Orpheris to Present Data on OP-101 at 2019 Keystone Symposia Conference on Neurodegenerative Diseases
Orpheris, a clinical stage neuroscience company leveraging a novel platform technology that enables the selective delivery of therapeutic agents across the blood barrier into reactive microglia, today announced that it will present data on its lead candidate OP-101 and preclinical GCPII inhibitor at the 2019 Keystone Symposia Conference on Neurodegenerative Diseases: New Insights and Therapeutic Opportunities, on June 16-20, 2019, in Keystone, Colorado.
In addition, Jeffrey Cleland, Ph.D., Executive Chair and Co-founder of Orpheris, has been selected to present a short talk at the conference entitled: "OP-101: Human Safety and Pharmacokinetics of a Microglia-targeted Systemic Therapy for Neuroinflammation."
OP-101 is a dendrimer-drug conjugate being developed as a microglia-targeted treatment of neuroinflammation associated with CNS disorders. Neuroinflammation, mediated by reactive glia, is considered a key driver of disease progression in several CNS disorders including childhood cerebral adrenoleukodystrophy (ccALD), Amyotrophic Lateral Sclerosis (ALS) and Parkinson's Disease.
Details of the presentations are listed below:
Title: OP-101: Human Safety and Pharmacokinetics of a Microglia-targeted Systemic Therapy for Neuroinflammation
Poster Number: 1025
Session: Poster Session 1
Date: Monday, June 17, 2019 (7:30 PM ? 10:00 PM MDT)
Location: Keystone Resort, Colorado Rockies Ballroom
Title: Systemic therapy with microglial-targeted drug, OP-101, decreases neuroinflammation in a Parkinson's Disease Model
Poster Number: 2009
Session: Poster Session 2
Date: Tuesday, June 18, 2019 (7:30 PM ? 10:00 PM MDT)
Location: Keystone Resort, Colorado Rockies Ballroom
Title: Targeted dendrimer-2PMPA therapy inhibits glial GCPII and improves cognition in a mouse model of multiple sclerosis
Poster Number: 2023
Session: Poster Session 2
Date: Tuesday, June 18, 2019 (7:30 PM ? 10:00 PM MDT)
Location: Keystone Resort, Colorado Rockies Ballroom
Short Talk: OP-101: Human Safety and Pharmacokinetics of a Microglia-targeted Systemic Therapy for Neuroinflammation
Session: Translating Therapeutics for Neurodegeneration to the Clinic
Date: Wednesday, June 19, 2019 (8:30 AM ? 11:30 AM MDT)
Presenter: Jeffrey Cleland, Ph.D.
Location: Keystone Resort, Grays/Longs Peak Room
Orpheris' technology platform consists of hydroxyl dendrimers that specifically target reactive microglia and reactive astrocytes associated with neuroinflammation resulting in demyelination and neuronal loss. The hydroxyl dendrimers rapidly distribute throughout the body after systemic administration, do not bind to tissues or proteins, are non-toxic, and are excreted intact in the urine. The company has also used this platform to enhance the penetration of hydrophilic therapeutic compounds that otherwise have limited brain penetration.
"We are excited to present this data on our dendrimer platform, and our lead candidate OP-101 which is in clinical development for the treatment of neuroinflammation in ccALD and early enabling studies in Parkinson's. This data suggests that systemic hydroxyl dendrimers (synthetic nanostructured polymers) enhance delivery of drugs to reactive glia at sites of brain inflammation. OP-101 crosses the impaired blood-brain barrier, selectively targets reactive microglia, and leads to significant improvement in neurobehavioral outcomes in multiple preclinical models with neuroinflammation," said Jeffrey Cleland, Ph.D., Executive Chair and Co-founder of Orpheris.
"These results show that neuroinflammation caused by reactive microglia is present in the majority of neurological diseases and suppression of the reactive microglia has the potential to be a potent therapeutic target across a number of indications including Parkinson's Disease, multiple sclerosis and other neurodegenerative disorders," added Dr. Barbara Slusher, Ph.D., MAS, Professor of Neurology, Psychiatry, Neuroscience, Medicine, and Oncology at Johns Hopkins School of Medicine and the Director of Johns Hopkins Drug Discovery program.
About Orpheris
Orpheris is a clinical stage neuroscience company leveraging a novel platform technology from Johns Hopkins that enables the selective delivery of therapeutic agents across the blood barrier into reactive microglial cells. In a number of neurodegenerative diseases such as ALS, Parkinson's, and Alzheimer's, reactive microglia are shown to be responsible for the increased neuroinflammation, subsequent neuronal loss and disease progression in these patients.
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