Orpheris' pipeline is focused on the treatment of neuroinflammatory orphan diseases that result in death or severe disabilities, including childhood cerebral andrenoleukodystrophy (ccALD), Parkinson's disease (PD), amyotrophic lateral sclerosis (ALS), Huntington's disease (HD), Rett syndrome and autism spectrum disorders.

The lead product, OP-101, is a new chemical entity containing an approved anti-inflammatory drug, that is effective at reducing inflammation and oxidative stress. OP-101 is selectively targeted to activated microglia and astrocytes in the brain and releases the drug upon internalization.

OP-101 consists of a hydroxyl poly(amidoamine) (PAMAM) dendrimer conjugated to an anti-inflammatory drug with a disulfide bond that is designed to be cleaved only under intracellular conditions. In clinical studies, the free drug is administered IV to ccALD patients before and after bone marrow transplant resulting in increased survival with minimal impact on progressive loss of neurological function. By directly targeting the drug to activated microglia in the brain, we believe that OP-101 will be effective in increasing survival and reducing the loss of neurological function in ccALD patients not eligible for bone marrow transplant.