Micot Pharma has reported positive topline results from a Phase II clinical trial of MT200605, its investigational small-molecule therapy for acute ischemic stroke, with the study meeting its primary endpoint.
The multicentre, randomised, double-blind, placebo-controlled trial enrolled 360 patients with acute ischemic stroke.
At 90 days, the proportion of patients achieving a modified Rankin Scale score of 0–1 was higher in both the medium- and high-dose MT200605 groups than in the placebo group. In the high-dose group, the proportion of patients reaching this functional outcome was more than double that of the placebo group, with a statistically significant between-group difference.
Consistent efficacy trends were also observed across secondary endpoints.
No serious adverse events related to the investigational drug were reported during the trial. Treatment-emergent adverse events of grade 3 or higher were evenly distributed across the treatment groups, and no new safety risks were identified.
MT200605 is being developed as a bifunctional neuroprotective therapy designed to target multiple stages of ischemic brain injury.
According to Micot Pharma, the candidate crosses the blood-brain barrier and targets the BDNF/TrkB pathway while exerting neurorestorative activity.
Its proposed mechanism involves improving cerebral perfusion through modulation of calcium signalling in vascular smooth muscle, limiting oxidative and excitotoxic injury, and supporting neurological repair through sustained TrkB activation.
The company said sustained TrkB activation is intended to mimic the activity of endogenous brain-derived neurotrophic factor, supporting processes including synaptogenesis, axonal regeneration and neural network remodelling.
MT200605 was selected in May 2026 for China’s National Science and Technology Major Project for Innovative Drug Development, supporting its continued development in cardiovascular and cerebrovascular disease.
The candidate also received US Food and Drug Administration Orphan Drug Designation for Huntington’s disease in March 2026.
Micot Pharma has also been notified that an abstract titled “MT200605: A Novel Bifunctional Neuroprotectant for Ischemic Stroke” has been accepted for oral presentation at the 18th World Stroke Congress 2026.
The presentation will cover non-clinical and Phase I findings for MT200605, while the positive Phase II results provide the clinical basis for advancing the candidate into the next stage of development.
