Orpheris to Present Data on OP-101 at 2019 Keystone Symposia Conference on Neurodegenerative Diseases

REDWOOD CITY, Calif.–(BUSINESS WIRE)–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.

Contacts

Investors
Matt Brewer
Orpheris, Inc.
[email protected]
303-717-9446

Media
Kimberly Ha
KKH Advisors
[email protected]
917-291-5744

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