Clene Shares 3.5-Year Survival Data and New NfL Responder Results from HEALEY ALS Trial at 2024 ENCALS Meeting

Clene Inc., a biopharmaceutical company focused on neurodegenerative diseases, has unveiled new long-term findings regarding its treatment CNM-Au8 at the European Network for the Cure of ALS (ENCALS) meeting in Stockholm, Sweden. The data stems from the HEALEY ALS Platform Trial and its open label extension (OLE), a study aimed at evaluating the efficacy and safety of CNM-Au8 for amyotrophic lateral sclerosis (ALS) patients.

The presentation, entitled “Long-Term CNM-Au8 Treatment Reduces Neurofilament Light Levels and Improves Survival: Results from the HEALEY ALS Platform Trial,” showcased survival data up to 3.5 years and neurofilament light (NfL) biomarker outcomes over 76 weeks. Participants included those initially randomized to CNM-Au8 30 mg and placebo participants who later transitioned to CNM-Au8 in the OLE phase.

Key findings demonstrated that CNM-Au8 significantly improved survival rates compared to matched controls from the PRO-ACT database. Specifically, the treatment group exhibited a 60% reduced risk of death over a 3.5-year period, with a hazard ratio of 0.431 and a p-value of 0.0002. This suggests a notable survival benefit for CNM-Au8 treated individuals.

Additionally, the study focused on a subset of participants classified as NfL responders. These individuals consistently showed a reduction in NfL levels post-baseline, a marker associated with neuronal degradation. Within this subset, an average 28% reduction in NfL levels was observed, indicating decreased axonal loss. The geometric mean ratio (GMR) at week 76 compared to baseline was 0.72, with a p-value of less than 0.0001, underscoring the statistical significance of these findings.

The broader analysis also reported that long-term CNM-Au8 treatment resulted in continued significant declines in plasma NfL levels, even for those who initially received a placebo for 24 weeks before switching to the active treatment. At week 76, the GMR compared to the placebo group was 0.841, with a confidence interval between 0.73 and 0.98, and a p-value of 0.023.

Safety data revealed that CNM-Au8 was well-tolerated, with no significant safety signals identified throughout the study duration. Benjamin Greenberg, M.D., Head of Medical at Clene, highlighted the robustness of the clinical evidence supporting CNM-Au8’s potential as a treatment for ALS, citing its impact on both plasma neurofilament reduction and improved long-term survival.

Clene Inc. continues to emphasize its commitment to developing therapies that enhance mitochondrial health and neuronal function. CNM-Au8, an investigational therapy, targets mitochondrial function and the NAD pathway, aiming to reduce oxidative stress and support central nervous system cell survival and function.

Based in Salt Lake City, with R&D and manufacturing operations in Maryland, Clene is dedicated to advancing treatments for neurodegenerative diseases. The company’s strategic focus includes conditions such as ALS, Parkinson’s disease, and multiple sclerosis.

This new data presentation reinforces Clene’s position in the biopharmaceutical landscape, showcasing the promising potential of CNM-Au8 to make a significant impact on ALS treatment. The company continues to advance its clinical programs, driven by a mission to improve patient outcomes in neurodegenerative diseases.