CorestemChemon Inc.  announced  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. Dr. Ryung-A Lee Head of R&D Innovation at CorestemChemon introduced the company and shared an overview of the Phase 3 trial results. The full Phase 3 clinical trial (ALSummit) data were then presented by Professor Ki-Uk Oh principal investigator and neurologist at the Advanced Regenerative Medicine Center Hanyang University Hospital.

Subgroup Analysis Demonstrates Statistically Significant Benefit
While the overall trial did not meet its primary and secondary endpoints Neuronata-R® demonstrated 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 patient quality of life and survival.

Biomarker Data Underscore Neuroprotective Effect
Biomarker analysis revealed sustained reductions in neurofilament light chain (NfL) and MCP-1 suggesting a strong link between neuroprotection and clinical outcomes. This supports biomarker-guided patient stratification and a precision-medicine approach to ALS therapy aligned with evolving regulatory expectations.

Regulatory Pathway Toward Accelerated Approval
CorestemChemon stated that the PACTALS 2025 results mark an important milestone 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 review the dataset and subgroup efficacy. Based on this dialogue CorestemChemon expects to submit a Biologics License Application (BLA) in 2026 targeting an accelerated approval pathway consistent with precedents such as FDA’s decision on Tofersen.

Innovative Stem Cell Therapy
Neuronata-R® is designed to address 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 signalling secrete trophic factors cytokines and extracellular vesicles that modulate the microenvironment and reduce neuroinflammation. By targeting these pathological processes Neuronata-R® aims to slow disease progression and improve outcomes for patients with ALS.