MTS-004 eases pseudobulbar affect in people with MS, other disorders
Data: Improvement in uncontrolled laughing, crying seen as early as 2 weeks
Metis Biotech‘s experimental oral therapy MTS-004 was safe and eased symptoms of pseudobulbar affect (PBA), a neurological condition marked by bouts of sudden, involuntary, and inappropriate laughter or crying, in people with multiple sclerosis (MS) and other neurological disorders.
That’s according to data from a Phase 3 clinical trial that took place at 48 sites in China. The trial enrolled 264 people with PBA secondary to MS, amyotrophic lateral sclerosis, Parkinson’s disease, stroke, or traumatic brain injury.
MTS-004’s therapeutic benefits were observed as early as two weeks, and patients had continued increases in symptom-free days and lasting improvement throughout the 12-week treatment period, according to a company press release.
Because MTS-004 significantly outperformed a placebo at easing PBA symptoms, Metis Biotech plans to submit an application to China’s regulators in 2026 seeking approval of the drug for the treatment of PBA.
Pseudobulbar affect marked by involuntary outbursts of laughing or crying
Pseudobulbar affect is characterized by sudden, involuntary outbursts of laughing or crying that don’t reflect a person’s actual feelings. The exact cause of PBA is not completely known, but it is thought to be related to a disruption in the brain’s circuits that regulate the expression of emotions.
As a result, it frequently occurs in people with disorders affecting the brain and spinal cord, leading to feelings of embarrassment, social isolation, and mental health issues that can impact patients’ quality of life.
Currently, Nuedexta (dextromethorphan hydrobromide and quinidine sulfate) is the only medication approved in the U.S. for the treatment of PBA. However, Nuedexta is available as capsules that can be challenging for people with swallowing difficulties, or dysphagia, which is a common complication of certain neurological disorders, including MS.
MTS-004 is an oral disintegrating tablet developed with Metis’ proprietary AiTEM platform, which uses advanced algorithms to design and optimize drug formulations.
The medication was specifically tailored to those with frequent dysphagia. It dissolves rapidly in the mouth without the need for water and offers a pleasant taste, which is expected to improve treatment adherence and patient experience.
AI-powered platform shortens MTS-004’s preclinical development period
The company’s AI-powered platform enables researchers to perform predictive modeling, which shortened the preclinical development of MTS-004 from the industry average of about 1 to 2 years to less than three months.
MTS-004’s full development cycle, from program initiation to Phase 3 completion, was completed in a matter of months.
“Artificial intelligence is reshaping how new drugs are discovered and developed,” said Dongsheng Fan, MD, PhD, the chair of neurology at the Peking University Third Hospital in China and the trial’s principal investigator.
Metis’ CEO Chris Lai, PhD, sees these initial positive results as something to build on.
“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,” said Lai, who is also the co-founder of Metis.
The Phase 3 trial randomly assigned participants to receive MTS-004 or a placebo for 12 weeks, or about three months. After that period, MTS-004 significantly eased PBA symptoms, as assessed by a self-administered questionnaire that measures the frequency of PBA episodes.
Artificial intelligence is reshaping how new drugs are discovered and developed.
Improvements were also observed in clinician- and patient-reported assessments of PBA symptoms and in mental health. In addition, MTS-004 significantly reduced swallowing and drooling symptoms.
MTS-004’s “ability to improve patients’ quality of life — especially its clear impact on swallowing function — can reduce complications and may positively influence survival,” Fan said.
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