Positive results announced from phase 2 trial of oral MS therapeutic
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Clinical-stage biopharmaceutical company Clene Inc. announced proof-of-concept evidence of potential neuroprotective effects of CNM-Au8, an oral therapeutic for patients with stable relapsing-remitting MS.
According to a release from Clene, data from the VISIONARY-MS trial were presented at the 14th Congress of the Pan-Asian Committee for Treatment and Research in Multiple Sclerosis Nov. 24 to 26 in Singapore.
The phase 2, randomized, double-blind, placebo-controlled trial included patients with stable relapsing-remitting MS and chronic optic neuropathy. Participants received 15 or 30 mg per day of CNM-Au8 — an oral suspension of gold nanocrystals developed to restore neuronal health and function — or placebo over 48 weeks. Ninety-two percent of participants also received disease-modifying therapy as standard of care, per the release.
According to Clene, the primary study endpoint — change in low contrast letter acuity compared with placebo at week 48 — demonstrated significant improvement (modified intent-to-treat population, least squares mean difference = 3.13; 95% CI, –0.08 to 6.33). Another outcome, the modified Multiple Sclerosis Functional Composite, also showed significant improvement (mITT population, LS mean difference = 0.28; 95% CI, 0.04-0.52).
Other improvements reported by Clene for CNM-Au8 were multifocal visual evoked potential amplitude and latency, measurements of retinal structure with optical coherence tomography and novel MRI endpoints examining myelin and axonal integrity.
In addition, placebo-treated patients generally worsened across clinical and paraclinical measures during the trial duration, while CNM-Au8 was well-tolerated and no significant safety findings were observed, the release stated.
“Remyelination and neuroprotection are key unmet needs for patients with multiple sclerosis,” Michael Barnett, MBBS, FRACP, PhD, of the University of Sydney in Australia, said in the release. “The phase 2 VISIONARY-MS trial results demonstrated promising efficacy of the cellular energetic nanocatalyst, CNM-Au8, across remyelination and neuroprotection domains. When these results are confirmed by a future, larger phase 3 study, CNM-Au8 would be a remarkable advance for patients with MS as an adjunct to conventional anti-inflammatory DMTs.”
Earlier this year, per the release, the trial was stopped prematurely due to COVID-19 pandemic operational challenges after enrolling just 73 of the 150 planned participants.
“Despite the operational challenges presented by COVID and the primary endpoint marginally exceeding the traditional P = 0.05 statistical threshold, Clene and its MS expert advisers believe these results strongly support the hypothesis that improving brain energetic metabolism results in improved neurological function when CNM-Au8 is administered as adjunct to standard immunomodulatory disease-modifying MS therapies,” Michael Hotchkin, Clene’s chief development officer, said in the release.
Clene plans to initiate a phase 3 study to demonstrate improved global neurological function in patients with progression independent of relapse activity after consulting with regulatory authorities, the company stated in the release.
Editor's Note: This article was updated on Dec. 5, 2022, to correct an affiliation.