CNM-Au8 Exhibits Long-Term Improvements in Multiple Sclerosis

Long-term treatment with Clene Nanomedicine's CNM-Au8 in a Phase 2 clinical trial (VISIONARY-MS) showed sustained improvements in nerve and myelin health for patients with relapsing-remitting multiple sclerosis (RRMS) over three years. CNM-Au8, an oral therapy containing gold nanoparticles, targets nerve cell energy production, a novel mechanism distinct from other MS therapies. The findings support Clene's plans for a Phase 3 trial and potential applications for drug approval.

Clene Nanomedicine's CNM-Au8 Demonstrates Long-Term Improvements in Multiple Sclerosis

Denver, Colorado, April 18, 2024 - Data from the three-year VISIONARY-MS clinical trial presented at the American Academy of Neurology (AAN) 2024 Annual Meeting provide further evidence of the potential therapeutic benefits of Clene Nanomedicine's experimental oral therapy CNM-Au8 in relapsing-remitting multiple sclerosis (RRMS).

Sustained Nerve and Myelin Improvements

Long-term treatment with CNM-Au8 led to sustained improvements in the health of nerves and myelin over three years, according to the findings. This was evident in improvements in nerve function, as measured by visual evoked potentials, and in markers of myelin repair, or remyelination, as measured by magnetic resonance imaging (MRI).

Mechanism of Action

CNM-Au8 is a liquid suspension of gold nanoparticles that is designed to boost nerve cells' energy production. This mechanism is distinct from approved disease-modifying therapies (DMTs) for MS, which work to reduce inflammation in the brain.

Phase 3 Trial Planned

The findings support Clene's plans to launch a Phase 3 trial of CNM-Au8 in MS. Such a trial would provide further evidence of the therapy's efficacy and safety and may support applications toward its approval.

VISIONARY-MS Extension Study

The VISIONARY-MS clinical trial (NCT03536559) initially tested CNM-Au8 against a placebo in 73 people with RRMS who had not had a relapse for at least six months before entering the study. After a year, those given CNM-Au8 had significant improvements in vision, suggesting that the therapy improved the health of nerve fibers in the brain.

Fifty-five participants from the original trial then entered into an extension study (VISIONMS-LTE; NCT04626921), where they were treated with CNM-Au8 for an additional two years, for a total of three years of treatment.

Long-Term Efficacy Data

Data from the extension study showed that the improvements in vision and cognitive function observed after one year of CNM-Au8 treatment were maintained for three years. Additionally, those who switched from a placebo to CNM-Au8 in the extension study saw improvements in these measures.

Improvements in Nerve Damage

The new data presented at AAN included further evidence that the therapy may be repairing damaged optic nerves. Visual evoked potential latency and amplitude measures improved in patients who started on CNM-Au8 in the placebo-controlled part of the trial, suggesting that the damaged optic nerves were repairing.

Remyelination Markers

MRI data also indicated that CNM-Au8 led to long-term increases in markers of myelin repair. Myelin is a fatty substance that wraps around nerve fibers to help them send electrical signals, but is damaged by the misguided inflammatory attacks in MS.

Expert Commentary

"These results are particularly exciting and provide a clear impetus for a Phase 3 study," said Michael Barnett, PhD, a clinical advisor for the trial, during his presentation at AAN. "The development of adjunctive therapies that not only prevent neurodegeneration, but also improve neuronal function with measurable clinical benefit, will fill a major unmet need for people living with MS."

"Observing such a profound clinical benefit with corresponding improvements in physiologic measures utilizing a mechanism that does not target immune system modulation has never been demonstrated in prior multiple sclerosis trials," added Benjamin Greenberg, MD, head of medicine at Clene.

Conclusion

The long-term data from the VISIONARY-MS clinical trial provide further evidence of the potential therapeutic benefits of Clene Nanomedicine's CNM-Au8 in RRMS. The therapy demonstrates sustained improvements in nerve and myelin health and may represent a promising new treatment option for people with this debilitating condition.