Revalesio, a clinical-stage pharmaceutical company developing treatments for acute and chronic neurological disorders, today announced new data showing that Revalesio’s investigational therapeutic, RNS60, protected motor neurons in the prpTDP-43^A315T-UeGFP mouse model of ALS. PrpTDP-43^A315T-UeGFP mice express a mutation of the human TDP43 gene and develop progressive motor neuron degeneration, leading to gait abnormalities, muscle weakness, and eventually death, mimicking key features of ALS. TDP43-associated pathology is found in approximately 97% of people with ALS. The results were presented today during the Nanosymposium ALS Therapeutics at the Society for Neuroscience Annual Meeting.

“There is a great need for treatment options to slow the progression of ALS,” said Hande Ozdinler, Ph.D., Associate Professor of Neurology at Northwestern University Feinberg School of Medicine and Principal Investigator of the study. “These promising preclinical findings warrant further investigation of the potential role of RNS60 in protecting motor neurons and preserving mitochondrial function, key factors in the pathogenesis of ALS.”

“These exciting results suggest that RNS60 has potential to mitigate neurodegeneration due to TDP-43 pathology,” said Mukesh Gautam, PhD, Research Assistant Professor of Neurology at Northwestern University Feinberg School of Medicine and presenting author at the Society for Neuroscience Annual Meeting. “Since TDP-43 pathology contributes to a large population of both familial and sporadic ALS patients, these results are particularly promising.”

In this study, 30-day old prpTDP-43^A315T-UeGFP mice showing signs of degeneration were treated with RNS60 every other day for 60 days. Immunohistochemistry was performed to quantitatively assess activated microglia (immune cells that patrol the central nervous system [CNS] for pathogens and damage) and activated astrocytes (cells in the CNS that perform metabolic, structural, homeostatic, and neuroprotective tasks). Ultrastructural integrity of neuronal mitochondria was studied via electron microscopy. RNS60 treatment significantly a) improved mitochondrial ultrastructure in the motor neurons, b) reduced the extent of astrogliosis and microgliosis in mice and c) protected health and stability of upper motor neurons and neuromuscular junctions compared to placebo-treated mice.

“These results add to the growing body of preclinical and clinical evidence that RNS60 may provide neuroprotective effects in ALS,” said Bert van den Bergh, Revalesio’s Executive Chairman on the Board of Directors, and former President of Neuroscience Products at Eli Lilly and Company. “We are simultaneously advancing RNS60 in planned clinical trials for the treatment of patients with ALS and ischemic stroke with the goal of improving the lives of people with these devastating conditions.”

About RNS60

RNS60 is an investigational therapeutic being developed to provide disease modifying and potentially restorative treatments for neurological diseases. In preclinical studies, RNS60 activated intracellular signaling pathways to increase mitochondrial biogenesis and function and reduce inflammation. RNS60 safely protected neurons and oligodendrocytes and modulated the activity of immune cells to restore homeostasis. RNS60 has been granted Orphan Drug and Fast Track designations for ALS from the U.S. Food and Drug Administration.

About Revalesio

Revalesio is a clinical-stage pharmaceutical company with a vision to change the future of treatment for acute and chronic neurological disorders. The company’s lead clinical program for RNS60 is ischemic stroke with additional programs in ALS and other neurological disorders. The company’s pioneering technology, founded in physics, addresses fundamental mechanisms involved with proper cellular function to slow disease progression and improve quality of life.

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Media:
Lori Murray
lori.murray@captivate-comms.com