Nanocatalytic Gold Treatment Improves Multiple Sclerosis Functional Composite Scores in Phase 2 Studies
Treatment with the bioenergetic catalyst nanocrystalline gold (CNM-Au8; Clene Nanomedicine, Salt Lake City, UT) improved Multiple Sclerosis Functional Composite (MSFC) scores significantly more than placebo. Participants in the trial continued taking disease-modifying treatments approved for the indication relapsing multiple sclerosis (MS).
In the double-blind placebo-controlled phase 2 VISIONARY-MS study (NCT03536559) of nanocrystalline gold, changes in the 4 subscales of the MSFC were compared with baseline scores at weeks 12, 24, and 36. At each time point, there were notable improvements in overall MSFC scores and subscales for those whose were treated with nanocrystalline gold (mixed-effects model; P<.0001 vs baseline). The benefits of nanocrystalline gold are postulated to arise from the positive effect on metabolism to enable remyelination and repair of neurons.
“As VISIONARY-MS and REPAIR-MS continue to progress, we see consistent and complementary results,” said Robert Glanzman, MD, FAAN, chief medical officer of Clene. “Interim data from the REPAIR-MS program provide further evidence for CNM-Au8 to enter the brain and reverse bioenergetic failure, a key driver in the pathophysiology of MS and other neurodegenerative diseases. These mechanistic results provide important support for the updated, blinded interim VISIONARY data analysis, which suggest that CNM-Au8 has the potential to drive clinically meaningful improvements in recognized MS functional endpoints when administered in addition to standard of care. We look forward to the continued advancement of these trials.”
In the ongoing REPAIR-MS (NCT03993171) phase 2 study target engagement of nanocrystalline gold as assessed with high-resolution magnetic resonance spectroscopy (31P-MRS) was estaglished. Catalytic bioenergetic improvements in NAD+/NADH ratio and adenosine triphosphate (ATP) levels, 2 key CNS metabolic biomarkers were also observed.
Rob Etherington, president and chief executive officer of Clene, added, “The results we’ve seen from our MS clinical program to date continue to be very promising. They demonstrate CNM-Au8’s unique mechanism of action as well as its potential to address the unmet need for neuro-reparative MS therapies. Unlike currently approved treatments, CNM-Au8 acts to improve cellular bioenergetics and thus enhance processes that are fundamental to neuronal survival, repair and function. This mechanism is broadly applicable, and leaves CNM-Au8 well positioned to potentially improve the lives of patients living with neurodegenerative disease.”
These data were presented at the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum 2021 (online, February 25-27, 2021).