Clene's push for ALS drug approval sparks debate over expedited processes

In a significant development for ALS therapy, Clene Inc., based in Salt Lake City, Utah, announced on December 11, 2024, that it has received guidance from the U.S. Food and Drug Administration (FDA). The FDA advised Clene to utilize data gathered from ongoing expanded access programs to bolster its case for an accelerated approval application for its drug CNM-Au8. This guidance marks a crucial milestone for Clene as it seeks to expedite the availability of CNM-Au8, which is aimed at treating amyotrophic lateral sclerosis (ALS), a severe neurodegenerative condition that currently lacks effective treatments. Clene reported that the FDA specifically recommended the integration of neurofilament light chain biomarker analyses to provide evidence of the drug's efficacy, which could validate its survival benefits for ALS patients. Following this FDA recommendation, Clene is planning to compile and submit additional data by mid-2025 and will initiate the Phase 3 RESTORE-ALS clinical trial. The outcome of these initiatives could significantly impact Clene's roadmap and the potential delivery of CNM-Au8 as an accessible treatment option for ALS patients. Previous clinical findings shared during meetings with the FDA emphasized the drug's substantial survival benefits while maintaining a benign safety profile, strengthening the belief in CNM-Au8’s capability as a transformative treatment in the realm of ALS therapies. The initiative to accelerate the approval process comes amid rising urgency to address the unmet medical needs of ALS patients and their families who are desperately seeking effective treatment options. With this FDA guidance, Clene is positioned to potentially reduce the time it takes for ALS patients to gain access to new therapies that could improve their quality of life. Clene remains resolute in addressing these needs and is committed to furthering research that could lead to groundbreaking advancements in the treatment of ALS. The company’s ongoing operational activities span research and development as well as manufacturing, which are chiefly based in Maryland, enhancing its capabilities in pushing forward with CNM-Au8’s development and potential market introduction.