METiS TechBio says its internally developed small-molecule candidate MTS-004 has successfully met the primary endpoint in its phase III clinical trial.
MTS-004 is China’s first drug candidate designed by AI-driven formulation technologies to complete phase III development, and the first and only therapy for pseudobulbar affect (PBA) in the country to reach this milestone—representing a major step toward addressing an unmet clinical need in neurological disorders.
MTS-004 is a novel oral formulation designed for the treatment of PBA, utilizing METiS TechBio’s AiTEM platform to develop orally disintegrating tablet (ODT). Tailored for patients frequently experiencing dysphagia, MTS-004 dissolves rapidly in the mouth without the need for water, offering a pleasant taste and significantly improving medication adherence and patient experience.
PBA, also 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 frequently occurs secondary to central nervous system disorders such as amyotrophic lateral sclerosis, multiple sclerosis, stroke, traumatic brain injury, or Parkinson’s disease. The condition can severely impair social functioning and quality of life, while exacerbating the burden of underlying neurological diseases.
The AiTEM platform, powered by METiS TechBio’s NanoForge AI nanodelivery system, uses advanced algorithms to design and optimise pharmaceutical formulations. By integrating quantum chemistry and molecular dynamics simulations, AiTEM predicts drug-excipient interactions and generates nanoscale formulation optimisation solutions to enhance solubility, bioavailability, and pharmacokinetic performance.
In the case of MTS-004, AiTEM enabled predictive modelling shortened the preclinical formulation optimization period from the industry average of 1 to 2 years to under 3 months. From programme initiation to phase III completion, MTS-004 completed a full development cycle of in months, significantly accelerating the path from discovery to clinical validation.
The trial was led by Dongsheng Fan, chair of Neurology at the Peking University Third Hospital and a leading expert in ALS and neurodegenerative disorders. It enrolled 264 patients across 48 hospitals nationwide over a period of 9.5 months.
Currently, the only approved treatment worldwide for PBA is NUEDEXTA in the US. MTS-004, classified in China as a Category 2.2 improved new drug, introduces an ODT formulation that effectively addresses swallowing difficulties—an unmet need among PBA patients—and provides a user-friendly alternative to capsule formulations.
Results from the study demonstrated that MTS-004 was safe, well-tolerated, and effective in reducing PBA 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. MTS-004 significantly improved CNS-BFS (Bulbar Function Scale) score, particularly in swallowing and drooling symptoms—critical complications in patients with motor neuron and central nervous system diseases.
MTS-004 is expected to submit a New Drug Application (NDA) to the National Medical Products Administration (NMPA) in 2026 for the PBA indication. Following approval, post-marketing studies are planned to explore potential label expansion for the treatment of dysphagia.
Principal investigator Fan said: “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-related PBA, MTS-004 shows potential across broader neurological populations.”
Chris Lai, co-founder and CEO of METiS TechBio, said: “We extend our sincere gratitude to Professor Fan, all participating investigators and clinical staff, every patient and family, and the community supporting PBA research. 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.”
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