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Jul
22
ragupathyrenganathan
Altamira Bio buys NIH license & CRADAs to develop ManNac for rare genetic disorders
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Fortress Biotech, Inc. announced that its subsidiary, Altamira Bio, acquired from New Zealand Pharmaceuticals Ltd (NZP),  a license from the National Institutes of Health (NIH) and Cooperative Research and Development Agreements (CRADAs) for the development of oral N-acetyl-D-mannosamine (ManNAc), a key compound in the sialic biosynthetic pathway, for the treatment of hyposialylation disorders.

Hyposialylation disorders are a group of pathologies associated with abnormal sialylation of tissues, characteristic of Hereditary Inclusion Body Myopathy (HIBM), also known as GNE myopathy – a rare genetic disorder which causes progressive muscle-wasting and weakness.

New Zealand Pharmaceuticals Ltd (NZP) manufactures ManNAc (DEX-M74) and will remain the exclusive global supplier of ManNAc to Altamira Bio.

“HIBM, a severe debilitating muscle-wasting disease with unmet medical need, has a clearly established genetic link,” said Dr. Lindsay A. Rosenwald, chairman, president and chief executive officer of Fortress Biotech.

“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. We are pleased to be working with NIH scientists in the NHGRI division who are leaders in this field in bringing this potential therapy to patients. We are also encouraged by recent discoveries on the possible involvement of hyposialylation in kidney diseases and testing ManNAc in patients with nephropathies for improving their kidney function.”

HIBM is a rare genetic disease inflicting approximately 2000 people worldwide. Disease symptoms emerge in adulthood and slowly lead to progressive muscle weakness. Most patients develop symptoms in their early 20s and eventually require a wheelchair as their arm, hand and leg muscles weaken. The disease is caused by mutations in GNE gene, which codes for a rate-limiting enzyme in the sialic acid synthesis pathway. Dysfunction in GNE in HIBM leads to impaired sialylation of certain glycoproteins and glycolipids believed to be responsible for the gradual muscle deterioration.

ManNAc is an intermediate in sialic acid biosynthesis generated by functional GNE protein. Supplementation of ManNAc to the cells with dysfunctional GNE protein bypasses the need for GNE function and enables protein sialylation. ManNAc has been demonstrated in human studies to significantly increase circulating levels of sialic acid and is shown to treat disease in mouse models of both HIBM and kidney diseases. The FDA has provided orphan designation for ManNAc in HIBM. ManNAc is currently under investigation in an open label phase I/II study for the treatment of the rare muscle-wasting disease HIBM, also known as GNE Myopathy. A protocol for a phase I study to further investigate ManNAc safety and tolerability in various kidney diseases (nephropathies) associated with hyposialylation is under IRB review.

Altamira Bio is a development stage company focused on the clinical development and commercialisation of N-acetyl-D-mannosamine and other therapies for orphan/rare disorders.

Fortress Biotech is a biopharmaceutical company dedicated to acquiring, developing and commercialising novel pharmaceutical and biotechnology products.



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