Antigen-Specific Therapy for Anti-MAG Neuropathy Gets Orphan Drug Status

The Food and Drug Administration (FDA) has granted Orphan Drug designation to PN-1007 (PPSGG; Polyneuron Pharmaceuticals) for the treatment of anti-myelin-associated glycoprotein (MAG) neuropathy.

Anti-MAG neuropathy is a rare, chronic disorder of the peripheral nervous system that is caused by monoclonal IgM autoantibodies directed against the HNK-1 (human natural killer-1) epitope found on myelin of peripheral nerves. PN-1007 mimics the natural HNK-1 carbohydrate epitope and prevents the binding of these pathogenic antibodies to myelin.

“PN-1007 directly targets and eliminates the autoantibodies that cause the disease, which we believe could prevent demyelination and protect the nerves,” said Ruben Herrendorff, PhD, CEO and co-founder of Polyneuron. “Data published recently in the Journal of Neurochemistry supported this hypothesis by showing that PN-1007 was able to inhibit the binding of anti-MAG IgM antibodies to myelin of nonhuman primate nerves.”

Additionally, a dose titration study in mice showed that the intravenous administration of PN-1007 efficiently removed all anti-MAG IgM antibodies. Data from this study will support dose selection in an upcoming phase 1/2a study, which is expected to start later this year.

For more information visit polyneuron.com.