Neuropore’s Potential ALS Oral Treatment Found to be Safe and Well-Tolerated in Healthy Volunteers, Phase 1 Trial Shows

A recently completed Phase 1 trial studying NPT520-34 — an investigational small molecule being developed by Neuropore Therapies for the treatment of Parkinson’s disease and amyotrophic lateral sclerosis (ALS) — has shown the candidate to be safe and well-tolerated in a group of healthy volunteers.

The single-center study (NCT03954600) included 49 healthy adults who were randomly assigned single or multiple ascending doses of NPT520-34 to test its safety, tolerability, and pharmacokinetics, which assesses how a compound behaves inside the body with respect to its absorption, distribution, metabolism, and excretion.

Accordingly, treatment protocol was carried out in two phases. In an initial single-dose ascending phase, patients received a single administration of NPT520-34, in doses ranging from 125 to 1,000 milligrams (mg).

In the multiple-ascending part, patients received the compound in multiple administrations, also in ascending doses. In both phases, patients had the possibility of incremental dose adjustments.

In addition to safety and tolerability, the researchers will examine if the capsule formulation can be taken with food, and assess blood biomarkers that indicate therapy success. 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.

“We are excited to complete the Phase 1 clinical trial with NPT520-34. We believe NPT520-34 represents a promising new small molecule therapeutic opportunity for patients living with Parkinson’s disease and amyotrophic lateral sclerosis,” Doug Bonhaus, PhD, CEO and chief scientific officer of Neuropore, said in a press release.

“NPT520-34 proved to be safe and tolerable at all doses tested, including those believed to be therapeutically relevant,” he added.

NPT520-34 is a small oral molecule designed to cross the blood-brain barrier (a semipermeable membrane that shields the brain and spinal cord) and reduce inflammation in the brain, a common characteristic of neurodegenerative diseases such as ALS, Parkinson’s, and Alzheimer’s disease.

Animal studies indicate that NPT520-34 decreases the production of neurotoxic proteins, including superoxide dismutase-1, alpha-synuclein, and beta-amyloid, which have individually been linked to these neurodegenerative disorders. In theory, NPT520-34-mediated reduction in brain inflammation may help manage symptoms or even slow down disease progression.

In August, NPT520-34 was granted orphan drug status by the U.S. Food and Drug Administration for the treatment of ALS patients. Steps are now underway to allow NPT520-34 to be evaluated in patients.

“The results of this study support moving forward to a safety study in patients. Our team is currently evaluating the optimal study design and patient population for the next study,” Bonhaus added.