Clene Announces Statistically Significant ALS Biomarker Results Supporting Accelerated Approval Pathway for CNM-Au8®
- FDA recommended biomarker analyses show statistically significant reductions in NfL and GFAP in participants treated with CNM-Au8
- Biomarker improvements are strongly associated with longer survival, reinforcing CNM-Au8’s potential disease modifying activity
- Company has requested a Type C meeting in the first quarter of 2026 to present the newly completed analyses supporting a planned NDA submission under the accelerated approval pathway
- Clene is hosting an investor webcast today at 8:30 am ET
SALT LAKE CITY, Dec. 03, 2025 (GLOBE NEWSWIRE) -- Clene Inc. (Nasdaq: CLNN) (along with its subsidiaries, “Clene” or the “Company”) and its wholly owned subsidiary Clene Nanomedicine, Inc., a late clinical-stage biopharmaceutical company focused on revolutionizing the treatment of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS), today announced completion of the FDA-recommended biomarker analyses for CNM-Au8® in people living with ALS. The results demonstrate statistically significant reductions in both neurofilament light (NfL) and glial fibrillary acidic protein (GFAP) and provide compelling evidence linking biomarker decline to improved survival.
“We followed the FDA’s roadmap — and the data delivered,” said Rob Etherington, President and CEO of Clene. “Statistically significant NfL reductions in more advanced real-world ALS patients; a second independent disease-specific biomarker (GFAP) that moves in lock-step with NfL and is itself statistically significant; and clear evidence in placebo switchers (placebo-to-CNM-Au8) showing that CNM-Au8-treated participant’s biomarkers follow nearly identical trajectories seen in the original active arm during the double-blind treatment phase of the HEALEY ALS Platform Trial. This is a remarkably consistent dataset that makes a strong case for accelerated approval. We look forward to presenting these analyses to the Division in the requested Type C meeting in the first quarter of 2026 and working with the FDA toward an NDA submission for accelerated approval.”
The analyses follow FDA recommendations to support the persuasiveness of the original NfL findings observed in the HEALEY ALS Platform Trial by extending the analyses to the NIH-sponsored EAP (NIH-EAP) for CNM-Au8 in ALS. These biomarker findings build on prior constructive FDA interactions in support of a planned NDA submission under the accelerated approval pathway for the treatment of ALS.
New Biomarker Analyses
In late 2024, the FDA recommended three specific analyses to strengthen the persuasiveness of CNM-Au8’s effect on NfL and its relationship to clinical benefit (i.e., effects on survival): (1) NfL change in the NIH-EAP, (2) evaluation of additional disease-relevant biomarkers, and (3) evaluation of NfL trajectory in HEALEY placebo participants who later transitioned to CNM-Au8 in the open-label extension (OLE):
- Statistically significant decrease in NfL levels compared to matched ALS controls across the full analysis set (all evaluable matched participants) in the NIH-EAP. The Week 36 AUC (area under curve) difference (SEM) of NfL (Ln(pg/mL)*Week) was: –0.0899 (0.0430), p = 0.0373, equivalent to a geometric mean ratio (GMR) difference of 0.914, 95% CI: 0.840 – 0.995. The effect size was similar to the NfL decline observed in the original double-blind phase of the HEALEY ALS Platform Trial: HEALEY W24 AUC GMR of 0.901, 95% CI: 0.845 – 0.959, p=0.0013 compared to the NIH-EAP W24 AUC GMR of 0.911, 95% CI: 0.836 – 0.993, p=0.0339. Multiple pre-specified supportive analyses in the NIH-EAP across the full analysis set at Week 24 and Week 48 confirmed the robustness of the findings (p
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