Revalesio has announced new data from its completed Phase 2 RESCUE clinical trial of RNS60 in acute ischaemic stroke patients, demonstrating that patients were—on average—discharged 4.8 days sooner from the hospital following treatment with RNS60 plus standard-of-care endovascular therapy (EVT) compared to patients who only received EVT (p=0.022). A recent press release details that patients who received RNS60 also experienced a >50% reduction in brain infarct volume growth post-EVT (p<0.05).
These results were delivered during an oral presentation at the 2025 American Academy of Neurology (AAN) annual meeting (5–9 April, San Diego, USA).
“Revalesio’s choice to capture infarct growth post-EVT is a great example of how Phase 2 clinical trials should be designed to properly evaluate cytoprotective drugs with the use of imaging to confirm results,” said David Liebeskind (University of California Los Angeles [UCLA], Los Angeles, USA). “I congratulate the investigators and Revalesio on their results, and look forward to seeing RNS60 in a Phase 3 trial.”
In addition to the aforementioned findings suggesting reduced infarct growth and statistically significantly earlier hospital discharge with a high dose of RNS60, highlights from the oral presentation include 55% of patients treated with high-dose RNS60 being discharged to their home compared to 21% of those treated with placebo; 72% of patients on high-dose RNS60 being independent compared to 37% on placebo, based on dichotomised modified Rankin scale (mRS) scores of 0–2 at day 90; and RNS60 being safe and well tolerated overall.
“Our Phase 2 trial results sum up a decade’s worth of research where the consistency in results has been remarkable. In preclinical models of stroke and traumatic brain injury, RNS60 reduced brain loss by up to 50%,” said Greg Archambeau, president of Revalesio and co-inventor of RNS60. “Upon a potential FDA [Food and Drug Administration] approval, we will be ready to rapidly scale our specialised US manufacturing capability for RNS60 so patients won’t have to wait.”
In RESCUE—a multicentre, double-blinded, placebo-controlled, randomised Phase 2 clinical trial—Revalesio evaluated the safety and initial efficacy of RNS60. Eighty-two participants with acute ischaemic stroke eligible for EVT were enrolled and received intravenous RNS60 0.5mL/kg/h (low dose), RNS60 1mL/kg/h (high dose), or placebo, starting before completion of the EVT and continuing for 48 hours.
The trial had two primary endpoints: safety and mortality. Secondary endpoints for the study evaluated disability based on mRS, change in the extent of stroke as measured by magnetic resonance imaging (MRI) at 48 hours, and additional endpoints including Barthel index, National Institutes of Health stroke scale (NIHSS), and EQ-5D-5L.
“There is a lot of discussion currently taking place on improving human health while lowering the cost of healthcare,” commented Bert van den Bergh, Revalesio’s executive chairman of the board of directors and former president of neuroscience products at Eli Lilly and Company. “Given RNS60’s Phase 2 results showing reduced brain tissue loss, improved function, and reductions in both hospital stay and the need for long-term care, RNS60 has the potential to greatly benefit patients and their families while lowering the economic burden of stroke.”
