Top-line results from the Phase II REPAIR trial show a gold nanocrystal suspension, CNM-Au8®, significantly improved brain energetic metabolism in patients with Parkinson’s disease and multiple sclerosis.
Top-line results from the Phase II REPAIR trial show a gold nanocrystal suspension, CNM-Au8®, significantly improved brain energetic metabolism in patients with Parkinson’s disease (PD) and multiple sclerosis (MS).
Based on these new findings, clinical-stage biopharmaceutical company Clene says its lead candidate CNM-Au8 shows the potential to “drive meaningful neurological functional improvements” in patients with neurodegenerative disorders.
CNM-Au8, Clene’s lead drug candidate, is an aqueous suspension comprising catalytically active, clean-surfaced and faceted gold nanocrystals. The company explained in a statement that the therapy’s catalytically active nanocrystals fuel “critical cellular energy” that ultimately produce brain reactions enabling neurorepair and remyelination. This is reportedly accomplished via increases in neuronal and glial resilience to stressors relevant to neurologic diseases.
The REPAIR clinical trial program was designed to assess the effects of CNM-Au8® on brain energy metabolites in two studies of PD (REPAIR-PD) and MS (REPAIR-MS). Researchers in the studies imaged patients with non-invasive 31phosphorous magnetic resonance spectroscopy before and after ≥12 weeks of daily oral treatment with CNM-Au8. The primary endpoint included the mean change in the brain NAD+/NADH ratio.
In the pre-specified integrated analysis of the REPAIR-PD and REPAIR-MS trials, there was a statistically significant increase in the primary endpoint by 0.589 units (10.4%) after a 12-week treatment course of CNM-Au8 (p=0.037). Additionally, there was a significant increase in NAD+ fraction (p=0.026) and a significant decrease in the NADH fraction (p=0.026). There were also “consistent statistical trends” showing possible improvement in the NAD+/NADH ratio in the REPAIR-PD (p=0.11) and REPAIR-MS (p=0.14) studies.
“We believe the REPAIR program represents a critical breakthrough for Clene,” said Clene’s chief medical officer, Robert Glanzman, M.D., F.A.A.N., in a statement. He noted that the findings also demonstrated significant rebalancing of brain beta-ATP level, a treatment response suggesting improved ATP efficiency. “Our next step will be to demonstrate that these brain energetic changes result in clinically meaningful results in patients with PD and MS.”
Overall, the treatment-emergent adverse events (AEs) reported in the studies were mostly mild and unrelated to the drug. Treatment with CNM-Au8 was generally well tolerated, and no serious AEs or treatment discontinuations associated with AEs were reported.
“Clene is a company driven to pioneer the development of cellular energy-enhancing nanotherapeutics for the treatment of neurodegenerative diseases,” said Rob Etherington, chief executive officer of Clene. “We believe the study results also strongly support Clene’s ongoing Phase II and Phase III clinical programs investigating how CNM-Au8’s neuro-reparative and neuroprotective properties may impact disease progression in amyotrophic lateral sclerosis, MS, and PD, and may be broadly applicable to the treatment of neurodegenerative diseases.”
The company noted the study results for REPAIR-PD will soon be presented at the International Parkinson and Movement Disorders Society, MDS Virtual Congress 2021 meeting, which will be held between September 17 and 22, 2021. Findings from the REPAIR-MS trial will also be presented at the next 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis, from October 13 through 15, 2021.
Etherington told BioSpace in an email that the company is nearly fully enrolled for its part in the Healey ALS Platform Trial Phase III program in amyotrophic lateral sclerosis. Future studies with CNM-Au8 might also include patients with PD and non-active progressive MS.
