Neuropore Receives Orphan Drug Designation for ALS Treatment

Neuropore Therapies 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.

"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," Errol De Souza, President and CEO of Neuropore, said. "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.

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