Phase 2 Odatroltide Trials Show Promise for Acute Ischemic Stroke Patients
Key Clinical Summary
- LT3001 (odatroltide) improved functional outcomes in patients with disabling acute ischemic stroke in 2 independent phase 2 trials.
- Benefits were observed beyond standard reperfusion windows, including in patients ineligible for intravenous thrombolysis (IVT) or endovascular therapy.
- No increase in symptomatic intracranial hemorrhage was reported despite multi-day dosing.
Data from 2 phase 2 trials suggest that LT3001 (odatroltide), a novel dual-mechanism stroke therapy, may offer functional improvement in patients with acute ischemic stroke who cannot receive standard reperfusion treatments, announced manufacturer Lumosa Therapeutics. Findings were presented at the 2026 International Stroke Conference in New Orleans.
Study Findings
The randomized, placebo-controlled trials, LT3001-202 (n = 297) and LT3001-205 (n = 88), evaluated LT3001, which enhances endogenous fibrinolysis and scavenges free radicals, in patients within 24 hours of stroke onset and significant motor impairment of the arm or leg.
In Study 202, which was conducted in China, moderate stroke patients with disabling symptoms treated with LT3001 achieved 8% improvements in modified Rankin Scale (mRS) scores of 0-1 and 13% improvements in mRS scores of 0-2 compared with placebo.
Data from Study 202 further indicated that LT3001 led to functional improvements in patients with large artery atherosclerosis (LAA; n = 169), who saw an 11% improvement in mRS 0-2 and a 9% improvement in mRS 0-1 versus placebo.
In Study 205, which was conducted across the United States, Taiwan, and Europe and used advanced imaging-assisted selection, mismatch-positive patients saw a 10% absolute improvement in mRS 0-2 outcomes. Study 205 also demonstrated consistent efficacy signals, as 27% of patients with disabling features achieved mRS 0–1 compared with 17% in the placebo group.
Across both trials, LT3001 demonstrated a favorable safety profile, with no observed increase in symptomatic intracranial hemorrhage despite multi-dose administration over 3 days.
Clinical Implications
Many acute ischemic stroke patients may be ineligible for existing reperfusion therapies due to time constraints, anatomical factors, or contraindications. LT3001’s observed benefits beyond conventional thrombolytic windows suggest that the agent could expand treatment eligibility to a broader population, including patients with delayed presentation or those unsuitable for intravenous thrombolysis (IVT) or endovascular therapy.
If confirmed in larger-scale, phase 3 studies, LT3001 could represent a meaningful advance for patients with disabling acute ischemic stroke who have limited therapeutic options.
Expert Commentary
“The consistency of results across 2 independent trials, using different selection strategies, strengthens our confidence in LT3001's broad applicability,” said Thomas Devlin, MD, PhD, FSVIN, Director of the CHI Memorial Neuroscience Institute, Professor of Neurology, Morehouse School of Medicine and lead investigator of the LT3001-205 study, in a news release.
"Despite over 5 decades of research and thousands of potential drug targets, the development of neuroprotective drugs for conditions like stroke has not resulted in any Food and Drug Administration (FDA)-approved drugs in the US," added Sheng-Wen Yeh (Mimi), PhD, 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."
