- Clene Inc. reported new preclinical data on its drug candidate CNM-Au8 for Parkinson’s disease.
- The therapy improved mitochondrial health, restored cellular metabolism, reduced inflammation, and normalized gene expression in dopaminergic neurons.
- Findings were presented at the Michael J. Fox Foundation’s H2 Therapeutics Stewardship Meeting in New York City.
- CNM-Au8 previously showed positive energetic and metabolic effects in a Phase 2 Parkinson’s trial, aligning with the new preclinical results.
- The treatment demonstrated no toxicity in neuronal models, consistent with safety data from over 1,000 patient-years in ALS and MS trials.
- Clene plans to design a Phase 2 clinical study for Parkinson’s disease while advancing ALS and MS programs.
Clene (NASDAQ: CLNN) and its wholly owned subsidiary, Clene Nanomedicine Inc., a late clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases, has presented encouraging preclinical findings for its lead investigational therapy, CNM-Au8, in Parkinson’s disease. The data, announced earlier in September 2025, highlight the drug’s potential to address key cellular and energetic deficits that drive disease progression (https://ibn.fm/EECHU).
The results were unveiled at the Michael J. Fox Foundation’s H2 Therapeutics Stewardship Meeting in New York City. Researchers reported that CNM-Au8 improved mitochondrial health, reduced harmful inflammation, restored energy metabolism, and normalized disrupted gene expression in dopaminergic neurons derived from Parkinson’s patients.
The study was carried out by Associate Professor Jerome Mertens in collaboration with Dr. Fred Gage of the Salk Institute, and Clene’s Vice President of Translational Medicine, Karen S. Ho, Ph.D. Using skin cells collected from Parkinson’s patients, scientists directly converted them into dopaminergic neurons, the same type of brain cells most vulnerable in the disease.
Importantly, the model retained age-related features from patients, offering a more accurate representation of disease processes compared with traditional laboratory systems. The cohort included neurons from 14 familial Parkinson’s disease (“fPD”) patients, 8 sporadic PD (“sPD”) patients, and 13 healthy controls.
Across both familial and sporadic Parkinson’s disease neurons, CNM-Au8 delivered several notable improvements:
- Mitochondrial health: In fPD neurons, CNM-Au8 improved mitochondrial membrane potential, increased mitochondrial volume, and reduced damaging reactive oxygen species. Milder but similar effects were seen in sporadic PD neurons.
- Inflammation reduction: The therapy lowered inflammatory proteins such as CD40 and CXCL10, helping reduce neuroinflammation, a driver of disease progression.
- Restored metabolism: CNM-Au8 improved the NAD+/NADH ratio, a key measure of cellular energy, and corrected significant portions of dysregulated metabolites, particularly in energy and nucleotide pathways.
- Gene expression normalization: Treatment reversed many of the gene expression abnormalities associated with Parkinson’s disease, bringing patterns closer to healthy control neurons.
- Safety: The compound did not show toxicity toward dopaminergic neurons at any dose. This finding is consistent with safety data from ongoing amyotrophic lateral sclerosis (“ALS”) and multiple sclerosis (“MS”) trials, where CNM-Au8 has accumulated more than 1,000 patient-years of exposure without major concerns.
“Parkinson’s disease has complex and multifactorial causes, but mitochondrial dysfunction, chronic inflammation, and disrupted metabolism are consistent hallmarks,” said Dr. Mertens of the Salk Institute. “These results, generated in an advanced model that reflects both the genetics and the aging process of PD, add to the growing body of evidence that CNM-Au8’s catalytic metabolic effects on mitochondrial health, energy metabolism, neuroinflammation, and gene expression could have broad therapeutic potential for the treatment of Parkinson’s disease and other neurodegenerative disorders.”
Clene’s pipeline focuses on neurodegenerative conditions driven by mitochondrial dysfunction and energetic failure. CNM-Au8, an oral suspension of gold nanocrystals, is designed to restore energy metabolism and neuronal resilience. The therapy has already advanced into late-stage testing in other diseases. A Phase 2 Parkinson’s trial previously showed that oral CNM-Au8 altered key brain energy metabolites (NAD+, NADH, ATP) in patients, supporting the preclinical findings now presented.
Benjamin Greenberg, M.D., Clene’s Head of Medical, noted that the company is preparing a Phase 2 clinical study of CNM-Au8 in Parkinson’s, while continuing late-stage ALS trials and discussions with the FDA regarding a Phase 3 program in multiple sclerosis.
“CNM-Au8 has a unique nanocatalytic mechanism of action that favors its application to many neurodegenerative diseases that have mitochondrial dysfunction and energetic failure at their core,” said Greenberg. “We are grateful to the MJFF for supporting this work, and to the Salk Institute for such a meaningful collaboration. These promising preclinical results strengthen the body of evidence supporting further clinical development of CNM-Au8 for the treatment of Parkinson’s disease.”
For more information, visit the company’s website at www.Clene.com.
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