Lumosa Therapeutics has announced positive results from two independent Phase 2 clinical trials of its novel stroke drug LT3001 (odatroltide). LT3001 is described by the company as a first-in-class dual-functional therapy that combines safe reperfusion with direct neuroprotection, addressing “key limitations” of current acute ischaemic stroke treatments.
Data reported at the 2026 International Stroke Conference (ISC; 4–6 February, New Orleans, USA) indicate that LT3001 delivers meaningful functional improvements in patients suffering from disabling acute ischaemic stroke, offering “new hope” for those who cannot receive standard reperfusion treatments, Lumosa claims.
The novel therapeutic agent—which enhances endogenous fibrinolysis and scavenges harmful free radicals—showed “particularly impressive” results in moderate stroke patients with disabling symptoms. The LT3001-202 trial conducted in China revealed that 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, as compared to a placebo.
Furthermore, LT3001 demonstrated improvements in functional outcomes in large artery atherosclerosis (LAA) and mismatch-positive populations within the LT3001-202 study, as LAA patients (n=169) showed improvements of 11% in mRS 0–2 and 9% in mRS 0–1.
Additionally, the complementary LT3001-205 trial—spanning the USA, EU and Taiwan—validated these signals via advanced imaging-assisted patient selection, with mismatch-positive patients achieving a 10% absolute improvement in mRS 0–2. Lumosa also notes in a recent press release that, despite smaller sample sizes, the LT3001-205 study ultimately reinforced LT3001’s efficacy signals, as patients presenting with disabling features achieved an outcome of mRS 0–1 more often with LT3001 compared to a placebo (27% vs 17%, respectively).
“Across the two Phase 2 trials, LT3001 demonstrated a favourable safety profile, with no increase in symptomatic intracranial haemorrhage [sICH] despite multi-dose administration over three days,” said Thomas Devlin (CHI Memorial Neuroscience Institute, Chattanooga, USA), the LT3001-205 study’s principal investigator. “LT3001 showed potential benefit beyond the conventional thrombolytic time window, supporting its use in patients ineligible for IV [intravenous] thrombolysis or EVT [endovascular therapy]—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.”
“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 [Food and Drug Administration]-approved drugs in the USA,” added Lumosa general manager Sheng-Wen Yeh. “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 programmes.”
The company’s recent press release states that current reperfusion therapies, while effective, are not suitable for all patients due to timing constraints, contraindications, or anatomical factors. However, LT3001’s unique dual mechanism whereby it enhances the body’s natural clot-dissolving processes while protecting brain tissue from oxidative damage may offer a new therapeutic pathway for these underserved patients.
The trials from which late-breaking data were presented at ISC 2026 evaluated patients within 24 hours of stroke onset, focusing on those with disabling symptoms defined as significant arm or leg motor impairment—a population that represents “a substantial portion of stroke cases where current treatment options remain inadequate”. Both Phase 2 trials employed randomised, placebo-controlled methodologies, with LT3001-202 enrolling 297 patients and LT3001-205 including 88. According to Lumosa, the consistency of positive signals across different geographic regions and patient selection criteria underscores the robustness of the findings presented at ISC last week.
The company is currently advancing LT3001 through comprehensive clinical development and plans to conduct Phase 3 trials to further validate these promising results in larger patient populations.
