Novel Neuroprotective Drug Improves Stroke Recovery in Phase III Trial

Beijing, China – September 10, 2026

A phase III clinical trial published today in JAMA provides new evidence that a novel neuroprotective drug may improve recovery for patients with acute ischemic stroke. The study found that intravenous loberamisal, when administered within 48 hours of symptom onset, significantly increased the proportion of patients achieving excellent functional outcomes at 90 days compared to placebo.

The LAIS (Loberamisal for Acute Ischaemic Stroke) trial enrolled 998 patients across 32 centers in China. Participants received either 40 mg of loberamisal or placebo as a one-hour intravenous infusion once daily for 10 days, starting within 48 hours of stroke onset.

Why Neuroprotection Has Eluded Stroke Medicine

Neuroprotective therapies have been a holy grail in stroke research for decades, with more than 1,000 failed clinical trials recorded since 1990. While reperfusion therapies like tissue plasminogen activator (tPA) and mechanical thrombectomy can restore blood flow, they remain inaccessible to most patients who arrive at hospitals beyond treatment windows.

Trial Design and Patient Population

The multicenter, randomized, double-blind, placebo-controlled trial targeted adults aged 18 to 80 years with acute ischemic stroke presenting within 48 hours of symptom onset. Patients had to have a baseline National Institutes of Health Stroke Scale (NIHSS) score between 7 and 20, indicating moderate to moderate-severe stroke severity, and a prestroke modified Rankin Scale (mRS) score of 0 or 1.

Randomization was performed using a central web-based system that assigned participants to receive loberamisal or placebo in a 1:1 ratio. Stratification occurred by center and onset-to-randomization time (≤12 hours vs. >12 hours). Neither investigators nor patients knew treatment assignments, and assessors remained masked throughout the study.

The primary efficacy outcome was the proportion of participants achieving an excellent functional outcome, defined as mRS 0-1 at 90 days. Secondary outcomes included favorable functional outcome (thresholds adjusted for baseline severity), mRS distribution at 90 days, and changes in NIHSS scores at 10 and 30 days.

Key Findings

Among the 998 enrolled patients, 499 were randomized to loberamisal and 499 to placebo. The median age was 63 years, and 66% were male. Approximately 77% of patients received their first dose within 12 hours of symptom onset.

The trial met its primary endpoint: 69.7% of loberamisal recipients achieved mRS 0-1 at 90 days compared with 56.4% of placebo recipients, yielding a risk ratio of 1.24 (95% confidence interval 1.12 to 1.36). Sensitivity analyses using alternative intercurrent-event strategies produced consistent results.

The treatment effect was maintained across prespecified subgroups, including patients stratified by time to treatment and baseline stroke severity. In the exploratory analysis of depression and anxiety symptoms at 90 days, loberamisal recipients reported significantly lower rates of severe depression and moderate-to-severe anxiety compared with placebo recipients.

Safety Profile

Loberamisal demonstrated a favorable safety profile. Serious adverse events occurred in 8.6% of treated patients versus 10.7% of placebo recipients. All-cause mortality was low in both groups (1.2% for loberamisal versus 2.0% for placebo), with no statistically significant differences. No suspected unexpected serious adverse reactions were identified in either treatment group.

"This is the first neuroprotective agent to show a clear functional benefit in a phase III trial. The results suggest that targeting both excitotoxicity and GABA receptor modulation simultaneously may be more effective than single-target approaches." — Dr. Shuya Li, Beijing Tiantan Hospital and Capital Medical University

Implications for Stroke Care

The findings could reshape acute stroke management for patients who miss the narrow window for tPA administration or are not candidates for mechanical thrombectomy. Approximately two-thirds of ischemic stroke patients currently receive only supportive care, focusing on secondary prevention and rehabilitation.

Dr. Yongjun Wang, the study's principal investigator, emphasized that follow-up trials are needed to confirm these results in more diverse populations. "We need to validate whether these benefits extend to patients in other countries and those with more severe strokes," Wang said. "Large, multinational trials are planned to test this agent in broader patient populations."

The trial was sponsored by NeuroDawn Pharmaceutical, which developed loberamisal, with additional support from the National Natural Science Foundation of China. Study registration: NCT06517173.

Source: JAMA, Published online September 10, 2026. Li S, Feng B, He D, Wang X, Li H, Xu S, et al; LAIS Investigators. Loberamisal for Acute Ischemic Stroke: The LAIS Randomized Clinical Trial. JAMA. doi:10.1001/jama.2026.16557.