Neuropore’s NPT520-34 Given FDA’s Orphan Drug Designation for ALS Treatment

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by Patricia Inacio PhD |

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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to NPT520-34, an investigational anti-neuroinflammatory molecule, for the treatment of amyotrophic lateral sclerosis (ALS), Neuropore Therapies announced.

“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, Neuropore’s president and CEO, said in a press release.

Neurodegenerative diseases such as ALS are linked to a generalized inflammation in the brain. Researchers believe that blocking inflammation could help slow down and manage ALS symptoms.

NPT520-34 is a small oral molecule designed to cross the blood-brain barrier — a highly selective membrane that shields the central nervous system with its cerebrospinal fluid from the general blood circulation — and reduce inflammation in the brain.

Preclinical studies done in ALS, Parkinson’s, and Alzheimer’s disease animal models have shown that the anti-inflammatory action of NPT520-34 decreases the production of neurotoxic proteins such as superoxide dismutase-1, alpha-synuclein, and beta-amyloid.

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“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,” De Souza said.

Orphan drug designation will support the faster development of NPT520-34 for the treatment of ALS and other neurodegenerative diseases. This designation is provided to candidate therapies that show promise in the diagnosis or treatment of rare diseases. It facilitates treatment marketing and provides financial incentives for clinical testing.

NPT520-34’s safety and tolerability is currently being tested in a single-center Phase 1 clinical trial (NCT03954600) in healthy volunteers.

The study is being conducted in two separate phases: an initial single-dose ascending phase, in which NPT520-34 is administered in a single dose to all participants, with the possibility of incremental adjustments; and a second multiple-ascending dose phase, in which the compound is administered at different doses, again with the possibility of incremental adjustments.

The trial is testing ascending doses of 125–1,000 milligrams and repeated doses of NPT520-34. The single-dose stage has already been completed, and the trial is now moving to the multiple-ascending dose phase.

Neuropore is aiming to launch a biomarker-based study to assess the mechanisms behind the therapeutic effects of NPT520-34 in 2020.

“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,” De Souza said.