Could a new ALS treatment change lives?
Clene has received encouraging feedback from the U.S. Food and Drug Administration (FDA) regarding its potential new treatment for amyotrophic lateral sclerosis (ALS). The FDA indicated that the data from Clene’s research could support an application for accelerated approval of their drug, CNM-Au8, based on a specific biomarker known as neurofilament light (NfL). This biomarker may serve as a reliable indicator of the drug’s effectiveness in treating ALS, a progressive neurodegenerative disease that affects muscle control and movement.
This news is significant for anyone concerned about ALS or looking for advancements in treatments for neurodegenerative diseases. If approved, CNM-Au8 could potentially help slow the progression of ALS by improving mitochondrial function, which is crucial for energy production in cells. Clene plans to submit its New Drug Application in 2026, supported by data from various clinical trials, including the Phase 2 HEALEY ALS Platform Trial. These trials aim to establish a clear link between NfL reductions and real clinical benefits for patients.
While this development is promising, it’s important to note that the research is still in the early stages. The FDA has requested additional evidence to confirm that changes in the NfL biomarker will translate into tangible health benefits for people with ALS. Clene also plans to begin a Phase 3 study in early 2027 to further validate the drug’s effectiveness. For now, those interested in ALS treatments should stay informed about ongoing research and clinical trials that could lead to new options in the future.
Source: longevity.technology