Revalesio's RNS60 Protects Mitochondria in ALS Preclinical Model

Revalesio, a clinical-stage pharmaceutical firm based in Tacoma, Washington, has disclosed promising new data on its investigational treatment, RNS60, in the fight against Amyotrophic Lateral Sclerosis (ALS). The findings were shared during the Nanosymposium ALS Therapeutics at the Society for Neuroscience Annual Meeting.

ALS, a debilitating neurological disorder, is characterized by the gradual degeneration of motor neurons, leading to muscle weakness, gait abnormalities, and ultimately, death. The study focused on prpTDP-43A315T-UeGFP mice, which express a mutation of the human TDP43 gene. This mutation results in progressive motor neuron degeneration, closely mimicking human ALS pathology. Notably, TDP43-associated pathology is observed in approximately 97% of ALS patients.

According to Hande Ozdinler, Ph.D., Associate Professor of Neurology at Northwestern University Feinberg School of Medicine, there is a significant need for new treatments to slow the progression of ALS. Ozdinler, who is also the Principal Investigator of the study, emphasized that the preclinical findings with RNS60 are promising enough to warrant further research into its potential role in protecting motor neurons and preserving mitochondrial function, which are critical factors in ALS pathogenesis.

Mukesh Gautam, Ph.D., Research Assistant Professor of Neurology at Northwestern University Feinberg School of Medicine and the presenting author at the conference, highlighted that RNS60 may have the potential to mitigate neurodegeneration caused by TDP-43 pathology. Given that this pathology affects a significant portion of both familial and sporadic ALS patients, the results are particularly encouraging.

The study involved treating 30-day-old prpTDP-43A315T-UeGFP mice, which were already showing signs of degeneration, with RNS60 every other day for a period of 60 days. The research team employed immunohistochemistry to quantitatively assess activated microglia and activated astrocytes. Additionally, electron microscopy was used to study the ultrastructural integrity of neuronal mitochondria. The treatment with RNS60 significantly improved mitochondrial ultrastructure in motor neurons, reduced astrogliosis and microgliosis (inflammation of astrocytes and microglia, respectively), and protected the health and stability of upper motor neurons and neuromuscular junctions compared to the placebo-treated group.

Bert van den Bergh, the Executive Chairman of Revalesio's Board of Directors and former President of Neuroscience Products at Eli Lilly and Company, stated that these results contribute to the growing body of preclinical and clinical evidence suggesting that RNS60 may offer neuroprotective benefits for ALS patients. Revalesio is currently advancing RNS60 in planned clinical trials aimed at treating ALS and ischemic stroke, with the ultimate goal of improving the lives of individuals suffering from these severe conditions.

RNS60 is an investigational therapeutic in development intended to provide disease-modifying and potentially restorative treatments for neurological diseases. Preclinical studies have shown that RNS60 activates intracellular signaling pathways to enhance mitochondrial biogenesis and function while reducing inflammation. It has demonstrated safety in protecting neurons and oligodendrocytes and in modulating immune cell activity to restore homeostasis. The U.S. Food and Drug Administration has granted RNS60 both Orphan Drug and Fast Track designations for ALS.

Revalesio aims to revolutionize treatments for acute and chronic neurological disorders. The company's leading clinical program focuses on ischemic stroke, with additional efforts directed towards ALS and other neurological diseases. Through pioneering technology rooted in physics, Revalesio strives to address fundamental cellular functions to slow disease progression and improve patients' quality of life.