Neuropore Receives Orphan Drug Designation for NPT520-34 for the Treatment of Amyotrophic Lateral Sclerosis

SAN DIEGO--(BUSINESS WIRE)-- Neuropore Therapies, Inc. announced today that it has received orphan drug designation for NPT520-34 for the treatment of amyotrophic lateral sclerosis or ALS. The orphan drug designation will facilitate the development of NPT520-34 in the treatment of this debilitating medical condition for which limited treatments are available.

In addition, Neuropore reports the successful completion of the initial, single dose, safety and the food effect studies of NPT520-34 in healthy volunteers. NPT520-34 is now being assessed in a multiple dose safety study designed to evaluate the safety, tolerability and pharmacokinetics of repeated doses of NPT520-34 in normal healthy volunteers at potentially therapeutically relevant exposures.

Errol De Souza, President and CEO of Neuropore, stated, “We are very pleased with receiving this orphan drug designation from the FDA’s Office of Orphan Product Development for NPT520-34 for the treatment of ALS, a devastating disease with a very high unmet medical need. Furthermore, we are pleased to have identified safe and tolerable single oral doses that achieve the drug exposures in plasma that are efficacious in animal models of ALS and Parkinson’s disease. We are moving forward with multiple dose studies in healthy volunteers to assess the safety, tolerability and pharmacokinetics of NPT520-34 along with biomarkers before progressing into studies in patients.”

NPT520-34 is an orally bioavailable, blood-brain barrier penetrating small molecule. Its mode of action is to attenuate microglial and astroglial mediated neuroinflammation, resulting in reduced burden of neuropathic proteins such as superoxide dismutase-1, alpha-synuclein and beta-amyloid in animal models of neurodegenerative disorders such as ALS, Parkinson’s disease and Alzheimer’s disease, respectively. Neuropore is currently pursuing NPT520-34 for the treatment of both ALS and Parkinson’s disease.

For information on the NPT520-34-001 trial: NCT03954600

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 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 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.

For more information on the NPT520-34-001 trial: NCT03954600

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.

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.
President & Chief Executive Officer
T +1 858-273-1831


Source: Neuropore Therapies, Inc.

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