Clene Nanomedicine shared early results of the VISIONARY-MS trial, suggesting that CNM-Au8 — an investigational remyelinating therapy — leads to “notable” trends in better vision, as well as benefits in mobility and manual function in relapsing multiple sclerosis (MS) patients with chronic vision problems.
These findings were presented during the Joint NAIMS-IMSVISUAL Symposium at Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum 2020, running Feb. 27–29 in Florida, by Robert Glanzman, MD, Clene’s chief medical officer.
His presentation is titled “VISIONARY-MS Study: Phase 2 Design Rationale, Baseline Data, and Interim Blinded Results.“
It consists of a solution of gold (Au) nanoparticles with a patented crystal structure that act to support important energy-related reactions within cells, a process that Clene calls nanocatalysis.
By doing so, CNM-Au8 is designed to increase energy reserves within neurons and myelin-producing cells (oligodendrocytes), while decreasing toxic metabolic byproducts. This process is expected to improve neuronal survival and function, and to support the ability of oligodendrocytes to create new myelin.
Preclinical tests in mice demonstrated that CNM-Au8 stimulates the production of new myelin, and increases the number of myelin-wrapped nerve fibers in the brain and spinal cord, allowing animals to recover motor skills.
VISIONARY-MS (NCT03536559) is an ongoing Phase 2 trial investigating the efficacy and safety of CNM-Au8 as a remyelinating and neuroprotective treatment for adults with vision problems as a result of replacing MS, which includes relapsing-remitting and secondary progressive disease.
Eligible patients must have chronic optic neuropathy (damage to the optic nerve), with visual impairment and no episodes of acute optic neuritis within six months prior to entering the trial, as well as non-active disease (defined as no MS relapses) within the previous three months.
Up to 150 patients (ages 18 to 55) are being randomly assigned to CNM-Au8 at low-dose (15 mg) or high-dose (30 mg), or to a placebo, every day for six months (24 weeks). Of note, concomitant immunomodulatory MS therapies are allowed.
The study’s primary efficacy measure is improvement in low-contrast vision as determined by low-contrast letter acuity (an eye exam using a chart with low contrast between its letters and its background) after 24 weeks of treatment.
Secondary endpoints include electrophysiologic measures of remyelination (mfVEP, a vision test that measures the speed of conduction of electrical signals among neurons of the visual system), cognition, disability levels, mobility, and upper extremity function (arm and hand dexterity), as well as magnetic resonance imaging (MRI) of brain lesions, optic nerve exams, and other visual tests. These trial goals will be measured after 24 weeks and through to possibly 48 weeks of treatment.
Full study results are expected by mid-2021.
At ACTRIMS, Glanzman presented preliminary, blinded data from the first 34 participants (21 women and 13 men; mean age, 37.7) enrolled in the study, who have been treated with CNM-Au8 for up to 36 weeks.
Results showed “notable median improvements” in their low-contrast letter acuity, as well as in standard MS functional/clinical scores of gait (assessed by Timed 25-Foot Walk test), arm and hand function (measured by 9-Hole Peg Test), and cognitive function (measured by Symbol Digit Modalities Test).
Safety data to date indicates that CNM-Au8 is well-tolerated, and most adverse effects are mild — namely headache, upper respiratory infection, and sore throat. No serious adverse effects related to the treatment were reported.
“These preliminary results are encouraging as we progress to achieve the critical unmet therapeutic goals of remyelination and neuroprotection for patients with multiple sclerosis,” Glanzman said in a press release.
“These data add to the growing body of clinical evidence demonstrating that CNM-Au8, a suspension of catalytic, clean-surfaced, faceted gold nanocrystals, has the unique ability to improve remyelination and provide axonal neuroprotection,” Glanzman added. “The consistent median improvements observed across the MSFC functional domains [mobility, arm/hand function, and cognitive function] in the population of participants in VISIONARY-MS are exciting.”
VISIONARY-MS is currently enrolling patients at sites across Australia, with multiple sites in North America expected to soon open. The trial is supported by the National Multiple Sclerosis Society. Contact and site information is available here.
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