- Clene’s CNM-Au8® treatment was demonstrated to improve survival in ALS patients by up to 14.8 months.
- The findings come from a post hoc analysis of the HEALEY ALS Platform Trial, comparing CNM-Au8 to a concurrently enrolled control.
- Strong survival benefit was observed in patients meeting the planned criteria for Clene’s upcoming Phase 3 RESTORE-ALS trial.
- The company is preparing to submit a New Drug Application (“NDA”) to the FDA later this year for potential accelerated approval, and further regulatory discussions and a confirmatory Phase 3 trial are planned for mid-2025.
Clene (NASDAQ: CLNN) and its wholly owned subsidiary, Clene Nanomedicine Inc., a late clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases, including amyotrophic lateral sclerosis (“ALS”) and multiple sclerosis (“MS”), has released new data showing that its lead drug candidate, CNM-Au8, significantly extends survival in patients with ALS. The findings were derived from a cross-regimen post hoc analysis of long-term survival in HEALEY ALS Platform Trial participants (https://ibn.fm/EgWeb).
The study compared participants treated with CNM-Au8 30 mg (Regimen C) against those in Regimen A, which served as a large concurrent control group. Survival data was tracked over 48 months using public records and site reporting. The majority (78%) of patients in both groups received standard ALS treatments such as riluzole and edaravone, or both.
Key findings of the analysis include:
- Patients in the CNM-Au8 30 mg group of Regimen C had a median survival of 951 days, compared to 753 days in the control group — a 6.5-month improvement. When adjusted for key variables such as baseline disease severity and biomarker levels, the restricted mean survival time (“RMST”) analysis showed a statistically significant 4.1-month increase in survival (p=0.045).
- In patients with moderate to severe ALS, the median survival improvement was greater, rising from 589 days (Regimen A) to 951 days (a gain of 11.9 months). In this subgroup, the mortality risk was reduced by 44%, with an RMST improvement of 197 days.
- The largest benefit was seen in patients meeting the criteria for Clene’s upcoming Phase 3 RESTORE-ALS trial. This group experienced a median survival of 1079 days, 14.8 months longer than the control group (628 days), a 49% reduction in mortality risk, and an RMST improvement of 215 days.
These results align with prior studies, including the 24-week HEALEY ALS Platform Trial double-blind period, the RESCUE-ALS trial, and Expanded Access Program data. Commenting on the findings, Merit Cudkowicz, M.D., M.Sc., Principal Investigator and sponsor of the HEALEY ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, and executive director of the Mass General Brigham Neuroscience Institute, said, “The innovative design of the HEALEY ALS Platform Trial has enabled us to extract clear and meaningful survival data that helps make decisions about CNM-Au8 drug development.”
Clene is seeking to bring CNM-Au8 to market as soon as possible. The company is preparing to launch a confirmatory Phase 3 RESTORE-ALS trial in mid-2025, while also considering an accelerated approval pathway for CNM-Au8, following discussions with the FDA in late 2024. As part of this process, Clene will conduct additional biomarker analyses, focusing on neurofilament light chain (“NfL”) data—an important measure of ALS progression.
Rob Etherington, Clene’s President and CEO, expressed optimism about the results, stating that the survival benefit reinforces CNM-Au8’s potential to extend life for people living with ALS while also validating the company’s strategic direction as it prepares for the launch of the confirmatory Phase 3 RESTORE-ALS study in mid-2025. “We look forward to discussing these findings with the FDA as we advance toward potential commercialization,” he said.
For more information, visit the company’s website at www.Clene.com.
NOTE TO INVESTORS: The latest news and updates relating to CLNN are available in the company’s newsroom at https://ibn.fm/CLNN
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