This included the first 34 participants from the VISIONARY-MS study, which is currently ongoing with the orally delivered suspension of clean-surfaced, faceted gold nanocrystals.
Robert Glanzman, MD, chief executive officer, Clene Nanomedicine
Robert Glanzman, MD
Patients with stable relapsing multiple sclerosis (MS) who have chronic optic neuropathy treated with CNM-Au8—an orally delivered suspension of clean-surfaced, faceted gold nanocrystals—experienced gains in best-corrected low contrast letter acuity (BC-LCLA), according to interim data from the phase 2 VISIONARY-MS study (NCT03536559).1
The data were presented by Robert Glanzman, MD, chief executive officer, Clene Nanomedicine, the product’s developer, at the 2020 America’s Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum, February 27-29, 2020, in West Palm Beach, Florida. This dataset included the first 34 participants from the study, which is currently ongoing.2
"These preliminary results are encouraging as we progress to achieve the critical unmet therapeutic goals of remyelination and neuroprotection for patients with MS," Glanzman said in a statement. "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.”
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At baseline, the mean Expanded Disability Status Scale (EDSS) score was 1.9 (standard deviation [SD], 1.4), with the cohort’s average time from the last relapse being 3.5 (SD, 2.4) years. The majority of the participants were treated with disease-modifying therapy (DMT), with 50% (n = 17) receiving treatment with a monoclonal antibody, 38% (n = 13) taking oral agents, and 6% (n = 2) taking injectables. Only 6% (n = 2) were not on a DMT.
At Week 36 of treatment, those treated with either 15- or 30-mg CNM-Au8 also showed notable improvements in the 3 modified Multiple Sclerosis Functional Composite (MSFC) sub-scales assessed: the Symbol Digit Modalities Test (SDMT), 9-Hole Peg Test (9HPT), and Timed 25-foot Walk (T25W). The full unblinded results from the study are anticipated in mid-2021.
Additionally, the treatment appears to be safe and well-tolerated, with no serious adverse events (AEs) occurring. The most frequently reported AEs were headache, upper respiratory infection, and sore throat.
“The consistent median improvements observed across the MSFC functional domains in the population of participants in VISIONARY-MS are exciting,” Glanzman added. “We are gratified to share the details of the study and these early efficacy data with the MS community at one of the most prominent conferences committed to research and treatment for this devasting disease."
Interim safety data revealed 110 treatment-emergent AEs, of which 86% (n = 94) were of mild or moderate severity. All told, 80% (n = 80) were deemed unrelated, while 7% (n = 8) were possibly related to treatment. The remaining 13% (n =14) have yet to be determined.
In phase 1 studies, the 15 mg and 30 mg per day doses were determined to be well-tolerated doses, with human exposures surpassing those demonstrating preclinical efficacy. According to Clene Nanomedicine, CNM-Au8 acts as a nanocatalyst by enhancing cellular bioenergetics, showing robust activity in multiple preclinical models of neuroprotection and remyelination. VISIONARY-MS is presently at approximately 25% of the target enrollment, with 8 clinical sites in Australia and several in North America opening enrollment soon.
For more coverage of ACTRIMS 2020, click here.
1. Beadnall H, Hotchkin MT, Klistorner A, et al. VISIONARY-MS: A Phase 2 Clinical Trial Of Catalytic Gold Nanocrystals, CNM-Au8, For The Treatment Of Chronic Optic Neuropathy. Presented at 2020 ACTRIMS Forum. February 27-29, 2020; West Palm Beach, FL. Abstract P079.
2. Clene Nanomedicine Presents Blinded Interim Data from the VISIONARY-MS Phase 2 Study [press release]. Salt Lake City, UT; Clene Nanomedicine; Published February 27, 2020. Accessed March 1, 2020. prnewswire.com/news-releases/clene-nanomedicine-presents-blinded-interim-data-from-the-visionary-ms-phase-2-study-301012036.html.