The FDA has awarded Orphan Drug Designation to GeNeuro’s antibody for the treatment of a rare autoimmune disease following promising Phase IIb results in multiple sclerosis patients.
GeNeuro develops therapies targeting factors encoded by human endogenous retroviruses that are implicated in neurological and autoimmune disorders. The biotech’s drug, GNbAC1, which is being developed for multiple sclerosis (MS), will next be targeted at chronic inflammatory demyelinating polyneuropathy (CIDP), another condition characterized by the destruction of the myelin sheath covering neurons. The drug has been awarded the FDA’s Orphan Drug Designation for CIDP as it affects just 9 people per 100,000 in the US, allowing discussions over an upcoming Phase II study to begin.
Many MS treatments focus on bringing the immune system back under control, which is the case for Roche’s approved CD20 antibody, Ocrevus, and Topas Therapeutics’ approach that harnesses the liver’s capacity to induce immune tolerance. In contrast, GeNeuro’s antibody, GNbAC1, neutralizes a retroviral envelope protein that causes inflammation and lesions in all types and at all stages of multiple sclerosis. In a Phase IIb trial, the highest dose of the antibody reduced inflammation and stimulated remyelination. Rewind Therapeutics is also trying to repair neurons in this way and it raised €15.2M to boost the development of its small molecule candidate.
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