- The FDA offered supportive feedback on Clene’s proposed statistical analysis plan during a recent Type C meeting.
- The neurofilament biomarker analysis results could support an accelerated approval submission for CNM-Au8 later in 2025.
- Clene has two additional FDA meetings scheduled in Q3 2025 to discuss ALS survival data and its MS program.
- Nearly 200 ALS patients are participating in the NIH-sponsored Expanded Access Program for CNM-Au8.
- The analysis aims to validate the neuroprotective effects shown in previous HEALEY Platform Trial results.
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”), is set to move ahead with neurofilament biomarker analyses for its lead candidate CNM-Au8(R) ALS early in the fourth quarter of 2025. The announcement follows a recent productive Type C meeting with the U.S. Food and Drug Administration (“FDA”) (https://ibn.fm/F5AHk).
The planned analyses will examine changes in neurofilament light chain (“NfL”), a recognized biomarker for neurodegeneration, among nearly 200 ALS patients treated through the National Institutes of Health–sponsored Expanded Access Program (“EAP”) for CNM-Au8. The company’s statistical plan will compare these patients to matched ALS controls, with a primary analysis at nine months and a supportive analysis at six months of treatment.
In the Expanded Access Program, CNM-Au8 is being administered on a compassionate use basis to people living with ALS. The company will analyze whether reductions in NfL from this cohort mirror or exceed the neuroprotective trends reported in the double-blind HEALEY trial.
Benjamin Greenberg, MD, Head of Medical at Clene, described the FDA’s feedback as “constructive” and said the agency’s acceptance of Clene’s revised statistical plan is expected this summer. Clene has already resubmitted its updated analysis plan to the FDA to incorporate requested changes, aiming to finalize the evaluation framework ahead of its fourth-quarter biomarker review.
“We are encouraged by the FDA’s collaborative approach and their constructive feedback on our NfL biomarker analysis plan from the ongoing NIH-sponsored EAP program,” said Greenberg. “With two additional FDA meetings scheduled to discuss long-term ALS survival results and the End-of-Phase 2 MS results, we are advancing our ALS and MS programs to deliver an innovative therapy for people living with neurodegenerative diseases.”
A positive outcome from this NfL assessment is likely to strengthen Clene’s case for a new drug application (“NDA”) submission under the accelerated approval pathway before the end of 2025. The company is seeking to build on data from the HEALEY ALS Platform Trial, where CNM-Au8 showed encouraging neuroprotective signals over a six-month period.
Alongside the biomarker study, Clene confirmed two additional FDA meetings are scheduled for the third quarter of 2025. The first will review survival data from patients receiving 30 mg CNM-Au8 compared with controls from another arm of the HEALEY Platform Trial. The second meeting will focus on the company’s multiple sclerosis program, discussing Phase 2 data from the VISIONARY-MS trial and Clene’s plans for a Phase 3 study targeting cognitive outcomes.
The FDA’s engagement on multiple fronts highlights a potentially promising path forward for CNM-Au8, an oral suspension of gold nanocrystals designed to improve cellular energy production and utilization, which is critical for maintaining neuronal health. Clene hopes these data will support broader approval opportunities for ALS, where treatment options remain limited.
In addition to its ALS program, Clene is keeping momentum in its multiple sclerosis research, using similar bioenergetic principles focused on mitochondria within the central nervous system to develop therapies that could improve cognitive outcomes for people living with MS. The company expects its end-of-Phase 2 MS discussion with the FDA in the third quarter will set the stage for a larger Phase 3 trial.
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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