Revalesio has announced new analyses of the completed Phase 2 RESCUE clinical trial evaluating its drug candidate RNS60 in acute ischaemic stroke that demonstrate a nominally significant lowering of infarct growth in patients treated less than 12 hours from last known well. The lowering of infarct growth correlated with clinically meaningful improvements in several functional stroke measures for assessing a patient’s recovery, including the modified Rankin scale (mRS), Barthel index (BI), and National Institutes of Health stroke scale (NIHSS), the company claims in a recent press release.
These results were delivered during an oral presentation at the ongoing International Stroke Conference (ISC; 5–7 February, Los Angeles, USA).
“This analysis of the RESCUE trial adds further information regarding the beneficial effects of RNS60, confirming that it has favourable effects on MRI [magnetic resonance imaging]-confirmed ischaemic lesion growth that translated into improved clinical outcomes,” said former World Stroke Organization (WSO) president Marc Fisher (Beth Israel Deaconess Medical Center, Boston, USA). “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 ischaemic stroke patients.”
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 endovascular therapy (EVT) were enrolled and received either intravenous RNS60 0.5mL/kg/h (low dose), RNS60 1mL/kg/h (high dose), or placebo, starting before completion of EVT and continuing for 48 hours.
The trial had two primary endpoints: safety and mortality. Secondary endpoints for the study assessed disability based on mRS scores, changes in the size of the stroke measured via MRI at 48 hours, and additional standard stroke scales like BI and NIHSS.
Highlights from the oral presentation—given by Revalesio’s acting chief medical officer Jordan Dubow on 5 February at ISC 2025—are as follows:
- The high dose of RNS60 significantly lowered infarct growth by 50% (nominal p<0.05) when compared to placebo, based on imaging performed at approximately 48 hours compared to immediately post-EVT, both in patients treated within 12 hours and 24 hours of last known well
- The high dose of RNS60 was also numerically better than placebo for each prespecified functional endpoint at day 90 (mRS, with 72% of subjects on high-dose RNS60 being independent [mRS 0–2] at day 90 compared to 37% on placebo; BI, with 72% of subjects on high-dose RNS60 returning to normal activities of daily living [BI³ 95] compared to 37% on placebo; and patient health status as measured by the EQ-5D-5L index, with 1 being ideal, which was 0.79 for RNS60 subjects and 0.57 for placebo)
- RNS60 was safe and well tolerated
“As the number-one cause of disability worldwide, the impact of stroke is staggering, accounting for US$721 billion annually to the global healthcare system,” said Bert van den Bergh, Revalesio’s executive chairman of the board of directors. “Additionally, more than 80% of patients in the USA have no treatment options following a stroke, underscoring the significant need for new and effective treatment options. These highly encouraging results further demonstrate the potential of RNS60 to greatly reduce the likelihood of disability in patients following a stroke, and we plan to advance RNS60 into a Phase 3 clinical trial in order to bring this promising therapy to patients.”
Initial topline results from the RESCUE Phase 2 study—which evaluated patients who were 24 hours from last known well, and saw RNS60 meet its safety- and mortality-related endpoints—were presented as a late-breaking oral presentation at last year’s ISC (7–9 February 2024, Phoenix, USA).
