Novel Treatment for ALS Gains Orphan Drug Status

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.

“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. 

For more information visit Neuropore.com.