Investigational ALS Treatment NPT520-34 Begins Phase 1 Trial in Healthy Volunteers

NPT520-34, an investigational small molecule for the treatment of Parkinson’s disease and amyotrophic lateral sclerosis (ALS), is being tested on healthy individuals in a new Phase 1 clinical trial, its developer Neuropore Therapies has announced.

Brain inflammation is a common feature among many neurodegenerative disorders, including Parkinson’s, Alzheimer’s disease, and ALS. For that reason, there has been an increasing interest in therapies that are able to minimize or prevent neuroinflammation.

NPT520-34 has been shown to reduce the levels of brain inflammation markers and toxic proteins in the central nervous system (CNS, which comprises the brain, brainstem, and cerebellum) of animal models of Parkinson’s, Alzheimer’s, and ALS.

The compound is administered orally, and is small enough to be able to cross the blood-brain barrier, a highly selective, semipermeable membrane that isolates the brain from the blood that circulates in the body.

NPT520-34’s safety, tolerability, and pharmacokinetic properties are now being tested in up to 48 healthy volunteers participating in a single-center Phase 1 clinical trial. (Pharmacokinetics studies how a drug is absorbed, distributed, metabolized, and eliminated from the body.)

“We are excited to start the Phase 1 clinical trial with NPT520-34 which represents Neuropore’s second therapeutic candidate to enter clinical development. We have extensively studied NPT520-34 in several transgenic animal models of neurodegenerative diseases, including [Parkinson’s disease], ALS and Alzheimer’s disease,” Errol De Souza, PhD, president and CEO of Neuropore, said in a press release.

“We are encouraged by the broad and robust effects seen in animal models including decreases in markers of brain neuroinflammation (microglial and astrocytic activation) and decreases in neurotoxic misfolded proteins (for example, alpha-synuclein for Parkinson’s, superoxide dismutase 1 for ALS, and beta-amyloid for Alzheimer’s),” he added.

The trial will have two distinct phases: an initial single-dose ascending phase, in which NPT520-34 will be administered at a single dose in all participants, with the possibility of incremental adjustments; and a second multiple-ascending dose phase, in which the compound will be administered at different doses, again with the possibility of incremental adjustments.

The study’s primary objective will be to assess the safety, tolerability and pharmacokinetic properties of single and multiple doses of the compound. The pharmacokinetic properties of a capsule formulation of NPT520-34 taken with and without food, will be tested separately in another group of healthy subjects. Exploratory outcomes will include the assessment of blood biomarkers indicative of therapy success.

Phase 1 trials assessing the properties of NPT520-34 in patients with neurodegenerative disorders should start in 2020.

“We believe NPT520-34 represents a promising new small molecule therapeutic opportunity for patients with severe unmet needs,” De Souza said.