FDA Provides Guidance on Accelerated Approval Pathway for Clene's ALS Drug Candidate

Clene Inc., a late clinical-stage biopharmaceutical company, has received crucial guidance from the U.S. Food and Drug Administration (FDA) regarding a potential accelerated approval pathway for its lead drug candidate CNM-Au8 in treating amyotrophic lateral sclerosis (ALS). This development marks a significant milestone in the company's efforts to bring a potentially life-changing treatment to patients suffering from this debilitating neurodegenerative disease.

The FDA's Division of Neurology 1 provided Clene with a roadmap outlining how the company might meet the regulatory standard for substantial evidence of effectiveness to support accelerated approval. This guidance came following an in-person Type C meeting on November 1, where Clene presented additional clinical trial data and analyses. The FDA has recommended that Clene leverage additional Neurofilament Light (NfL) data from its three Expanded Access Programs (EAPs) to support earlier findings.

Rob Etherington, CEO and President of Clene, expressed optimism about the FDA's guidance, stating, "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." The company plans to include this additional data in a New Drug Application (NDA) submission under the accelerated approval pathway in mid-2025.

This development is particularly significant for the ALS community, as there is a pressing need for effective treatments for this progressive and fatal neurological disease. ALS, also known as Lou Gehrig's disease, affects nerve cells in the brain and spinal cord, leading to loss of muscle control and eventually paralysis. The potential for an accelerated approval pathway could mean faster access to a novel treatment option for patients who currently have limited therapeutic choices.

CNM-Au8, Clene's investigational first-in-class therapy, aims to improve central nervous system cells' survival and function by targeting mitochondrial function and the NAD pathway while reducing oxidative stress. This approach addresses fundamental aspects of neurodegeneration, potentially offering a new paradigm in treating not only ALS but also other neurodegenerative diseases such as Parkinson's disease and multiple sclerosis.

The FDA's guidance on leveraging additional NfL data is particularly noteworthy. Neurofilament Light chain is a biomarker that can indicate the degree of neuronal damage and has been increasingly recognized as a valuable tool in assessing the progression and treatment effects in various neurological disorders. By incorporating this data from its Expanded Access Programs, Clene may be able to provide more robust evidence of CNM-Au8's efficacy, potentially strengthening its case for accelerated approval.

The implications of this development extend beyond Clene and its stakeholders. If successful, the accelerated approval of CNM-Au8 could pave the way for more innovative approaches in treating neurodegenerative diseases. It could also encourage further research and investment in this critical area of medicine, potentially leading to breakthroughs that could benefit millions of patients worldwide.

As Clene moves forward with its plans to submit an NDA in mid-2025, the company's progress will be closely watched by the medical community, patients, and investors alike. The potential for a new treatment option in ALS offers hope to patients and their families who are grappling with this devastating disease. Moreover, the company's focus on improving mitochondrial health and protecting neuronal function represents a promising approach that could have far-reaching implications in the field of neurodegenerative disease treatment.

While the road to FDA approval is often long and complex, the guidance provided for an accelerated pathway signifies recognition of the urgent need for new treatments in ALS and the potential promise of CNM-Au8. As Clene continues its work, the broader impact of this development serves as a reminder of the critical importance of ongoing research and innovation in addressing some of the most challenging diseases facing modern medicine.