- Breakthrough Phase 2 Trial Results Show Promise for Patients Ineligible for Standard Reperfusion Therapies
- Data Presented at International Stroke Conference
- Data Show That LT3001 Delivers Meaningful Functional Improvements in Patients Suffering from Disabling Acute Ischemic Stroke (AIS)
TAIPEI, Feb. 6, 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 dual-functional therapy that combines safe reperfusion with direct neuroprotection, addressing key limitations of current acute ischemic stroke treatments. These data revealed that LT3001 delivers meaningful functional improvements in patients suffering from disabling acute ischemic stroke (AIS), offering new hope for 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 LT3001’s potential to transform outcomes for stroke patients facing limited therapeutic options. The novel therapeutic agent, which enhances endogenous fibrinolysis and scavenges harmful free radicals, showed particularly impressive results in moderate stroke patients with disabling symptoms. In the LT3001-202 trial conducted in China, moderate stroke patients with disabling symptoms treated with LT3001 achieved 8% and 13% improvements in modified Rankin Scale (mRS) scores of 0-1 and 0-2 respectively, compared to placebo.
Most remarkably, LT3001 showed improvement in 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 annually.
The complementary LT3001-205 trial, spanning the US, EU, and Taiwan, utilized advanced imaging-assisted patient selection. Despite smaller sample sizes, the trial reinforced LT3001’s efficacy signals, with patients showing disabling features achieving mRS 0-1 outcomes more often with LT3001 (27%) compared to placebo (17%).
“Across the two Phase 2 trials, LT3001 demonstrated a favorable safety profile, with no increase in symptomatic intracranial hemorrhage (sICH) despite multi-dose administration over 3 days,” said Thomas Devlin, MD, PhD, FSVIN, Director of the CHI Memorial Neuroscience Institute, Professor of Neurology 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 high unmet need. The consistency of results across two independent trials, using different selection strategies, strengthens our confidence in LT3001’s broad applicability.”
Addressing a Critical Unmet Need
“Despite over five decades of research and thousands of potential drug targets, the development of neuroprotective drugs for conditions like stroke has not resulted in any FDA-approved drugs in the U.S.,” said Sheng-Wen Yeh (Mimi) Ph.D., general manager, Lumosa. “While many drugs frequently succeed in preclinical, animal-based studies, they fail to show safety or efficacy in human trials. Our efficacy data with LT3001, spanning diverse patient populations, is exciting and provides direction for our Phase 3 programs.”
Acute ischemic stroke affects millions globally, with many patients experiencing disabling symptoms that severely impact their quality of life and independence. Current reperfusion therapies, while effective, are not suitable for all patients due to timing constraints, contraindications, or anatomical factors. LT3001’s unique dual mechanism – enhancing the body’s natural clot-dissolving processes while protecting brain tissue from oxidative damage – offers a new therapeutic pathway for these underserved patients.
The trials evaluated patients within 24 hours of stroke onset, focusing on those with disabling symptoms defined as significant arm or leg motor impairment. This patient population represents a substantial portion of stroke cases where current treatment options remain inadequate. Both Phase 2 trials employed rigorous randomized, placebo-controlled methodologies, with LT3001-202 enrolling 297 patients and LT3001-205 including 88 patients. The consistency of positive signals across different geographic regions and patient selection criteria underscores the robustness of these findings.
About LT3001
LT3001 (Odatroltide) represents an innovative approach to acute stroke treatment, targeting both the 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 injury, potentially extending the therapeutic window and broadening patient eligibility.
The Company is advancing LT3001 through comprehensive clinical development, with plans for Phase 3 trials to further validate these promising results in larger patient populations.
Next Steps and Global Development Plans
These combined Phase 2 results provide critical clinical evidence supporting the continued development of LT3001 for acute ischemic stroke. Of note, Lumosa recently received feedback from the U.S. Food and Drug Administration regarding its Phase 3 development plans for LT3001.
Based on this feedback, Lumosa aims to accelerate global Phase 3 development of LT3001 and deliver innovative and effective treatment options for stroke patients worldwide.
About Acute Ischemic Stroke
According to the World Health Organization (WHO), stroke is the second leading cause of death for people over the age of 60 with approximately 6 million deaths in the world per year. Stroke can be categorized as hemorrhagic (bleeding) or ischemic (lack of blood flow to critical areas of the brain). Studies show ischemic stroke is most common, occurring in about 85% of all stroke cases. Stroke is notably a disease with significant global unmet medical need. There are 15 million people worldwide who suffer a stroke each year. Because only surgery and limited therapeutic options are currently available, 80% of stroke patients are left with no other treatment options or without desired outcomes.
About Lumosa
Lumosa Therapeutics, Inc. (https://www.lumosa.com.tw/) is a clinical-stage pharmaceutical company dedicated to the development of novel therapies and solutions for neurologic diseases with urgent unmet medical need. In addition to utilizing its own technology platform, the Company is also actively engaged in scientific licensing and co-development collaboration — building a pipeline from early to late-stage assets consisting of first-in-class and best-in-class drugs. Lumosa’s mission is to enrich patients’ quality of life through pioneering medical science, diverse collaborations, and a commitment to remain genuine and ever-evolving. Central to this mission is the Company’s objective to develop its novel small molecule, LT3001, for the treatment of acute ischemic stroke, a medical disease for which more effective therapies are greatly needed.
