The Food and Drug Administration (FDA) has granted Orphan Drug designation to an investigational therapy for the treatment of amyotrophic lateral sclerosis (ALS). 

NPT520-34 (Neuropore Therapies) is an orally bioavailable, blood-brain barrier penetrating small molecule which has been shown to reduce markers of brain neuroinflammation by decreasing neurotoxic misfolded proteins (superoxide dismutase 1) in animal models.

A study evaluating a single dose of NPT520-34 in healthy individuals has been successfully completed, according to the Company. Another trial is currently assessing repeated doses in healthy subjects. The Company is also exploring the use of NPT520-34 for Parkinson disease treatment.

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“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,” said Errol De Souza, President and CEO of Neuropore. 

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