Meaningful efficacy observed in slow-progressor subgroup; NfL biomarker improvements support potential for accelerated regulatory pathway
SEOUL, South Korea, May 29, 2025 /PRNewswire/ -- Neuronata-R®, an autologous bone marrow-derived mesenchymal stem cell (MSC) therapy for amyotrophic lateral sclerosis (ALS), has demonstrated meaningful efficacy signals in a subgroup of participants with slow disease progression in its recently completed Phase 3 trial. The therapy also showed consistent reductions in neurofilament light chain (NfL) levels — a biomarker that served as the basis for FDA accelerated approval in ALS, including the recent decision on Tofersen — supporting its potential to pursue a similar regulatory pathway.
Developed by South Korean biotech company CorestemChemon (KOSDAQ: 166480), Neuronata-R® utilizes MSCs derived from a participant's own bone marrow to modulate inflammation, protect motor neurons, and alter the neurodegenerative microenvironment through paracrine signaling. ALS, a rare and fatal disease with no cure, remains one of the most urgent areas of unmet medical need. Neuronata-R® aims to address the disease's complex pathology by leveraging the therapeutic potential of autologous MSCs.
In December 2024, CorestemChemon announced topline results from the ALSummit trial (NCT04745299), noting that the study did not meet its primary endpoint — a combined assessment of function and survival known as CAFS — in the overall patient population. However, a post hoc analysis conducted after the completion of the trial and full data collection revealed significant clinical improvements in a subgroup of patients with slower disease progression. To discuss these findings, the company plans to meet with the U.S. Food and Drug Administration (FDA) later this year to discuss these findings, with the aim of submitting a biologics license application by the end of 2025 and potentially securing accelerated approval by mid-2026.
Subgroup Analysis Supports Targeted Efficacy
The final Clinical Study Report (CSR) confirmed that Neuronata-R® demonstrated statistically significant improvements in key efficacy endpoints among participants with slow disease progression. Recognizing the potential efficacy of Neuronata-R® in early-stage ALS, CorestemChemon stratified participants into slow and fast progressors.
Among slow progressors, Neuronata-R® showed statistically significant improvements across multiple measures — including the primary composite endpoint, CAFS (Combined Assessment of Function and Survival), functional outcomes assessed by ALSFRS-R scores, and respiratory function measured by slow vital capacity (SVC).
Notably, the five-dose treatment arm (Group 2) demonstrated statistically significant improvement in ALSFRS-R scores beginning at Month 9 post-treatment — one month earlier than the two-dose arm (Group 1), which reached significance at Month 10.
This final CSR also included full analyses of CAFS and SVC, which had not been previously disclosed. While CAFS served as the trial's primary efficacy endpoint, SVC is recognized as a clinically meaningful measure of respiratory decline in ALS.
At Month 6, participants in Group 1 receiving Neuronata-R® showed a statistically significant improvement in CAFS (LS Mean 20.95 vs 13.66; 95% CI; p<0.024) compared with placebo. Group 2 also showed improvement (LS Mean 24.78 vs 17.92; 95% CI; p<0.041). In Group 2, significant divergence in SVC compared to the control group emerged from Month 8 post-treatment, suggesting a potential effect in delaying disease progression.
NfL Biomarker Reduction Mirrors Regulatory Precedent
CorestemChemon also highlighted the reduction in NfL levels — a biomarker that served as the basis for FDA accelerated approval in ALS, including the recent decision on Tofersen. In both the two-dose (Group 1) and five-dose (Group 2) arms, NfL levels consistently declined over time. Notably, in Group 2, NfL levels were significantly lower than placebo at both Month 4 and Month 10, suggesting a potential dose-dependent effect.
The consistency between the company's internal analysis and validation by an independent CRO strengthens the reliability of the dataset, which has been incorporated into the final CSR.
"This subgroup analysis lends strong support to a biomarker-driven approval strategy," a company official said. "The consistency of our internal analysis with external CRO validation adds credibility to the dataset and provides a concrete basis for regulatory discussions with the MFDS."
Regulatory Pathway Toward Accelerated Approval
The company views these findings as strategically significant, particularly in light of their alignment with the precedent set by Tofersen, where the FDA granted accelerated approval based on NfL reduction rather than survival benefit — a pathway CorestemChemon now seeks to pursue.
The company plans to request a Pre-BLA or Type C meeting with the FDA in Q3 2025, with the goal of submitting a biologics license application by Q4 2025 and targeting regulatory approval by mid-2026. CorestemChemon will finalize its submission package in collaboration with a global CRO and actively engage with regulatory agencies to pursue worldwide market entry for Neuronata-R®.
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.
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Neuronata-R® Stem Cell Therapy Shows Promise in ALS Phase 3 Subgroup Analysis, Moves Toward FDA Accelerated Approval
