The National Multiple Sclerosis Society awarded more than $339,000 to Clene Nanomedicine to support clinical tests into its lead candidate CNM-Au8 in treating multiple sclerosis (MS) by protecting nerve cells and restoring myelin.

The grant was awarded under the National MS Society’s Fast Forward program, which aims to support research organizations working to develop new therapies and diagnostic tools for MS.

CNM-Au8 is the first of a new class of neurotherapeutic medicines being developed by Clene that work through an innovative nanocatalytic mechanism. The therapy is made of gold nanocrystals that contribute to and support the normal activity of several cellular reactions.

In particular, CNM-Au8 was shown to prevent the death of nerve cells that control movement in MS animal models. It also showed an ability to help cells overcome the bioenergetic imbalance induced by the disease, as well as to support the production of myelin — the protective layer of neurons that is destroyed in MS.

“We are very pleased to have been awarded a Fast Forward research grant from the National Multiple Sclerosis Society for the development of CNM-Au8, the lead asset in our clean-surfaced gold nanocrystal therapeutic pipeline. We believe the unique mechanism of CNM-Au8 to improve cellular bioenergetics represents a promising new approach for the treatment of MS as a remyelinating agent,” Rob Etherington, president and CEO of Clene, said in a press release.

Phase 1 trials in healthy volunteers found that CNM-Au8 is safe, and several preclinical studies demonstrated that it has both remyelination and neuroprotection effects.

Supported by these early safety and efficacy data, Clene is exploring CNM-Au8’s potential in treating MS, as well as Parkinson’s disease and amyotrophic lateral sclerosis (ALS) in several ongoing Phase 2 clinical trials.

“CNM-Au8 may help improve myelin integrity and restore function of patients with MS – outcomes presently being investigated in the ongoing VISIONARY-MS Phase 2 study,” Etherington said.

VISIONARY-MS (NCT03536559), underway in Australia, is exploring the potential of CNM-Au8 to induce remyelination in optical nerve cells in MS patients with chronic vision problems related to their disease.

The Phase 2 trial plans to enroll 150 adults with clinically confirmed relapsing-remitting MS (RRMS), who will be randomly assigned to one of two oral and once daily doses of CNM-Au8 (15 or 30 mg suspension in water) or a matching placebo for up to 48 weeks.

Researchers will assess changes in patients’ visual acuity, as a measure of improved remyelination, as well as changes in the speed of communication between the eye and visual cortex.

Clene expects to announce results of the VISIONARY-MS study by early 2021.

The Fast Forward grant will support studies into CNM-Au8’s impact on several biological markers in patients in the VISIONARY-MS study, the company said.

“We’re pleased to provide funding to advance our understanding of biomarkers related to lipid metabolism, bioenergetics and oxidative stress, not only as potential indicators of target engagement for CNM-Au8, but also to expand the biomarker repertoire available to the MS field,” said Mark Allegretta, PhD, vice president of research at the National MS Society.

Clene is also preparing, in collaboration with the University of Texas Southwestern Medical Center, another Phase 2 trial called REPAIR-MS (NCT03993171). This open-label trial plans to include 24 adults with RRMS diagnosis within the last 15 years, recruited at that clinical site, who will be given increasing doses of CNM-Au8 for 12 consecutive weeks. Contact information is available here.

Following treatment, all participants will undergo magnetic resonance spectroscopy, a non-invasive technique to measure biochemical and tissue changes in the brain.