Clene Provides Update on Its NIH-Funded Expanded Access Program for CNM-Au8® in ALS (ACT-EAP)

Core Points - Clene Inc. is expanding its National Institutes of Health (NIH)-funded Accelerating Access to Critical Therapies Expanded Access Program (ACT-EAP) for its investigational drug CNM-Au8 in ALS, increasing enrollment by 80% to a maximum of 180 participants [1][2][3] - The ACT-EAP aims to collect 'real-world' drug efficacy data, monitoring potential effects on survival and disease progression, alongside safety data [1][2] - The first patient visit for the ACT-EAP is scheduled for early June 2024 [1][3] Company Updates - Clene has received a four-year grant from the National Institute of Neurological Disorders and Stroke (NINDS) to support the EAP for CNM-Au8 in ALS [2] - The company has implemented significant efficiencies in manufacturing, operations, and personnel to accommodate the expanded program without increasing the budget [2] - Clene's ACT-EAP is designed to contribute to the understanding of ALS disease progression and response to therapy, adding to the clinical safety database for CNM-Au8 [2][3] Drug Efficacy and Safety - CNM-Au8 treatment has shown a lowered risk of death and delayed clinical worsening in independent Phase 2 clinical studies [3] - The EAP will provide broader data on ALS patients compared to traditional clinical trials, enhancing the understanding of the drug's safety and efficacy [3] - The study will also collect biomarker data to analyze alongside the efficacy data [2] Collaboration and Innovation - Clene is collaborating with Columbia University and Synapticure to enhance the reach of the EAP through a telehealth platform, making it accessible to more ALS patients across the US [3] - The ACT-EAP is not a standard program; it is designed to yield important insights into the real-world impact of CNM-Au8 treatment on ALS [3] Background on Clene - Clene Inc. is a late clinical-stage biopharmaceutical company focused on treating neurodegenerative diseases, including ALS, Parkinson's disease, and multiple sclerosis [5] - CNM-Au8 is a first-in-class investigational therapy aimed at improving mitochondrial health and reducing oxidative stress in central nervous system cells [5]