Clene Advances CNM-Au8 Toward Potential ALS Approval with FDA Meetings and Biomarker Analysis
核心洞察
Clene plans to submit a New Drug Application for CNM-Au8 in ALS (搜索) under the accelerated approval pathway by the fourth quarter of 2025, following constructive FDA feedback on its statistical analysis plan.
The company will analyze neurofilament light (搜索) biomarker data from its NIH-sponsored expanded access program in Q4 2025, intended to support the NfL (搜索) changes previously demonstrated in the HEALEY ALS (搜索) Platform Trial.
A second Type C meeting with the FDA is scheduled for Q3 2025 to review long-term survival benefits from CNM-Au8 30 mg treatment compared to controls from the HEALEY ALS (搜索) Platform Trial.
Clene Inc. (搜索) is advancing its investigational therapy CNM-Au8 toward a potential New Drug Application submission for amyotrophic lateral sclerosis (搜索) (ALS (搜索)) by the end of 2025, following productive discussions with the U.S. Food and Drug Administration (搜索) regarding its accelerated approval strategy. The late-stage biopharmaceutical company announced second quarter 2025 financial results and provided updates on its neurodegenerative disease programs.
FDA Engagement for ALS Program
Following constructive FDA feedback on its proposed statistical analysis plan, Clene plans to analyze neurofilament light (搜索) (NfL (搜索)) biomarker data from its National Institute of Health-sponsored expanded access program early in the fourth quarter of 2025. The analysis will examine NfL changes following 9 months of treatment as the primary analysis and after 6 months of treatment as supportive analysis.
"We look forward to engaging with the FDA in our upcoming meeting this quarter focused on the extensive survival data that CNM-Au8 has generated in ALS (搜索) patients," said Rob Etherington, President and CEO of Clene. "These meetings and biomarker analyses represent the final steps to our potential submission of an NDA under the accelerated approval pathway for ALS by the end of 2025."
The analyses are intended to provide supportive data of the NfL (搜索) change previously demonstrated in the HEALEY ALS (搜索) Platform Trial double-blind period following 6 months of treatment with CNM-Au8. A second Type C meeting with the FDA is scheduled for the third quarter of 2025 to review long-term survival benefit from CNM-Au8 30 mg treatment compared to concurrently randomized controls from another HEALEY ALS Platform Trial regimen.
Multiple Sclerosis Program Advances
In April, Clene presented new evidence of remyelination and neuronal repair with CNM-Au8 treatment at the American Academy of Neurology (搜索) Late-Breaking Science Session. The presentation, titled "Physiologic and Anatomical Evidence of Neuronal Repair and Remyelination from the Long-Term Open-Label Extension of the Phase 2 VISIONARY-MS (搜索) Trial," highlighted significant and clinically meaningful improvements in cognition and visual function.
The improvements were supported by corresponding objective biomarkers, including advanced MRI Diffusion Tensor Imaging and multi-focal Visual Evoked Potential assessments, which confirmed anatomical and physiological improvements indicating remyelination and neuronal repair in the brains of MS (搜索) patients treated with CNM-Au8 during the long-term extension of VISIONARY-MS.
The company plans to hold an end-of-Phase 2 Type B meeting with the FDA in the third quarter of 2025 to review results from the Phase 2 VISIONARY-MS (搜索) trial and discuss the planned Phase 3 study focusing on cognition improvement as an adjunct to standard-of-care MS therapies.
CNM-Au8 Mechanism of Action
CNM-Au8 is an oral suspension of gold nanocrystals developed to restore neuronal health and function by increasing energy production and utilization. The catalytically active nanocrystals drive critical cellular energy producing reactions that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors. The therapy improves central nervous system cells' survival and function via a mechanism that targets mitochondrial function (搜索) and the NAD pathway (搜索) while reducing oxidative stress.
Financial Position
Clene's cash and cash equivalents totaled $7.3 million as of June 30, 2025, compared to $12.2 million as of December 31, 2024. The company expects that its resources as of June 30, 2025, together with $1.9 million raised in equity and a $1.5 million increase in its convertible debt facility subsequent to June 30, 2025, will be sufficient to fund operations into the first quarter of 2026.
Research and development expenses were $3.5 million for the quarter ended June 30, 2025, compared to $4.2 million for the same period in 2024. The year-over-year decrease was primarily related to cost saving initiatives and decreased manufacturing personnel due to the conclusion of various clinical programs, partially offset by increased expenses related to ALS (搜索) clinical programs and planning activities for the RESTORE-ALS Phase 3 clinical trial.
Clene reported a net loss of $7.4 million, or $0.78 per share, for the quarter ended June 30, 2025, compared to a net loss of $6.8 million, or $1.06 per share, for the same period in 2024.