New Data from the NIH-Funded CNM-Au8® Expanded Access Program Provides Support for a Survival Benefit in ALS

Data Highlights ACT for ALS as a Catalyst for Continued Progress in ALS

WASHINGTON, Dec. 4, 2025 /PRNewswire/ -- I AM ALS celebrates promising new analyses released by Clene Nanomedicine demonstrating significant improvements in survival associated with their treatment, CNM-Au8^®, as measured through the biomarker neurofilament light chain (NfL) in cohorts from the ACT for ALS Expanded Access Program (EAP) and clinical trial participants. These results offer renewed optimism for a community urgently seeking effective treatments and meaningful progress toward slowing this devastating disease.

Clene's newly announced findings show statistically significant improvements in overall survival reflected in two biomarkers: reductions in NfL—a critical marker of neuronal injury—in real-world, more advanced ALS patients treated with CNM-Au8^®, and concordant reductions in a second disease-relevant biomarker, glial fibrillary acidic protein (GFAP). GFAP's movement in "lock-step" with NfL strengthens the biological case for treatment benefit.

"These results collectively represent one of the most cohesive biomarker stories we've seen in ALS to date," said Rob Etherington, Clene President and CEO. "In a disease where progress has long been slowed by the lack of reliable biomarkers, this new finding is a critical milestone—providing a powerful tool to speed therapy development and strengthen the path to approval."

"This is a remarkably consistent dataset that makes a strong case for accelerated approval," Etherington continued. "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."

For the estimated 30,000 people in the United States living with ALS, and the countless caregivers and loved ones impacted alongside them, these findings represent hope grounded in rigorous scientific evidence. These findings also highlight the power and importance of ACT for ALS—not only in expanding access to promising therapies, but also in accelerating scientific learning and speeding progress toward approval for treatments showing meaningful signs of efficacy.

"This is a powerful testament to how ACT for ALS is accelerating progress against this disease," said Andrea Goodman, CEO of I AM ALS. "First, with more confirmatory data now in hand, we are grateful that hundreds of people with ALS have already gained access to CNM-Au8; access that may have meant being alive today rather than dying while waiting for traditional trials and slow, procedural FDA processes."

"Second, unlike traditional trials, the ACT for ALS EAP enables participation and data collection from an understudied population—patients whose ALS is more advanced," Goodman says. "And importantly, by leveraging data from this early, real-world access, the EAP is advancing global understanding of promising treatments and speeding the path toward approval. Faster access, greater hope, and real treatment progress are exactly why we're fighting for ACT for ALS reauthorization before it expires in September 2026."

"This is what people living with ALS have been fighting for: potential treatments in development with thorough and meaningful data," said Dr. Jinsy Andrews, director of the NYU Langone ALS Center and principal investigator of the NIH-funded EAP. "ACT for ALS EAPs are providing data that deepens our biomarker knowledge not only in a traditional clinical trial population, but also in later-stage ALS in ways we've never seen before. This progress could mark a turning point for ALS basic science research and therapeutic development in the future, and reflects the persistence of patients, advocates, researchers, and companies determined to change the trajectory of this disease."

As a next step, Clene has requested a Type C meeting with the FDA and anticipates the meeting will occur during the first quarter of 2026. The Company plans to submit an NDA under the accelerated approval pathway in the same quarter, with the planned Phase 3 RESTORE-ALS trial serving as the post-approval confirmatory study. I AM ALS urges the FDA to act swiftly, recognizing both the strength of the biomarker evidence and the urgent unmet needs of people living with ALS. As I AM ALS simultaneously and urgently seeks fully funded reauthorization of ACT for ALS, these data demonstrate the critical importance of sustaining this landmark law.

Learn more about ACT for ALS here.

About I AM ALS
I AM ALS is a nonprofit organization leading what STAT News called the most successful patient advocacy campaign this century. We built a community movement to harness collective power and find treatments and a cure for ALS faster, while also creating lasting, systemic change. Our focus is on three areas:

• Advocating for federal policy change to drive research, support, and treatments for ALS.
• Improving quality of life by providing volunteer and support opportunities to advocates and people living with ALS.
• Mobilizing and empowering advocates to raise awareness about ALS and other neurodegenerative diseases, and increase visibility of the ALS experience.

Learn more and join us at www.iamals.org.

SOURCE I AM ALS