CorestemChemon Inc (KOSDAQ:166480), a South Korea-based biotech company, announced on Wednesday 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 authority in stem cell-based ALS research -- brought together a panel of seven 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).

Although the overall trial did not meet its primary and secondary endpoints, the company says that Neuronata-R showed meaningful efficacy in patients with slower disease progression. The findings confirmed Neuronata-R's potential to preserve function and respiratory capacity in ALS -- outcomes directly linked to quality of life and survival.

Additionally, 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, CorestemChemon said.

The company plans to request a Type-C meeting with the US 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) in 2026, aiming to pursue an accelerated approval pathway aligned with precedents in ALS, including the FDA's decision on Tofersen.