Revalesio's Investigational RNS60 Extended Survival in Patients with Amyotrophic Lateral Sclerosis (ALS) in Post-Hoc Analysis of Phase 2 Investigator-Sponsored Trial

RNS60 Also Slowed the Decline of Respiratory Function in ALS Patients 

Results Published in Brain, Behavior, and Immunity

TACOMA, Wash., Sept. 17, 2024 /PRNewswire/ -- Revalesio, a clinical-stage pharmaceutical company developing treatments for acute and chronic neurological disorders, today announced the publication of data in the journal Brain, Behavior, and Immunity, showing that Revalesio's investigational therapeutic, RNS60, extended median survival by 6 months compared to placebo in a post-hoc analysis of a Phase 2 investigator-sponsored trial. The trial was coordinated by the Research Center for ALS, Istituto di Ricerche Farmacologiche Mario Negri IRCCS in Milano, Italy.

After a 6-month double-blind treatment phase, participants were followed for a mean duration of 2.8 years and monitored for survival. Median survival was 6 months longer in the RNS60 treatment group compared to placebo (3 years vs. 2.4 years, p = 0.0519). As reported by the trial investigators in an earlier publication, the decline of forced vital capacity (FVC) - the maximum volume of air that a patient can exhale after a maximum inspiration, used as a primary test to evaluate breathing function of people living with ALS - was significantly slower in the RNS60 group compared to the placebo group at the end of treatment (FVC, difference 0.41 per week, standard error 0.16, p = 0.0101), showing a positive effect of RNS60 on respiratory and bulbar function. The new analysis showed that the extension of survival correlated with the rate of decline of FVC (median survival was 3.7 years in participants with a slow decline of FVC and 1.6 years in participants with faster FVC decline, p < 0.0001). Survival in the RNS60 group was significantly longer compared to the placebo group in participants with low baseline levels of neurofilament light chain, NfL (p = 0.012) or monocyte chemoattractant protein-1, MCP-1 (p < 0.01). Blood levels of NfL, a marker of neuronal damage, have been shown to correlate with the progression rate of ALS. MCP-1 is an inflammatory marker whose blood levels have been reported to increase during the course of ALS. These biomarker results suggest that this could be a subgroup to target in future studies investigating the effects of RNS60 on survival.

"These promising findings suggest that RNS60 may have a beneficial effect on survival and slowing the decline of respiratory function in patients with ALS," said Letizia Mazzini, Professor of Neurology and Director of the Tertiary ALS Center at the University of Piemonte Orientale and participating center in the Phase 2 clinical trial. "These results warrant further investigation, and I look forward to the future development of RNS60 for the treatment of ALS."

"With these encouraging results, RNS60 has shown benefit across two randomized clinical trials in ALS and ischemic stroke, underscoring its potential to impact patients with both acute and chronic neurological disorders. We are committed to advancing RNS60 in additional clinical trials to further evaluate RNS60's potential to benefit people with ALS and other neurological disorders," 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. "On behalf of Revalesio, I would like to thank the investigators for conducting this important additional analysis, as well as the patients and their families for their participation. I would also like to thank the ALS Association for contributing to the original study through the ALS Ice Bucket Challenge."

RNS60 Phase 2 Clinical Trial Design

The investigator-sponsored study was a multi-center, randomized, double-blind, placebo-controlled Phase 2 clinical trial, evaluating 147 people living ALS. Patients were enrolled across 22 Italian expert ALS centers and randomized to either RNS60 (n=74) or placebo (n=73) for 24 weeks. Participants were diagnosed with ALS and classified as slow or fast progressors with respect to FVC based on the FVC slope of decline during trial treatment. The primary endpoints were safety and certain biomarker levels associated with ALS and secondary endpoints were the ALS functional rating scale, FVC and survival.

Following the 6-month treatment phase, participants were monitored for survival to support the post-hoc analysis.

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.

For more information, please visit revalesio.com and engage with us on LinkedIn.

Media
Lori Murray
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SOURCE Revalesio