Investigational Therapy Associated with Reduced Infarct Growth in Individuals with Acute Ischemic Stroke

Treatment with the investigational therapy RNS60 (Revalesio, Tacoma, WA) was associated with a significant reduction in infarct growth and clinically meaningful improvements for individuals with acute ischemic stroke (AIS) treated within 12 hours of symptom onset. These results were presented at the American Stroke Association’s International Stroke Conference 2025. RNS60 is designed to reduce inflammation and increase mitochondrial biogenesis and function by activating intracellular signaling pathways.

The multicenter, double-blind, placebo-controlled, randomized, phase 2 RESCUE clinical trial (NCT04693715) included 82 individuals with AIS who were eligible for endovascular thrombectomy (EVT). Participants received an intravenous infusion of either RNS60 at low dose (0.5 mL/kg/h), high dose (1.0 mL/kg/h), or placebo for 48 hours, which was initiated before EVT was performed. The primary endpoints included safety and mortality. Efficacy endpoints included disability (measured using the modified Rankin Scale [mRS]), infarct volume (measured by MRI at 48 hours), and scores on the Barthel Index (BI), EQ-5D-5L index, and National Institutes of Health Stroke Scale (NIHSS). Researchers monitored participants for 90 days of follow-up.

Participants treated with high dose RNS60 demonstrated the following results compared with placebo:

• A 50% reduction in infarct growth at 48 hours post-EVT (nominal P<.05).
• A 16% higher number of participants having mRS scores of 0-2 (independence) at day 90 (22% predicted probability; odds ratio [OR], 3.7; P=.36).
• An increase in the return to normal activities of daily living (BI ≥ 95) at day 90 (71% in high dose RNS60 group vs 43% in placebo group; OR, 5.8; P=.13).
• An improvement in EQ-5D-5L index score (0.74 ± 0.13 in high dose RNS60 group vs 0.58 ± 0.13 in placebo group; P=.09)
• An improvement in NIHSS score (absolute value and change from baseline) at each pre-specified time point.

Additionally, treatment with RNS60 was shown to be safe and well tolerated.

"These are exciting results that suggest that RNS60 should be evaluated in a large Phase 3 clinical trial that will hopefully confirm its benefits and lead to approval as the first therapeutic agent in decades to demonstrate significant efficacy in improving outcomes for acute ischemic stroke patients," said Marc Fisher, MD, neurologist at Beth Israel Deaconess Medical Center.