Clene has recently presented updated trial results for its Visionary MS Phase II trial, in which its lead pipeline candidate, CNM-Au8, is being investigated as a treatment for relapsing forms of multiple sclerosis (MS) with chronic optic neuropathy*. The agent has a first-in-class mechanism of action within the MS portfolio, as it is the first drinkable treatment based on a nanocrystalline gold suspension that has reached late-stage development for MS. Its position on the market would be as an adjuvant therapy to other existing treatments, which are administered with the aim of slowing MS progression, says GlobalData, a leading data and analytics company.

In comparison to currently marketed and late-stage pipeline MS agents, CNM-Au8 doesn’t target disease progression or relapse rate. Instead, the main endpoints of the Phase II trial are to investigate visual and neurological functions induced by remyelination and neuroprotection of the brain and spinal cord. Topline results of the Visionary trial were presented Clene in August 2022, showing a significant improvement in the low contrast letter acuity score relative to placebo (least squares mean difference of 3.13) after 48 weeks. Updated results from the Visionary trial then demonstrated a significant improvement in multi-focal visual evoked potential across both eyes by a mean difference of 7.9%.

Barbora Salcman, Neurology Analyst at GlobalData, comments: “It is definitely refreshing to see a treatment whose primary purpose is not to improve progression of the disease itself, but to support remyelination in MS patients. Improving nerve cell function and survival can help MS patients in a broad range of problems, such as vision improvement, as chronic vision problems are experienced by around one in five MS patients. In this subset, CNM-Au8 therefore has promising potential as an adjacent therapy for patients who are already on disease modifying therapies, especially as the agent exhibits a good safety profile, with common side effects being headache and upper respiratory infection.”

While clinical data seems promising for CNM-Au8, key opinion leaders (KOLs) interviewed by GlobalData are not completely convinced about the agent, as yet. They expect CNM-Au8 to be an interesting addition to the MS portfolio, however, they would like to see more mechanistic data and clear overall benefits the agent can bring to the patients.

Salcman continues: “These sentiments may partly reflect the limited sample size of the Visionary trial, which achieved enrollment of only 72 patients out of an expected 150, due to the COVID-19 pandemic complications, leading to the pre-specified significance threshold at p=0.1 before the submission to the FDA. The Phase III study would therefore need to demonstrate strong mechanistic data for the KOLs to recommend the agent to their patients.”

*CNM-Au8 is also being investigated as an adjuvant therapy for its potential to enhance brain function and reduce cell death in trials for MS (REPAIR-MS), as well as amyotrophic lateral sclerosis (ALS) (NCT05299658) and Parkinson’s disease (PD) (REPAIR-PD).