Clene Inc. (NASDAQ: CLNN) Receives FDA Guidance on Accelerated Approval Pathway for CNM-Au8(R) for ALS

• The FDA asked for additional Neurofilament Light (“NfL”) data from the company’s three Expanded Access Protocols to support earlier clinical trial findings.
• The company has already begun collecting and analyzing this data, with plans to complete the process in Q2 2025 and then submit a New Drug Application (“NDA”) for CNM-Au8 by mid-2025.
• A follow-up meeting with the FDA will be held early next year to discuss and finalize the statistical analysis plan for the EAP NfL biomarker analyses.
• The company is meanwhile preparing a confirmatory Phase 3 trial (“RESTORE-ALS”) to evaluate the survival benefit of CNM-Au8 with initial participant enrollment set to begin before NDA submission.

Clene (NASDAQ: CLNN) and its wholly owned subsidiary, Clene Nanomedicine Inc., a late clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases, including amyotrophic lateral sclerosis (“ALS”) and multiple sclerosis (“MS”), has received additional written guidance and a roadmap from the U.S. Food and Drug Administration’s (“FDA”) Division of Neurology 1 regarding a potential accelerated approval pathway for lead drug candidate CNM-Au8 in ALS (https://ibn.fm/8VQGo).

CNM-Au8, an oral suspension of gold nanocrystals, works by improving cellular energy production and utilization, which is critical for maintaining neuronal health. The drug candidate has already been shown to improve central nervous system cells’ survival and function via a mechanism that targets mitochondrial function and the nicotinamide adenine dinucleotide (“NAD”) pathway while reducing oxidative stress. By targeting mitochondrial dysfunction, CNM-Au8 can provide neuroprotection and promote remyelination, potentially altering the course of neurodegenerative conditions.

The FDA guidance on a potential path to meet the regulatory standard for substantial evidence of effectiveness supporting accelerated approval came following an in-person November 1 Type C meeting with the DN1 where the company presented additional clinical trial data and analyses. The FDA recommended that Clene leverage additional Neurofilament Light (“NfL”) data from its three Expanded Access Programs (“EAPs”) to support earlier findings.

Clene intends to follow the FDA’s recommendation to provide data from the ongoing EAPs and believes that it can address the FDA’s requests. The company plans to complete the additional NfL biomarker collection and analyses to support an NDA submission for CNM-Au8 during the second quarter of 2025:

• NfL biomarker analyses: Present evidence of NfL reductions in participants enrolled in the three FDA-authorized compassionate use EAPs.
• Survival pharmacometric modeling: Analyze NfL and related disease-specific biomarkers to evaluate their association with clinical survival benefits and observed clinical changes in Phase 2 trial data.
• ALS-specific biomarker insights: Examine additional ALS-specific biomarkers to further support the pharmacodynamic activity of CNM-Au8 as a treatment for ALS.

Clene will have another meeting with the FDA in early 2025 to review and finalize its statistical analysis plan for the EAP NfL biomarker analyses. The FDA is expected to review whether NfL can serve as a likely surrogate endpoint for the effects of CNM-Au8 in ALS and whether the magnitude of change observed on NfL in patients treated with CNM-Au8 is likely to predict clinical benefit for ALS.

The FDA’s guidance was welcomed by Rob Etherington, President and CEO of Clene, who said the company was grateful for the regulatory body’s willingness to consider how available EAP data may support existing clinical study data to allow for the review of an accelerated regulatory pathway for CNM-Au8.

“Together with the survival and supportive biomarker data generated thus far, the drug’s benign safety profile, and the emerging EAP NfL data, we look forward to continued discussions with the Agency,” Etherington added. “Clene plans to include the additional data in an NDA submission under the accelerated approval pathway in mid-2025. We remain dedicated to the ALS community and honored to help critically ill patients and their families.”

Before filing the NDA, Clene intends to begin patient enrolment in a confirmatory Phase 3 RESTORE-ALS trial designed to investigate the effects of CNM-Au8 on improved survival (primary endpoint) and delayed time to ALS clinical worsening events (secondary efficacy endpoint).

Phase 2 clinical trial data as presented to the FDA revealed significant improvement in survival rates, functional status and combined assessment of function and survival. In addition, over 700 patient years of use of CNM-Au8 showed no significant safety concerns or safety trends. No serious adverse events have been identified as related to CNM-Au8 treatment by any investigator to date.

For more information, visit the company’s website at www.Clene.com.

NOTE TO INVESTORS: The latest news and updates relating to CLNN are available in the company’s newsroom at https://ibn.fm/CLNN

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