#South Korea #Seoul #ALS #Neuronata-R #CorestemChemon

Neuronata-R® Phase 3 Results: Promising Advances in ALS Treatment

On September 17, 2025, CorestemChemon Inc. made a landmark announcement regarding the findings from the Phase 3 ALSummit trial of Neuronata-R® (lenzumestrocel), an innovative autologous bone marrow-derived mesenchymal stem cell (MSC) therapy specifically targeting amyotrophic lateral sclerosis (ALS). These findings were presented at the PACTALS 2025 congress in Melbourne, Australia, drawing significant attention from the medical community and ALS specialists worldwide.

Key Findings from the Phase 3 Trial

During the initial session led by Professor Seung-Hyun Kim from Hanyang University Hospital, a panel of seven esteemed ALS experts gathered to discuss the trial's impactful results. Dr. Ryung-A Lee, CorestemChemon's Head of R&D Innovation, provided an insightful overview of the trial's outcomes.

Although the overall results of the trial did not reach primary and secondary endpoints as initially hoped, Neuronata-R® exhibited promising efficacy among patients experiencing a slower progression of ALS. Notably, the following improvements were observed at the 12-month mark for participants in the slow-progressor subgroup:

• - ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised): 31.2 in the Neuronata-R® group compared to 26.4 in the placebo group, with a significant p-value of 0.001.
• - CAFS (Combined Assessment of Function and Survival): Scores of 20.95 in the treatment group versus 17.92 in placebo, demonstrating statistical relevance (p=0.024; p=0.041).
• - SVC (Slow Vital Capacity): An impressive 62.2% in the Neuronata-R® group, against 50.6% in placebo, with p-value of 0.017, illustrating enhanced respiratory function.

These outcomes are particularly critical, as they correlate directly with improved quality of life and increased survival rates among ALS patients.

Biomarker Insights

A deeper examination into biomarker results shed light on neuroprotective effects associated with Neuronata-R®. Significant reductions in neurofilament light chain (NfL) and MCP-1 were observed, establishing a notable relationship between these markers and clinical outcomes. These findings underscore the importance of biomarker-guided patient stratification in the ongoing development of ALS therapies, aligning with the shifting global regulatory perspectives.

Pathway for Accelerated FDA Approval

CorestemChemon's results from PACTALS 2025 signify a pivotal moment for future regulatory discussions related to Neuronata-R®. The company intends to request a Type-C meeting with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2025. This meeting aims to discuss the implications of the trial's data and insights into the efficacy observed in specific patient subgroups. Following these discussions, CorestemChemon plans to submit a Biologics License Application (BLA) within 2026, pursuing an expedited approval based on precedents established in the field of ALS treatments, such as FDA's recent approval of Tofersen.

Innovative Approach to ALS Treatment

Neuronata-R® represents a groundbreaking attempt to confront the multifaceted challenges posed by ALS. By utilizing MSCs sourced from the patient’s own bone marrow, this therapy capitalizes on the unique anti-inflammatory and immunomodulatory properties of these cells. Moreover, they are known to protect motor neurons and secrete various trophic factors that help modulate the surrounding microenvironment, thereby reducing neuroinflammation.

CorestemChemon started its research on ALS back in 2002 and successfully gained marketing approval from the MFDS for Neuronata-R® in 2014. Over 400 patients have received this treatment without serious adverse effects. Neuronata-R® has also earned Orphan Drug Designation from both the U.S. FDA and EMA, underlining its potential in treating rare diseases.

In conclusion, the findings from the Phase 3 ALSummit trial signify a substantial advancement in the treatment of ALS, showcasing the potential for Neuronata-R® to not only enhance patient quality of life but also pave the way for innovative treatment strategies in the landscape of neurodegenerative diseases.