METiS TechBio, a global leader in AI-driven nanodelivery and formulation innovation, has announced that its small-molecule candidate MTS-004 has successfully met the primary endpoint in its Phase III clinical trial.

According to the company, 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.

MTS-004 is a novel orally disintegrating tablet (ODT) developed using METiS TechBio’s proprietary AiTEM platform, powered by the company’s NanoForge AI nanodelivery system.  Designed specifically for patients with swallowing difficulties (dysphagia), the formulation dissolves rapidly in the mouth without the need for water, improving taste, adherence, and overall patient experience.

Pseudobulbar Affect (PBA)—commonly known as "pathological laughing and crying"—is a neurological condition characterised by sudden, involuntary and inappropriate episodes of laughing or crying that do not match a patient's true emotional state. It frequently occurs secondary to central nervous system disorders such as amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), stroke, traumatic brain injury (TBI) or Parkinson's disease (PD).

Currently, the only approved treatment worldwide for PBA is NUEDEXTA in the US. In China, MTS-004 is classified as a Category 2.2 improved new drug, introducing an innovative ODT formulation that offers a user-friendly alternative to conventional capsules.

The Phase III trial, led by Professor Dongsheng FAN, Chair of Neurology at the Peking University Third Hospital, was a randomised, double-blind, placebo-controlled, multicenter study evaluating the efficacy and safety of MTS-004 in patients with PBA secondary to ALS, MS, stroke, TBI or PD.

Results demonstrated that MTS-004 was safe, well-tolerated and effective in reducing PBA symptoms in patients with neurological disorders, with therapeutic benefits observed as early as week two, with continued increases in symptom-free days and sustained improvement throughout the 12-week treatment period. 

The drug achieved statistically significant improvements over placebo in 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 (Bulbar Function Scale) score, particularly in swallowing and drooling symptoms—critical complications in patients with motor neuron and central nervous system diseases.

METiS TechBio plans to submit a New Drug Application (NDA) to China’s National Medical Products Administration (NMPA) in 2026 for the PBA indication, followed by post-marketing studies to explore potential expansion into dysphagia treatment.

“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,” Dr. said Chris Lai, Co-founder and CEO of METiS TechBio.