Clene Nanomedicine announced the dosing of the first participant enrolled in the REPAIR-PD study with its lead nanocatalytic therapy, CNM-Au8, for the treatment of Parkinson's disease (PD).
"The objective of the REPAIR-PD Phase 2 study is to demonstrate improvements in brain bioenergetic metabolism in Parkinson's disease patients treated with CNM-Au8. Participants will undergo 31phosphorous magnetic resonance spectroscopy (31P-MRS) imaging to show how treatment with CNM-Au8 results in improvements in brain metabolic and membrane biomarkers," said Robert Glanzman, MD, FAAN, Clene's Chief Medical Officer.
"We are excited to be advancing CNM-Au8 clinically into Parkinson's patients starting with the REPAIR-PD Phase 2 study at UT Southwestern in this key target biomarker study," said Rob Etherington, President and CEO of Clene. "Our preclinical data with CNM-Au8 demonstrated improvements in neuronal bioenergetics which may improve the survival of dopaminergic neurons in patients with PD, thereby helping slow the progression of this devastating disease.”
REPAIR-PD is single-center open label, sequential group, investigator blinded study examining the brain metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who have been diagnosed with PD within three years of screening. Participants drink a 2 oz. dose of the nanocrystal suspension daily each morning for 12 weeks. The objective of this study is to advance pharmacologic understanding of CNM-Au8 treatment effects on central nervous system biomarkers related to bioenergetics, neuronal metabolism, and oxidative stress, as indicators of target engagement for CNM-Au8 in patients with PD.
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CNM-Au8 is a concentrated, aqueous suspension of clean-surfaced faceted nanocrystalline gold (Au) that acts catalytically to support important intracellular biological reactions. CNM-Au8 consists solely of gold atoms organized into faceted, geometrical crystals held in suspension in sodium bicarbonate buffered, pharmaceutical grade water. CNM-Au8 has demonstrated safety in Phase 1 studies in healthy volunteers and both remyelination and neuroprotection effects in multiple preclinical models. Preclinical data presented at scientific congresses demonstrated treatment with CNM-Au8 in neuronal cultures improved survival of dopaminergic neurons, protected neurite networks, decreased intracellular levels of reactive oxygen species, and improved mitochondrial capacity in response to cellular stress, induced by multiple disease-relevant neurotoxins. Oral treatment with CNM-Au8 restored functional behaviors in a rodent model of Parkinson's disease. CNM-Au8 has received regulatory approval to proceed to clinical studies for the treatment of remyelination failure in patients with multiple sclerosis and neuroprotection in patients with amyotrophic lateral sclerosis (ALS) and Parkinson's disease.
Parkinson's disease, a progressive central nervous system disorder, is the second most common neurodegenerative disorder, affecting approximately 1.2% of the world population over the age of 70. PD predominately affects dopaminergic neurons—the neurons in the brain that make dopamine. Loss of dopaminergic neurons results in tremor, slowness, stiffness, difficulty walking, and balance problems. PD may also be associated with non-movement related symptoms, such as constipation, depression, and memory problems.