METiS TechBio's AI-Designed Drug MTS-004 Becomes First in China to Complete Phase III Trial for Pseudobulbar Affect
核心洞察
MTS-004 has become China's first AI-enabled formulation drug candidate to successfully complete Phase III clinical trials, marking a significant milestone in AI-driven pharmaceutical development.
The drug targets Pseudobulbar Affect (搜索) (PBA (搜索)), a neurological condition causing involuntary laughing and crying episodes, representing the first and only therapy for this indication to reach Phase III in China.
Using METiS TechBio (搜索)'s proprietary AiTEM platform, the drug development timeline was compressed from the typical industry average to just 38 months from concept to Phase III completion.
METiS TechBio (搜索) announced that its internally developed small-molecule candidate MTS-004 has successfully met the primary endpoint in its Phase III clinical trial, becoming China's first drug candidate designed by AI-driven formulation technologies to complete Phase III development. The achievement represents a major breakthrough in addressing an unmet clinical need for Pseudobulbar Affect (搜索) (PBA (搜索)), a neurological condition for which no approved treatments currently exist in China.
Revolutionary AI-Driven Development Timeline
MTS-004 utilized METiS TechBio (搜索)'s proprietary AiTEM platform, powered by the company's NanoForge AI nanodelivery system, to dramatically accelerate development timelines. The platform uses advanced algorithms integrating quantum chemistry and molecular dynamics simulations to predict drug-excipient interactions and generate nanoscale formulation optimization solutions.
In the MTS-004 project, AiTEM enabled predictive modeling that shortened the preclinical formulation optimization period from the industry average of 1-2 years to under 3 months. From program initiation to Phase III completion, the drug completed a full development cycle in just 38 months, significantly accelerating the path from discovery to clinical validation.
Addressing Critical Unmet Medical Need
Pseudobulbar Affect (搜索), commonly known as "pathological laughing and crying," is a neurological condition characterized by sudden, involuntary, and inappropriate episodes of laughing or crying that do not match a patient's true emotional state. PBA (搜索) frequently occurs secondary to central nervous system disorders such as amyotrophic lateral sclerosis (搜索) (ALS (搜索)), multiple sclerosis (搜索) (MS), stroke (搜索), traumatic brain injury (搜索) (TBI), or Parkinson's disease (搜索) (PD). The condition can severely impair social functioning and quality of life while exacerbating the burden of underlying neurological diseases.
Currently, the only approved treatment worldwide for PBA (搜索) is NUEDEXTA® in the U.S. MTS-004, classified in China as a Category 2.2 improved new drug, introduces an innovative orally disintegrating tablet (ODT) formulation that effectively addresses swallowing difficulties—an unmet need among PBA patients—and provides a user-friendly alternative to capsule formulations.
Comprehensive Phase III Trial Results
The Phase III trial was led by Professor Dongsheng Fan, Chair of Neurology at the Peking University Third Hospital and a leading expert in ALS (搜索) and neurodegenerative disorders. This randomized, double-blind, placebo-controlled, multicenter study evaluated the efficacy and safety of MTS-004 in patients with PBA (搜索) secondary to ALS, MS, stroke (搜索), TBI, or PD.
The trial enrolled 264 patients across 48 top-tier hospitals nationwide over a period of 9.5 months, including Beijing Xuanwu Hospital, Beijing Tiantan Hospital (搜索), West China Hospital Sichuan University (搜索), The First Affiliated Hospital of China Medical University, and other leading medical institutions.
Results demonstrated that MTS-004 was safe, well-tolerated, and effective in reducing PBA (搜索) symptoms in patients with neurological disorders. Therapeutic benefits were observable as early as week 2, with continued increases in symptom-free days and sustained improvement throughout the 12-week treatment period.
Significant Clinical Outcomes
MTS-004 achieved statistically significant improvements over placebo in multiple key measures:
- CNS-LS (Center for Neurologic Study–Lability Scale) total score
- CGI-C (Clinical Global Impression of Change) and PGI-C (Patient Global Impression of Change)
- SF-36 Mental Health Index, reflecting enhanced emotional regulation and quality of life
Additionally, MTS-004 significantly improved CNS-BFS (搜索) (Bulbar Function Scale) score, particularly in swallowing and drooling symptoms—critical complications in patients with motor neuron and central nervous system diseases.
Expert Perspectives and Future Outlook
Professor Dongsheng Fan, Principal Investigator, commented: "Artificial intelligence is reshaping how new drugs are discovered and developed. The MTS-004 clinical trial was challenging to initiate but was completed with outstanding quality. Its ability to improve patients' quality of life—especially its clear impact on swallowing function—can reduce complications and may positively influence survival. While this study focused on ALS (搜索)-related PBA (搜索), MTS-004 shows potential across broader neurological populations."
Dr. Chris Lai, Co-founder and CEO of METiS TechBio (搜索), stated: "AI's mission in medicine is to empower clinicians and race against diseases, driven by a deep understanding of unmet patient needs. That mission inspired our 38-month sprint from concept to Phase III completion. MTS-004 marks METiS TechBio's first end-to-end success from discovery to late-stage clinical validation—a foundation that will accelerate every pipeline that follows."
MTS-004 is expected to submit a New Drug Application (NDA) to the National Medical Products Administration (NMPA) in 2026 for the PBA (搜索) indication. Following approval, post-marketing studies are planned to explore potential label expansion for the treatment of dysphagia (搜索).