Neuropore gets FDA orphan drug designation for NPT520-34 for ALS

Neuropore Therapies has secured orphan drug designation for NPT520-34 from the US Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS).

The California-based biopharma company said that the orphan drug designation will enable the development of NPT520-34 in the treatment of the debilitating medical condition for which there are not many treatments options.

Apart from achieving the regulatory milestone, Neuropore Therapies has wrapped up the initial, single-dose, safety and the food effect studies of the molecule in healthy volunteers.

NPT520-34 is being evaluated in a multiple-dose safety study designed to assess its safety, tolerability, and pharmacokinetics of repeated doses at potentially therapeutically relevant exposures in normal healthy volunteers.

Neuropore gets FDA orphan drug designation for NPT520-34 for Amyotrophic Lateral Sclerosis. Photo courtesy of Frank Gaillard/Wikipedia.org.

Errol De Souza – President and CEO of Neuropore Therapies said: “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.”

According to Neuropore Therapies, NPT520-34 is an orally bioavailable small molecule that can penetrate the blood-brain barrier.

It has been designed to reduce the microglial and astroglial mediated neuroinflammation. Due to this action, the molecule can bring down the burden of neuropathic proteins like superoxide dismutase-1, beta-amyloid, and alpha-synuclein in animal models of neurodegenerative disorders like ALS, Alzheimer’s disease, and Parkinson’s disease, respectively.

Currently, Neuropore Therapies is aiming to have NPT520-34 approved for the treatment of amyotrophic lateral sclerosis and Parkinson’s disease.

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