Clene Inc. (Nasdaq: CLNN) is set to meet in person with senior leadership from the U.S. Food and Drug Administration (FDA) before the end of November 2024 to discuss the development of CNM-Au8 for amyotrophic lateral sclerosis (ALS). The meeting follows initial communication from the FDA that Clene's briefing package did not support an NDA submission under the accelerated approval pathway. However, after further dialogue, the FDA agreed to reassess the submission in person. This decision marks a potentially significant step for CNM-Au8, an investigational therapy aimed at improving mitochondrial health and protecting neuronal function in neurodegenerative diseases. The outcome of this meeting could significantly influence the regulatory pathway and timeline for CNM-Au8's potential approval.
Focus on Biomarkers and Clinical Data
During the meeting, Clene plans to present comprehensive data on ALS biomarkers, clinical endpoints, and survival data related to CNM-Au8. This presentation will involve key FDA officials, including the Director of the Office on New Drugs, the Director of the Office of Neuroscience, and the Division of Neurology 1 (DN1) review team, as well as recognized key opinion leaders in ALS, biostatistics, and biomarkers. Clene aims to address questions and provide expert views on the essential elements for understanding CNM-Au8's potential in treating ALS.
Safety Profile of CNM-Au8
Clene has amassed substantial safety data for CNM-Au8, with over 700 patient-years of data indicating no significant safety concerns or trends. According to the company, clinical trial investigators have not identified any serious adverse events (SAEs) related to CNM-Au8 treatment to date. This extensive safety profile could be a crucial factor in the FDA's evaluation of the therapy's risk-benefit profile.
CNM-Au8: Mechanism of Action and Therapeutic Potential
CNM-Au8 is an oral suspension of gold nanocrystals designed to restore neuronal health and function by enhancing energy production and utilization within cells. The catalytically active nanocrystals are intended to drive critical cellular energy-producing reactions, thereby promoting neuroprotection and remyelination. By increasing neuronal and glial resilience to disease-relevant stressors, CNM-Au8 aims to address the underlying mechanisms of neurodegeneration in ALS and other neurological conditions.
Regulatory Context and Future Prospects
The FDA's initial stance on Clene's NDA submission suggests that the agency requires more robust evidence to support accelerated approval. The upcoming in-person meeting provides Clene with an opportunity to address the FDA's concerns and present a compelling case for CNM-Au8's efficacy and safety. The focus on biomarkers is particularly important, as establishing reliable biomarkers for ALS progression could revolutionize drug development and clinical management of the disease.
This meeting represents a critical juncture for Clene. A positive outcome could significantly boost Clene's prospects, potentially leading to faster approval and market entry. Conversely, if the FDA remains unconvinced, it could lead to delays and additional costly trials. Investors should closely monitor the outcome of this meeting, as it will likely have a substantial impact on Clene's valuation and future prospects.
