and weakness among some 2,000 people worldwide.
ManNAc is the subject of an open-label Phase I/II study for HIBM sponsored by NIH’s National Human Genome Research Institute (NHGRI), in collaboration with NIH’s National Center for Advancing Translational Science (NCATS).
A program of NCATS, Therapeutics for Rare and Neglected Diseases (TRND), supported the completion of two pivotal animal toxicology studies needed to demonstrate drug safety. TRND also generated required data on the manufacturing processes employed to produce the final drug product, and in 2011 began a natural history study of GNE myopathy disease progression. Data from that study was used for supporting a Phase I trial launched the following year.
ManNAc has been demonstrated in human studies to significantly increase circulating levels of sialic acid and has been shown to treat disease in mouse models of both HIBM and kidney diseases, Fortress Biotech said.
A protocol for a Phase I study of the compound’s safety and tolerability in various nephropathies associated with hyposialylation is under IRB review, Fortress Biotech said. ManNAc—which has received the FDA’s orphan drug designation for HIBM—is manufactured by NZP, which will remain Altamira’s exclusive global supplier of the compound as part of the deal.
“Thanks to the work conducted by NIH scientists to date, we have very compelling data on how ManNAc administration could supplement the genetic insufficiency to help these patients to normalize protein sialylation and retain their muscle strength,” Lindsay A. Rosenwald, M.D., Fortress Biotech’s chairman, president and CEO, said in a statement.