VISIONARY-MS Trial of CNM-Au8 to End Early Due to Pandemic

VISIONARY-MS, a Phase 2 clinical trial evaluating the safety and effectiveness of CNM-Au8 in people with relapsing multiple sclerosis (MS) with disease-related visual impairment, will conclude early due to COVID-19 pandemic-related challenges.

The release of trial data is expected in the second half of this year, according to the therapy’s developer, Clene Nanomedicine. The company will assess the data collected from up to 48 weeks (just under a year) of clinical visits to evaluate the efficacy and safety profile of CNM-Au8.

Those findings will be used to support further clinical development of the oral therapy for MS.

“On behalf of Clene, I want to thank the investigators, site staff, and, most importantly, the participants and their families for their contribution to the VISIONARY-MS study,” Robert Glanzman, MD, Clene’s chief medical officer, said in a press release. “We will leverage the learnings from VISIONARY-MS to inform the design of our next Phase 2/3 clinical trial in MS.”

CNM-Au8 is an oral liquid suspension of gold nanocrystals designed to increase energy reserves and help clear toxic substances resulting from the metabolism of cells that produce myelin — the fatty protective coating around nerve fibers that is mistakenly targeted by the immune system in MS. The therapy is expected to protect nerve cells and aid in generating new myelin.

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The VISIONARY-MS Phase 2 trial (NCT03536559) is testing the remyelination potential of two doses of CNM-Au8 relative to a placebo for 24 weeks, or nearly six months. So far, the study has enrolled 73 of its planned 150 participants — all relapsing MS patients with chronic vision problems, ages 18 to 55. The study had been expected to finish in September of this year.

People with MS are typically treated with disease-modifying therapies (DMTs), which can compromise the immune system. As a result, MS trials that require regular in-person visits to the clinic have experienced ongoing enrollment challenges and operational problems due to the COVID-19 pandemic.

According to Clene, a recent analysis of interim blinded data from the VISIONARY-MS study demonstrated significant dose-dependent improvements in low contrast letter acuity (LCLA), the trial’s primary goal. LCLA is an eye exam chart with low contrast between the letters and the background that assesses visual disability in MS.

In key secondary measures, improved scores were seen in the averaged components of the modified-Multiple Sclerosis Functional Composite (MSFC) subscales, including the symbol digit modalities test for cognitive abilities, the 9-hole peg test for arm and hand function, and the timed 25-foot walk to assess gait.

The company also announced the expansion of its open-label REPAIR-MS Phase 2 pilot study (NCT03993171), which is evaluating CNM-Au8 in relapsing MS, to include patients with non-active progressive MS, a more severe form of the disease. The company noted that patients with this disease type “currently have limited therapeutic options and high unmet need.”

The trial is assessing the safety and pharmacological properties of CNM-Au8 in up to 30 participants enrolled at the University of Texas Southwestern.

Top-line results showed CNM-Au8 enhanced energy metabolism in the brains of participants. Citing robust target engagement, Clene says it will now recruit non-active progressive MS patients to confirm these findings. Location and contact information can be found here.

CNM-Au8 also is being developed for the progressive neurological diseases amyotrophic lateral sclerosis (ALS) and Parkinson’s disease.