In trial, Neuronata-R benefits ALS patients with slower progression
Developer plans to file FDA application next year for therapy's approval
In adults with amyotrophic lateral sclerosis (ALS) who have slower disease progression, the stem cell therapy Neuronata-R (lenzumestrocel) — conditionally approved in South Korea for treating ALS — was found to help preserve daily function and breathing capacity, while also reducing signs of nerve damage and inflammation.
That’s according to complete data from ALSUMMIT (NCT04745299), a Phase 3 clinical trial underway at five South Korean sites that’s testing the therapy’s safety and effectiveness. The study was evaluating how well Neuronata-R works, for as long as three years, to slow disease progression in these patients.
Developer Corestemchemon now plans to seek a meeting with the U.S. Food and Drug Administration (FDA), before the end of the year, to discuss these results, per a company press release. The bio company then expects to file a biologics license application next year seeking accelerated approval of Neuronata-R in the U.S.
This strategy builds on past regulatory decisions, like the FDA’s approval of Biogen’s Qalsody (tofersen) for treating ALS, the release stated.
The new data were presented earlier this month by Ki-Uk Oh, MD, PhD, principal investigator of ALSUMMIT and a professor at Hanyang University Hospital in Seoul, at the PACTALS 2025 conference in Australia. According to the developer, “meaningful improvements [were] confirmed in [the] slow-progressor subgroup” in the trial.
ALS is a progressive disease in which motor neurons — the nerve cells responsible for controlling muscle movement — gradually degenerate and die, leading to a loss of muscle strength and control. Over time, this can limit patients’ daily activities, such as walking, eating, and speaking, and shorten their lifespan.
Neuronata-R uses mesenchymal stem cells, a type of adult stem cell that can be collected from a patient’s bone marrow. These stem cells can renew themselves and develop into many other types of cells, which may help repair damaged motor neurons.
After being grown in the lab, these cells are mixed with cerebrospinal fluid, the liquid that surrounds the brain and spinal cord, and returned to the patient through an intrathecal injection, or directly into the spinal canal.
Complete ALSUMMIT data show Neuronata-R preserved lung function
ALSUMMIT was launched in 2021 after a Phase 1/2 clinical trial (NCT01363401) involving 72 adults with ALS showed that Neuronata-R had the potential to slow the disease’s progression. The Phase 3 trial was designed to confirm the benefits of Neuronata-R in an estimated 115 patients diagnosed with familial or sporadic ALS less than two years previously.
The participants, ages 25 to 75, were randomly assigned to one of three groups. One group received two intrathecal injections of Neuronata-R followed by three of a placebo (group 1), while another was given five intrathecal injections of Neuronata-R (group 2). Group three, the control group, received only the placebo.
In all groups, the first two injections were given 26 days apart, and the remaining ones at four, seven, and 10 months.
Neuronata-R did not meet the main goal of improving Combined Assessment of Function and Survival (CAFS) scores — a measure that combines ALS Functional Rating Scale-Revised (ALSFRS-R) scores and survival — after six months. However, benefits were seen among patients whose disease progressed more slowly.
After 12 months, patients with slower disease progression who had received treatment with Neuronata-R had significantly higher ALSFRS-R scores on average than those on the placebo (31.2 vs. 26.4 points). The ALSFRS-R scores patients on daily activities, and higher scores indicate better function.
Benefits seen only among patients with slower disease progression
This subgroup of patients also scored significantly higher on CAFS after one year (20.95 in group 1 and 24.78 in group 2 vs. 17.92 points in the placebo), confirming that individuals treated with Neuronata-R fared better in daily activities and lived longer in better health compared with those on the placebo.
Lung function was tested using slow vital capacity, which measures how much air a person can slowly exhale after taking a deep breath. Patients in group 2 who were treated with Neuronata-R had significantly higher slow vital capacity than those on the placebo (62.2% vs. 50.6%), suggesting the therapy helps preserve lung function.
Researchers also looked at the levels of neurofilament light chain, a biomarker of damage to nerve cells, and monocyte chemoattractant protein 1, a biomarker of inflammation. Both biomarkers were lowered in Neuronata-R-treated patients, suggesting the therapy may protect motor neurons and reduce inflammation.
“[The] findings confirm Neuronata-R’s potential to preserve function and respiratory capacity in ALS — outcomes directly linked to quality of life and survival,” the release stated.
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