Neuropore Initiates Phase 1 Clinical Trial in Healthy Volunteers with NPT520-34, a Therapeutic Candidate Aimed at Treating Parkinson’s Disease and Amyotrophic Lateral Sclerosis

SAN DIEGO–(BUSINESS WIRE)–lt;a href="https://twitter.com/hashtag/ALS?src=hash" target="_blank"gt;#ALSlt;/agt;–Neuropore
Therapies, Inc
. announced today that it has initiated a Phase 1
clinical trial in healthy volunteers with NPT520-34. This Phase 1 study
is designed to evaluate the safety, tolerability and pharmacokinetics of
NPT520-34. NPT520-34 is an orally bioavailable, blood-brain barrier
penetrating small molecule. Its mode of action is attenuation of
neuroinflammation and reduction of neurotoxic misfolded proteins in the
central nervous system, key features of neurodegenerative diseases
including Parkinson’s disease (PD) and amyotrophic lateral sclerosis

De Souza
, President and CEO of Neuropore, stated, “We are excited to
start the Phase 1 clinical trial with NPT520-34 which represents
Neuropore’s second therapeutic candidate to enter clinical development.
We have extensively studied NPT520-34 in several transgenic animal
models of neurodegenerative diseases, including PD, ALS and Alzheimer’s
disease (AD). We are encouraged by the broad and robust effects seen in
animal models including decreases in markers of brain neuroinflammation
(microglial and astrocytic activation) and decreases in neurotoxic
misfolded proteins (for example, α-synuclein for PD, superoxide
dismutase 1 for ALS and β-amyloid for AD). We believe NPT520-34
represents a promising new small molecule therapeutic opportunity for
patients with severe unmet needs. In addition to striking effects on
neuroinflammation and neuropathology, which are predicted to slow
disease progression, NPT520-34 treatment in the transgenic animal model
of PD demonstrated preservation of dopamine transporter levels in
striatum and functional improvements in grip strength, gait and balance.
Impaired gait and balance are motor deficits representing major risk
factors for patients with Parkinson’s disease leading to increased
hospitalization and morbidity.”

The single-center Phase 1 study of NPT520-34 is being conducted in up to
48 healthy subjects and will be carried out in two phases: a
single-ascending dose phase and a multiple-ascending dose phase. The
primary objectives of the study are to evaluate the safety,
tolerability, and pharmacokinetics of single and multiple doses of
NPT520-34. An additional arm of the study will examine the
pharmacokinetics of a capsule formulation of NPT520-34 taken with and
without food. Exploratory measures will include measurement of blood
biomarkers indicative of target engagement.

About Parkinson’s disease
Parkinson’s disease (PD) is a
debilitating neurodegenerative disorder afflicting an estimated seven to
ten million patients worldwide. Current treatments for PD are effective
at managing the early motor symptoms of the disease, mainly through the
use of levodopa or dopamine agonists. As the disease progresses and
dopaminergic neurons continue to be lost, these drugs become less
effective at treating the symptoms.

About Amyotrophic Lateral Sclerosis
Amyotrophic Lateral
Sclerosis (ALS) is a devastating progressive neurological disease for
which there is no cure. A little over 5,000 people in the U.S. are
diagnosed with ALS each year with an estimated 16,000 Americans
suffering with the disease at any given time. Only 10% of ALS patients
survive longer than 10 years.

About NPT520-34
NPT520-34 is a clinical stage orally
bioavailable small molecule that reduces astrocytic and microglial
markers of neuroinflammation with robust beneficial effects on
neuropathology and motor function in animal models of Parkinson’s
disease. NPT520-34 also has been shown to reduce the expression of
markers of neuroinflammation and neuropathology in animal models of
amyotrophic lateral sclerosis and Alzheimer’s disease. NPT520-34 is
currently being evaluated for safety, pharmacokinetics and target
engagement in Phase 1 clinical studies in healthy volunteers.
Biomarker-based proof-of-mechanism studies in patients with
neurodegenerative disorders are scheduled to start in 2020.

About Neuropore Therapies, Inc.
Neuropore Therapies Inc. is
a San Diego, California based biopharmaceutical company developing novel
disease modifying small molecule therapeutics for the treatment of
neurodegenerative disorders. Neuropore’s therapeutic candidates block
the neurotoxic oligomeric aggregates of misfolded proteins which are the
primary drivers of disease pathology by preventing the induction of
damaging neuroinflammation mechanisms common to many neurodegenerative
disorders. By targeting the underlying mechanisms by which
neuroinflammation drives a vicious cycle of protein pathology and
neurodegeneration, Neuropore’s therapeutic candidates may have broad
applications in slowing disease progression and improving symptoms
across a wide range of neuroinflammatory / neurodegenerative disorders.

For more information visit www.neuropore.com

Forward looking statements
This press release contains
forward-looking statements based on current information, projections and
assumptions of management. Statements in this press release that are not
strictly historical in nature are forward-looking statements. By their
nature forward-looking statements are not guarantees of future
performance and are subject to risks and uncertainties which may cause
actual results to differ materially from the forward-looking statements
contained in this press release.

Important factors that could result in such differences include the
risks and uncertainties inherent in drug discovery, development,
approval and commercialization, collaborations with others, and the fact
that past results of clinical trials and regulatory decisions may not be
indicative of future trial results or regulatory decisions.


Media Contact Neuropore:
Errol De Souza, Ph.D.
& Chief Executive Officer
T +1 858-273-1831
[email protected]

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