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)-- 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 (ALS).

Errol 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.

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