Lumosa Therapeutics has announced encouraging findings from two Phase 2 clinical trials of its innovative stroke treatment, LT3001 (Odatroltide). The trials demonstrated significant functional improvements in patients suffering from disabling acute ischemic stroke (AIS), especially those who are not candidates for standard reperfusion therapies. Results were presented on February 7, 2026, at the International Stroke Conference in New Orleans, underscoring the potential of LT3001 to improve outcomes for many stroke patients.
The drug LT3001 represents a first-in-class dual-functional therapy that combines safe reperfusion with direct neuroprotection. This approach addresses the limitations of existing acute ischemic stroke treatments. In the LT3001-202 trial conducted in China, moderate stroke patients with disabling symptoms showed notable improvements, with an 8% increase in modified Rankin Scale (mRS) scores of 0-1 and a 13% increase in scores of 0-2 compared to those receiving a placebo. Additionally, patients with large artery atherosclerosis (LAA) demonstrated an 11% improvement in mRS 0-2 and a 9% gain in mRS 0-1.
Encouraging Results Across Trials
The complementary LT3001-205 trial, which involved participants from the United States, European Union, and Taiwan, utilized imaging-assisted patient selection to further validate these promising results. Although the sample sizes were smaller, the trial found that 27% of patients receiving LT3001 achieved mRS 0-1 outcomes, compared to only 17% in the placebo group.
Dr. Thomas Devlin, MD, PhD, FSVIN, the principal investigator of the LT3001-205 study, highlighted the favorable safety profile of LT3001. He noted that there was no increase in symptomatic intracranial hemorrhage (sICH) despite multi-dose administration over three days. “LT3001 showed potential benefit beyond the conventional thrombolytic time window, supporting its use in patients ineligible for IV thrombolysis or endovascular therapy (EVT),” Dr. Devlin stated. This population represents a significant unmet need in stroke care.
A New Therapeutic Pathway
Dr. Sheng-Wen Yeh (Mimi), Ph.D., General Manager of Lumosa, emphasized the importance of these developments. “Despite over five decades of research, the development of neuroprotective drugs for stroke has not resulted in any FDA-approved options in the U.S.,” he explained. “Our efficacy data with LT3001, which spans diverse patient populations, is exciting and provides direction for our Phase 3 programs.”
Acute ischemic stroke affects millions globally, often leading to severe disability and diminished quality of life. Current reperfusion therapies are not suitable for all patients due to timing constraints, contraindications, or anatomical factors. LT3001’s unique dual mechanism enhances the body’s natural clot-dissolving processes while protecting brain tissue from oxidative damage, offering a new therapeutic option for underserved patients.
Both Phase 2 trials employed rigorous randomized, placebo-controlled methodologies. The LT3001-202 trial enrolled 297 patients, while the LT3001-205 trial included 88 participants. Consistent positive signals across different geographic regions and patient selection criteria underscore the robustness of these findings.
Next Steps in Development
The promising results from these Phase 2 trials provide critical clinical evidence that supports the continued development of LT3001 for acute ischemic stroke. Lumosa Therapeutics has received feedback from the U.S. Food and Drug Administration regarding its Phase 3 development plans for LT3001. The company aims to accelerate global Phase 3 development to deliver innovative treatment options for stroke patients worldwide.
According to the World Health Organization (WHO), stroke is the second leading cause of death for individuals over the age of 60, with approximately six million deaths globally each year. Stroke can be classified as either hemorrhagic or ischemic, with ischemic strokes accounting for around 85% of all cases. The World Health Organization estimates that 15 million people worldwide suffer a stroke annually, highlighting a significant global unmet medical need.
Lumosa Therapeutics, a clinical-stage pharmaceutical company, is dedicated to developing novel therapies for neurologic diseases that require urgent attention. The company aims to enhance the quality of life for patients through innovative medical solutions and collaborations, focusing on the development of LT3001 as a critical treatment for acute ischemic stroke.
