Phase III Trial Shows Neuroprotective Drug Loberamisal Improves Stroke Recovery When Administered Early

Stroke patients treated intravenously with loberamisal, a novel neuroprotective medication, daily for 10 days and starting within 48 hours of stroke symptoms, had better recovery than patients who received a placebo, according to a preliminary late-breaking science presentation at the American Stroke Association’s International Stroke Conference 2026. The study is a Phase III clinical trial, a large-scale study to evaluate the effectiveness of a new treatment. The study goal is to test loberamisal, a new-generation dual-target treatment strategy designed to protect brain cells within the first 2 days after a stroke.

Neuroprotective agents may help improve patient outcomes since they are aimed at preserving the function of neurovascular units. However, trials for most of these agents have not been successful. In this trial, we tested loberamisal, a small-molecule, dual-acting neuroprotective agent that was an effective neuroprotectant in rodent studies. New treatments for stroke may come from multi-target neuroprotective agents, which could lead to important advancements in reducing or preventing disability after a stroke. The American Stroke Association’s new 2026 Guideline for the Early Management of Patients With Acute Ischemic Stroke notes that neuroprotection has garnered renewed interest. Current knowledge gaps need to be addressed in future research.

In this study trial participants were patients who received stroke care at 32 centers in China. 998 adults, ages 18 to 80, were treated for 10 days with either a daily, intravenous infusion of 40 mg loberamisal for 10 days or a matched placebo, started within 48 hours of a moderate to severe stroke caused by a blocked vessel. All had a confirmed clogged brain-vessel stroke, and treatment began within 48 hours of when stroke symptoms began. Only about 17% of participants received standard IV clot-busting medication, limiting assessment of combined effects of both treatments. Patients who received surgical treatment for the blockage were excluded from the trial.

At 90 days after treatment, the analysis found 69% of participants treated with loberamisal had excellent functional recovery compared to about 56% in the placebo group. The treatment was considered safe because patients did not appear to have an increased risk of serious side effects or death compared to those in the placebo group. Study limitations include that the trial was conducted only in China, therefore, the results cannot be directly translated to people living in other countries. We want to confirm our findings with larger groups of people, including people from different racial and ethnic backgrounds, patients with more severe strokes and those who also have had vascular surgery. We need to better understand how loberamisal works by studying biomarkers in multiple population groups.

Other limitations were that most patients in the study had moderate to severe strokes, which may affect applicability to people who have a more severe stroke. No blood or imaging biomarkers were assessed, which limits the applicability of the study’s understanding of how loberamisal affects the body. Researchers conducted the multicenter, randomized, double-blind, parallel, placebo-controlled Phase III clinical trial over a 9-month period, from July 2024 to April 2025. Although 20 participants did not complete the 90-day follow-up assessment, they were still included in the final statistical analysis. Randomization to loberamisal or placebo was computer-generated; neither the investigators nor the participants knew who was receiving the medication or the placebo.

Trial participants all had National Institutes of Health Stroke Scale scores between 7 and 20, indicating moderate to severe stroke. Functional outcomes were measured on the modified Rankin Scale. A score of 0-1 indicates little to no disability. This score was assessed and determined by trained and certified researchers at the 90-day follow-up, conducted via face-to-face interviews or standardized telephone questionnaires using a structured assessment form. Patients were excluded from the study if they had any history of bleeding strokes; severe consciousness impairment; transient attacks; blood pressure higher than 220/120 mm Hg with blood pressure treatment; history of severe mental health condition, dementia, depression or anxiety; severe liver or kidney disease; had undergone vascular surgery; had malignant tumors with a life expectancy of less than 90 days; were pregnant or lactating; had known allergy to loberamisal; major surgery scheduled within four weeks; or if they had participated in another clinical trial.

Statements and conclusions of studies that are presented at the American Heart Association/American Stroke Association’s scientific meetings are solely those of the study authors and do not necessarily reflect the Association’s policy or position. The Association makes no representation or guarantee as to their accuracy or reliability. Abstracts presented at the Association’s scientific meetings are not peer-reviewed, rather, they are curated by independent review panels and are considered based on the potential to add to the diversity of scientific issues and views discussed at the meeting. The findings are considered preliminary until published as a full manuscript in a peer-reviewed scientific journal. According to the American Heart Association’s 2026 Heart Disease and Stroke Statistics, stroke is now the #4 leading cause of death in the U.S.