Meaningful improvements confirmed in slow-progressor subgroup; biomarker reductions support precision-medicine approach and potential FDA accelerated pathway
SEOUL, South Korea, Sept. 17, 2025 /PRNewswire/ -- CorestemChemon Inc. (KOSDAQ: 166480) announced today that it has presented key findings from the Phase 3 ALSummit trial of Neuronata-R® (lenzumestrocel), an autologous bone marrow-derived mesenchymal stem cell (MSC) therapy for amyotrophic lateral sclerosis (ALS), at the PACTALS 2025 congress in Melbourne, Australia.
The first session, chaired by Professor Seung-Hyun Kim of Hanyang University Hospital - a global leader in stem cell-based ALS research - brought together a panel of seven distinguished ALS experts from six countries. As part of the panel, Dr. Ryung-A Lee (Head of R&D Innovation, CorestemChemon) introduced the company and shared a brief overview of the Phase 3 trial results.
In the second session, the full Phase 3 clinical trial (ALSUMMIT trial) results were formally presented by Professor Ki-Uk Oh (Department of Neurology, Advanced Regenerative Medicine Center, Hanyang University Hospital, and principal investigator of the ALSUMMIT trial).
Subgroup Analysis Demonstrates Statistically Significant Benefit
While the overall trial did not meet its primary and secondary endpoints, Neuronata-R® showed meaningful efficacy in patients with slower disease progression.
At 12 months, participants in this subgroup receiving Neuronata-R® achieved:
- ALSFRS-R (functional rating scale): 31.2 vs. 26.4 in placebo (p=0.001)
- CAFS (Combined Assessment of Function and Survival): 20.95 and 24.78 vs. 17.92 in placebo (p=0.024; p=0.041)
- SVC (slow vital capacity, respiratory function): 62.2% vs. 50.6% in placebo (p=0.017, Study Group 2)
These findings confirm Neuronata-R®'s potential to preserve function and respiratory capacity in ALS - outcomes directly linked to quality of life and survival.
Biomarker Data Underscore Neuroprotective Effect
Biomarker analysis revealed sustained reductions in neurofilament light chain (NfL) and MCP-1, indicating a strong link between neuroprotection and clinical outcomes.
This strengthens the case for biomarker-guided patient stratification and a precision-medicine strategy in ALS drug development, reflecting the evolving global regulatory landscape.
Regulatory Pathway Toward Accelerated Approval
CorestemChemon stated that the PACTALS 2025 results mark an important step for regulatory discussions. The company plans to request a Type-C meeting with the U.S. Food and Drug Administration (FDA) in Q4 2025 to discuss the dataset and biomarker-driven subgroup efficacy.
Based on this discussion, CorestemChemon plans to submit a Biologics License Application (BLA) within 2026, aiming to pursue an accelerated approval pathway aligned with precedents in ALS, including FDA's decision on Tofersen.
Innovative Stem Cell Therapy
Neuronata-R® uniquely addresses the complex mechanisms of ALS by leveraging MSCs derived from the patient's own bone marrow. These cells exert anti-inflammatory and immunomodulatory effects, protect motor neurons, and, through paracrine signaling, secrete trophic factors, cytokines, and extracellular vesicles that modulate the microenvironment and reduce neuroinflammation. By targeting these underlying pathological processes, Neuronata-R® is designed to interrupt the neurodegenerative cascade.
About Neuronata-R®
Neuronata-R® (Lenzumestrocel), developed and commercialized by CorestemChemon Inc. (KOSDAQ: 166480), is an autologous MSC therapy for ALS patients. The company began ALS research in 2002 and obtained MFDS approval for Neuronata-R® in 2014. To date, it has been administered to more than 400 commercial patients and 190 clinical trial participants with no treatment-related serious adverse events reported.
Neuronata-R® holds Orphan Drug Designation from both the U.S. FDA (2018) and the EMA (2019). CorestemChemon completed a Phase 2 trial (NCT01363401) in 2014 and its Phase 3 trial (NCT04745299) in 2024; the final CSR has been submitted to the MFDS.
SOURCE CorestemChemon Inc.
