Despite significant improvements in early reperfusion therapy, there remains a need for cerebro-protective therapies to enhance the brain’s resilience to and recovery from a stroke.

RNS60 is an experimental neuroprotective drug that showed significant promise in rodent and primate models of stroke. RESCUE is a proof-of-concept trial to test RNS60 treatment in patients with large vessel occlusion and acute ischemic stroke.

We utilized a multicenter, placebo controlled, blinded Phase 2 study design in participants with large vessel occlusion who are eligible for endovascular thrombectomy. The study includes participants with an NIHSS score >5, ASPECTS >5, evidenced presence of penumbra/collaterals, and pre-stroke mRS score ≤2. Participants received a 48-h infusion of 0.5 mL/kg/h RNS60, 1 mL/kg/h RNS60, or 1 mL/kg/h placebo starting prior to arterial access closure and were followed for 90 days. Randomization was 1:1:1 with block urn randomization to balance age, NIHSS, and ASPECTS. Imaging post-EVT occurred at ~2 hours, 48 hours, and 90 Days.

The study recruited 82 participants at multiple sites across the U.S. The primary objective is safety (proportion of participants with serious adverse events and mortality at 90 days). Secondary efficacy endpoints include changes in infarct volume,  mRS score, NIHSS, Barthel Index, mortality, and worsening of stroke over 90 days in the RNS60 vs. placebo groups. Exploratory endpoints include imaging biomarkers, plasma biomarkers, and quality of life measured by the EQ-5D-5L. An independent DSMB reviewed the safety data three times during the study and recommended continuing without any changes. Results of the study are expected in Q1 of 2024.