Balipodect for the treatment or prevention of autism spectrum disorder

PDE10A inhibitors, such as 1-[2-fluoro-4-(1H-pyrazole-1-yl)phenyl]-5-methoxy-3-(1-phenyl-1H-pyrazole-5-yl)pyridazine-4(1H)-one, provide a promising treatment for autism spectrum disorders by addressing motor and seizure symptoms with reduced side effects.

JP2026086421APending Publication Date: 2026-05-26TAKEDA PHARMA CO LTD
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Patent Information

Application Number
JP2026003939
Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2018-09-28
Filing Date
2026-01-14
Publication Date
2026-05-26

AI Technical Summary

Technical Problem

Current treatments for autism spectrum disorders such as CDKL5 deficiency and Fragile X syndrome are limited, with a need for disease-modifying therapies and effective management of seizures and behavioral issues.

Method used

The use of PDE10A inhibitors, specifically compounds like 1-[2-fluoro-4-(1H-pyrazole-1-yl)phenyl]-5-methoxy-3-(1-phenyl-1H-pyrazole-5-yl)pyridazine-4(1H)-one, to treat or prevent autism spectrum disorders by administering an effective dose to mammals, potentially combined with a second active ingredient.

Benefits of technology

PDE10A inhibitors demonstrate efficacy in reducing motor impairment, hyperactivity, and seizure frequency in mouse models of CDKL5 deficiency and Fragile X syndrome, with potential for improved therapeutic outcomes through reduced side effects compared to existing antipsychotics.

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Abstract

To provide treatment or preventive medication for autism spectrum disorder. [Solution] The present invention provides a PDE10A inhibitor for treating or preventing autism spectrum disorder. The inhibitor is 1-[2-fluoro-4-(1H-pyrazole-1-yl)phenyl]-5-methoxy-3-(1-phenyl-1H-pyrazole-5-yl)pyridazine-4(1H)-one or a salt thereof, and is a therapeutic or prophylactic agent for autism spectrum disorder selected from the group consisting of childhood disintegrative disorder, Rett syndrome, Cleefstra syndrome, Pitt-Hopkins syndrome, Angelman syndrome, Kabuki syndrome, Asperger syndrome, Heller syndrome, and pervasive developmental disorder.
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