MICOT PHARMA-B (02335): The Phase II clinical trial of MT200605 for the treatment of acute ischemic stroke (AIS) has reached its primary endpoint.

MICOT PHARMA-B (02335) announced that the Phase II clinical trial of its self-developed candidate drug MT200605 for the treatment of Acute Ischemic Stroke (AIS) has obtained topline data. The data shows that the clinical trial has achieved its primary endpoint, with results demonstrating statistical significance and clinical relevance. The clinical trial was a multicenter, randomized, double-blind, placebo-controlled study aimed at evaluating the efficacy, safety, and pharmacokinetics of MT200605, involving a total of 360 participants. The results analysis indicates: In terms of efficacy, the primary efficacy endpoint (percentage of patients with mRS score of 0-1 at 90 days) shows that both the medium-dose and high-dose groups of MT200605 had a higher efficacy rate than the placebo group, with the high-dose group showing more than a 100% improvement in the likelihood of achieving good functional outcomes compared to the placebo. Logistic regression analysis demonstrated that the difference between the high-dose and placebo groups was statistically significant. All secondary efficacy endpoints also exhibited the same trend of effectiveness. Regarding safety, MT200605 showed good safety and tolerability, with no serious adverse events (SAEs) related to the investigational drug occurring during the entire study period. Treatment-emergent adverse events (TEAEs) of grade 3 or higher were balanced across the groups, and no new significant safety risks were identified. Overall, MT200605 demonstrated significant and robust efficacy in patients with ischemic stroke, along with good safety and tolerability. To the best of the company's knowledge, MT200605 is the only agent globally that can cross the blood-brain barrier (BBB) and act as an agonist to BDNF/TrkB, while also providing neuroprotection through dual actions of antioxidant stress and relieving calcium ion-induced vascular smooth muscle contraction that leads to microcirculatory occlusion. This comprehensive blocking approach aims to mitigate the ischemic cascade injury triggered after ischemic stroke while promoting nerve regeneration and remodeling. The company is collaborating with Beijing Tiantan Hospital, affiliated with Capital Medical University, on a project titled "Innovative Drug Development and Clinical Trials for the Prevention and Treatment of Cardiovascular and Cerebrovascular Embolic Diseases," which is part of the National Science and Technology Major Project for Innovative Drug Research and Development. The company is primarily responsible for the clinical development and validation research of MT200605 (TrkB small molecule agonist) for patients with acute ischemic stroke, aiming to evaluate its efficacy and safety. In addition to treating acute ischemic stroke (AIS), MT200605 is also being developed for the treatment of Huntington's Disease (HD). MT200605 selectively activates the TrkB signaling pathway while providing dual neuroprotective and antioxidant effects to protect the structure and function of nervous tissue. Preclinical studies have shown that MT200605 has the potential to directly reduce the aggregation of mutant huntingtin protein (mHTT), thus providing a treatment strategy with potential disease-modifying effects for HD. In March 2026, MT200605 received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the HD indication.