CorestemChemon Inc. (KOSDAQ: 166480) presented results from the Phase 3 ALSummit trial of Neuronata-R® (lenzumestrocel), an autologous bone marrow-derived MSC therapy for amyotrophic lateral sclerosis (ALS), at PACTALS 2025 in Melbourne, Australia.
- The first session, chaired by Professor Seung-Hyun Kim of Hanyang University Hospital, included a panel of seven global ALS experts.
- Dr. Ryung-A Lee, Head of R&D Innovation at CorestemChemon, introduced the company and previewed the trial outcomes.
- The second session featured a detailed presentation of the full Phase 3 data by Professor Ki-Uk Oh, principal investigator at Hanyang University Hospital.
Subgroup Benefits Despite Missed Endpoints
The ALSummit trial did not meet its primary and secondary endpoints in the overall population. Yet subgroup analysis revealed significant efficacy for patients with slower disease progression.
At 12 months, patients receiving Neuronata-R® achieved:
- ALSFRS-R: 31.2 vs. 26.4 in placebo (p=0.001)
- CAFS: 20.95 and 24.78 vs. 17.92 in placebo (p=0.024; p=0.041)
- SVC (respiratory function): 62.2% vs. 50.6% in placebo (p=0.017, Study Group 2)
These outcomes point to preserved function and respiratory capacity—two factors directly linked to quality of life and survival in ALS.
Biomarker Analysis Supports Neuroprotection
- Sustained reductions in neurofilament light chain (NfL) and MCP-1 were observed.
- Biomarker results confirm neuroprotective activity and connect biological effects to clinical benefit.
- Findings support a precision-medicine strategy with biomarker-guided patient stratification.
Regulatory Strategy
CorestemChemon plans to:
- Request a Type-C FDA meeting in Q4 2025.
- Discuss biomarker-driven subgroup efficacy and overall dataset.
- Submit a Biologics License Application (BLA) in 2026.
- Pursue accelerated approval, following precedents like FDA’s Tofersen decision.
How Neuronata-R® Works
Neuronata-R® is derived from the patient’s own bone marrow MSCs.
These cells:
- Exert anti-inflammatory and immunomodulatory effects.
- Protect motor neurons.
- Release trophic factors, cytokines, and extracellular vesicles.
- Alter the microenvironment to reduce neuroinflammation.
This multimodal action is designed to interrupt the neurodegenerative cascade in ALS.
About Neuronata-R® and CorestemChemon
- Neuronata-R® (lenzumestrocel) was first approved by the MFDS in 2014.
- Over 400 commercial patients and 190 clinical trial participants have received treatment with no therapy-related serious adverse events.
- Holds Orphan Drug Designation from both U.S. FDA (2018) and EMA (2019).
- Completed Phase 2 (NCT01363401) in 2014 and Phase 3 (NCT04745299) in 2024.
- The final CSR has been submitted to the MFDS.
CorestemChemon has been conducting ALS research since 2002 and continues to expand its regenerative medicine portfolio.
