Lumosa Reports Positive Phase 2 Data for LT3001 in Acute Ischemic Stroke

Lumosa Reports Positive Phase 2 Data for LT3001 in Acute Ischemic Stroke

By, Admin 9 February 2026

Lumosa Therapeutics announced positive results from two independent Phase 2 trials evaluating LT3001 (odatroltide) in patients with disabling acute ischemic stroke (AIS).

The data were presented at the International Stroke Conference in New Orleans. LT3001 demonstrated meaningful functional improvements, particularly in patients ineligible for standard reperfusion therapies, a group with limited treatment options.

Why LT3001 Is Different?

LT3001 is a first-in-class, dual-functional therapy designed to address two major gaps in current stroke care.

  • Enhances endogenous fibrinolysis to support safe reperfusion
  • Provides direct neuroprotection by scavenging harmful free radicals

This dual mechanism aims to extend the therapeutic window and expand eligibility beyond IV thrombolysis or endovascular therapy.

Phase 2 Trial Overview

LT3001-202 (China)

  • Design: Randomized, placebo-controlled
  • Patients enrolled: 297
  • Population: Moderate AIS with disabling symptoms

Key outcomes vs placebo:

  • +8% improvement in mRS 0–1
  • +13% improvement in mRS 0–2

Subgroup Highlights

  • Large artery atherosclerosis (LAA) patients (n=169):
    • +11% improvement in mRS 0–2
    • +9% improvement in mRS 0–1

LT3001-205 (US, EU, Taiwan)

  • Design: Randomized, placebo-controlled
  • Patients enrolled: 88
  • Selection: Imaging-assisted, mismatch-positive patients

Key outcomes:

  • 27% achieved mRS 0–1 with LT3001
  • 17% achieved mRS 0–1 with placebo

Mismatch-positive patients showed a 10% absolute improvement in mRS 0–2, validating signals seen in Study 202.

Consistent Efficacy Across Trials

Despite differences in geography, enrollment size, and selection strategy:

  • Functional outcome improvements were directionally consistent
  • Benefits were observed in patients beyond conventional thrombolytic windows
  • Signals were strongest in disabling stroke subgroups

These findings suggest broad applicability in real-world AIS populations.

Safety Profile

Across both Phase 2 studies:

  • No increase in symptomatic intracranial hemorrhage (sICH)
  • Multi-dose administration over three days was well tolerated

“LT3001 demonstrated a favorable safety profile with no increase in sICH,”

  • Dr. Thomas Devlin, Principal Investigator, LT3001-205.

Addressing a Long-Standing Unmet Need

Despite decades of research:

  • No FDA-approved neuroprotective drugs exist for stroke in the US
  • Many candidates fail when translated from animal models to humans

Lumosa believes LT3001’s human efficacy signals across diverse populations provide a strong rationale for late-stage development.

Patient Population Studied

  • Treated within 24 hours of stroke onset
  • Disabling symptoms defined by significant arm or leg motor impairment
  • Represents a large proportion of real-world stroke patients

This is the group most often excluded from existing reperfusion options.

Next Steps: Phase 3 Development

Lumosa has received US FDA feedback on its Phase 3 development strategy.

What’s Next?

  • Accelerated global Phase 3 program
  • Larger, confirmatory trials planned
  • Focus on patients ineligible for standard reperfusion therapies

Big Picture Takeaway

LT3001 does not compete directly with clot-removal therapies. It targets the patients left behind. If Phase 3 data confirm these results, LT3001 could become:

  • The first effective neuroprotective therapy in AIS
  • A viable option beyond current time and eligibility limits

That would be a real shift in stroke treatment.

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