CNM-Au8: An experimental agent for the treatment of amyotrophic lateral sclerosis (ALS)

Expert Opin Investig Drugs. 2023 Aug 29. doi: 10.1080/13543784.2023.2252738. Online ahead of print.ABSTRACTINTRODUCTION: The two established disease-specific therapies for the treatment of amyotrophic lateral sclerosis (ALS) are riluzole and edaravone. Limitations of these medications include minimal progression slowing or survival benefit, and effectiveness in only select populations, particularly for edaravone. AMX0035 and tofersen received US FDA approval in September 2022 and April 2023, respectively. However, phase 3 trials further examining both medications' efficacy are ongoing. CNM-Au8 is an efficient catalyst of energy metabolism, and is therefore a potential disease-modifying treatment in ALS, a neurodegenerative condition in which there is bioenergetics impairment.AREAS COVERED: In this review, we provide an overview of the current ALS treatment market, followed by description of the pharmacodynamics and pharmacokinetics of CNM-Au8. The main preclinical and available early clinical evidence of CNM-Au8 is then described, as well as its potential as an ALS treatment.EXPERT OPINION: Oral treatment with CNM-Au8 failed to meet primary clinical and electrodiagnostic endpoints in phase 2/3 clinical trials. Despite this failure, a number of exploratory endpoints included in phase 2/3 trials suggest CNM-Au8 has the potential to significantly slow clinical worsening, improve quality of life, and prolong survival in ALS. Further study of CNM-Au8 in a phase 3 clinical trial is...
Source: Expert Opinion on Investigational Drugs - Category: Drugs & Pharmacology Authors: Source Type: research