Clene Receives FDA Guidance on Accelerated Approval Pathway for ALS Drug Candidate

Clene Inc. (NASDAQ: CLNN), a late-stage biopharmaceutical company, has made significant progress in its pursuit of an accelerated approval pathway for its lead drug candidate CNM-Au8 in treating amyotrophic lateral sclerosis (ALS). The U.S. Food and Drug Administration's (FDA) Division of Neurology 1 has provided the company with additional written guidance and a roadmap for potentially expediting the drug's approval process.

This guidance follows an in-person Type C meeting held 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. This recommendation could prove crucial in strengthening the company's case for accelerated approval.

The potential for an accelerated approval pathway is significant for both Clene and the ALS community. ALS, a progressive neurodegenerative disease, currently has limited treatment options. If successful, CNM-Au8 could provide a new avenue for improving the quality of life for ALS patients. The drug's mechanism of action, which targets mitochondrial function and the NAD pathway while reducing oxidative stress, represents a novel approach to treating neurodegenerative diseases.

Rob Etherington, CEO and President of Clene, expressed optimism about the FDA's guidance, stating that the company plans to include the additional data in a New Drug Application (NDA) submission under the accelerated approval pathway in mid-2025. This timeline suggests that if approved, CNM-Au8 could potentially reach ALS patients sooner than through traditional approval processes.

The FDA's consideration of an accelerated approval pathway is based on several factors, including the drug's survival and supportive biomarker data, as well as its benign safety profile. The emerging NfL data from the EAPs is expected to play a crucial role in supporting the drug's efficacy claims. NfL is a biomarker that can indicate the degree of neuronal damage, and positive results could provide strong evidence for CNM-Au8's effectiveness in treating ALS.

Clene's progress with CNM-Au8 is not limited to ALS. The company is also investigating the drug's potential in treating other neurodegenerative diseases, including multiple sclerosis and Parkinson's disease. This broad approach to neurodegenerative disorders could position Clene as a significant player in the field of neurological treatments.

The potential accelerated approval of CNM-Au8 could have far-reaching implications for the biopharmaceutical industry and patients alike. It demonstrates the FDA's willingness to consider innovative approaches to drug development and approval, particularly for diseases with high unmet medical needs. For patients and their families, this development offers hope for faster access to potentially life-changing treatments.

As Clene moves forward with its plans to submit an NDA in mid-2025, the company's progress will be closely watched by investors, healthcare professionals, and the ALS community. The outcome of this process could not only impact Clene's future but also set a precedent for other companies developing treatments for neurodegenerative diseases.