- Groundbreaking Phase 2 Trial Results Show Promise for Patients Not Eligible for Standard Reperfusion Therapies
- Data Presented at International Stroke Conference
- Data Shows LT3001 Provides Significant Functional Improvements in Patients With Disabling Acute Ischemic Stroke (AIS)
TAIPEI, February 7, 2026 /PRNewswire/ — Lumosa Therapeutics (Lumosa; 6535.TWO) today announced positive results from two independent Phase 2 clinical trials with its novel stroke drug LT3001 (Odatroltide). LT3001 is a first-in-class bifunctional therapy that combines safe reperfusion with direct neuroprotection, addressing key limitations of current treatments for acute ischemic stroke. These data revealed that LT3001 provides significant functional improvements in patients suffering from disabling acute ischemic stroke (AIS), providing new hope to those who cannot receive standard reperfusion treatments. These data were reported here today at the International Stroke Conference in New Orleans.
The groundbreaking results demonstrate the potential of LT3001 to transform outcomes for stroke patients facing limited treatment options. The new therapeutic agent, which enhances endogenous fibrinolysis and eliminates harmful free radicals, has proven to be particularly effective. Impressive Results in Moderate Stroke Patients with disabling symptoms. In the LT3001-202 trial in China, moderate stroke patients with disabling symptoms treated with LT3001 achieved Improvements of 8% and 13% in modified Rankin Scale (mRS) scores of 0-1 and 0-2 respectively, compared to placebo.
The most remarkableLT3001 showed improved functional outcomes in LAA and Mismatch-positive populations. In Study 202, LAA patients (n=169) showed an 11% improvement in mRS 0-2 and a 9% gain in mRS 0-1. Study 205 validated these signals via imaging-assisted selection, with mismatch-positive patients achieving a 10% absolute improvement in mRS 0–2. These clinical advances could restore independence to thousands of patients each year.
The companion trial LT3001-205, conducted in the United States, European Union and Taiwan, used advanced imaging-assisted patient selection. Despite smaller sample sizes, the trial strengthened LT3001’s efficacy signals, with patients with disabling features achieving mRS 0-1 results. more often with LT3001 (27%) compared to placebo (17%).
“Across both Phase 2 trials, LT3001 demonstrated a favorable safety profile, with no increase in symptomatic intracranial hemorrhage (sICH) despite multidose administration over 3 days,” said Thomas Devlin, MD, PhD, FSVIN, director of the CHI Memorial Neuroscience Institute, professor of neurology at the Morehouse School of Medicine, and principal investigator of the LT3001-205 study. “LT3001 showed potential benefit beyond the conventional thrombolytic time window, supporting its use in patients ineligible for IV thrombolysis or EVT, a population with significant unmet need. The consistency of results from two independent trials, using different selection strategies, reinforces our confidence in the broad applicability of LT3001.
Addressing a critical unmet need
“Despite more than five decades of research and thousands of potential drug targets, the development of neuroprotective drugs for conditions such as stroke has not resulted in any FDA-approved drugs in the United States,” said Sheng-Wen Yeh (Mimi) Ph.D., Chief Executive Officer of Lumosa. “Even though many drugs are often successful in preclinical animal studies, they fail to demonstrate safety or effectiveness in human trials. Our efficacy data with LT3001, spanning diverse patient populations, is exciting and informs our Phase 3 programs.”
Acute ischemic stroke affects millions of people worldwide, and many patients experience disabling symptoms that have serious consequences on their quality of life and independence. Current reperfusion therapies, although effective, are not suitable for all patients due to time constraints, contraindications, or anatomic factors. The unique dual mechanism of the LT3001 – enhancing the body’s natural clot dissolution processes while protecting brain tissue from oxidative damage – provides a new therapeutic avenue for these underserved patients.
The trials evaluated patients within 24 hours of stroke onset, focusing on those with disabling symptoms defined as significant motor impairment of the arm or leg. This patient population represents a significant portion of stroke cases for which current treatment options remain inadequate. Both phase 2 trials used rigorous randomized, placebo-controlled methodologies, with LT3001-202 including 297 patients and LT3001-205 including 88 patients. The consistency of positive signals across different geographic regions and patient selection criteria highlights the robustness of these results.
About the LT3001
LT3001 (Odatroltide) represents an innovative approach to the treatment of acute stroke, targeting both thrombotic and oxidative components of stroke pathophysiology. Unlike traditional approaches that focus solely on clot removal, LT3001’s dual mechanism addresses multiple aspects of stroke, potentially expanding the therapeutic window and expanding patient eligibility.
The Company is advancing LT3001 into full clinical development, with plans for Phase 3 trials to further validate these promising results in broader patient populations.
Next steps and global development plans
These combined Phase 2 results provide crucial clinical evidence supporting the continued development of LT3001 for the treatment of acute ischemic stroke. It should be noted that Lumosa recently received back from the United States Food and Drug Administration regarding its Phase 3 development plans for LT3001.
Based on this feedback, Lumosa aims to accelerate the global Phase 3 development of LT3001 and bring innovative and effective treatment options to stroke patients worldwide.
About acute ischemic stroke
According to the World Health Organization (WHO), stroke is the second leading cause of death in people over the age of 60, with approximately 6 million deaths worldwide per year. Stroke can be classified as hemorrhagic (bleeding) or ischemic (lack of blood flow to critical areas of the brain). Studies show that ischemic stroke is the most common, occurring in approximately 85% of all stroke cases. Notably, stroke is a disease with significant unmet medical need globally. Every year, 15 million people around the world suffer a stroke. Because only surgery and limited treatment options are currently available, 80% of stroke patients are left without other treatment options or desired outcomes.
About Lumosa
Lumosa Therapeutics, Inc. (https://www.lumosa.com.tw/) is a clinical-stage pharmaceutical company dedicated to developing novel therapies and solutions for neurological diseases with urgent unmet medical needs. In addition to utilizing its own technology platform, the Company is also actively engaged in scientific licensing and co-development collaboration – building an early to late-stage pipeline of assets comprised of first and best quality medicines. Lumosa’s mission is to enrich patients’ quality of life through pioneering medical science, diverse collaborations, and a commitment to remaining authentic and constantly evolving. At the heart of this mission is the Company’s goal to develop its novel small molecule, LT3001, for the treatment of acute ischemic stroke, a disease for which more effective therapies are greatly needed.
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SOURCE Lumosa Therapeutics Co., Ltd.
