Potential Oral Treatment Shows Promise in Early Study in Volunteers, Neuropore Says

Potential Oral Treatment Shows Promise in Early Study in Volunteers, Neuropore Says
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A potential oral therapy for Parkinson’s disease and amyotrophic lateral sclerosis (ALS) was seen to be safe and well-tolerated at various doses in a Phase 1 trial in healthy volunteers, its developer, Neuropore Therapies, announced.

Steps are now underway to allow NPT520-34 to be evaluated in patients, the company said in a press release.

NPT520-34 is a small molecule that, in different animal models of Parkinson’s, was found to reduce levels of markers of brain inflammation and to improve motor function. Likewise, it was shown to ease inflammation and the buildup of toxic proteins in the central nervous system (brain and spinal cord) in animal models of Alzheimer’s disease and ALS.

The medication is administered orally and small enough to cross the blood-brain barrier, the highly selective and semipermeable membrane that encases and protects the brain.

The recently completed and placebo-controlled study (NCT03954600) investigated the safety, tolerability, and pharmacokinetic properties of oral NPT520-34 at multiple doses in 49 healthy volunteers. (Pharmacokinetics is the study of how a medicine is absorbed, distributed, metabolized, and eliminated from the body.)

The trial included an initial single-dose ascending phase, in which a one dose of NPT520-34 (125 mg, 250 mg, 500 mg and 1000 mg) or a matched placebo were given all study participants, with the possibility of incremental adjustments. This was followed by a second phase, in which participants received multiple-ascending doses (250 mg and 500 mg) of the compound, also with the possibility of incremental adjustments, or a placebo.

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

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

Trials of NPT520-34 in patients with neurodegenerative disorders are expected to begin this year.

We believe NPT520-34 represents a promising new small molecule therapeutic opportunity for patients living with Parkinson’s disease and amyotrophic lateral sclerosis.

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.
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Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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