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Publication Number
US-12486262-B2
Publication Date
2025-12-02
Expiration Date
Abstract
The present disclosure features compounds useful for the treatment of BAF complex-related disorders.
Core Innovation
The patent discloses compounds of Formula (I), or pharmaceutically acceptable salts thereof, for treating neurofibromatosis in a subject in need thereof. The compounds include BRG1/BRM inhibitor candidates with specific stereoisomers and isotopologues, such as pyrrole-3-carboxamide enantiomers and isotopologues.
The treatment is associated with BRG1 and/or BRM activity, including BRG1 loss-of-function mutation contexts and BRG1 loss of function disorder. The document links BRG1/BRM inhibition to BAF complex-related disorders, including neurofibromatosis and cancer.
The partial content further states that the compounds are evaluated for BRM/BRG1 ATPase catalytic activity using an ADP-Glo ATPase assay and reports IP50 potency against BRM/BRG1 for specific stereoisomers. It also reports cellular growth inhibition observations across cancer cell line panels and comparative cellular efficacy versus PKC and MEK inhibitors.
Claims Coverage
The independent claim coverage centers on a method of treating neurofibromatosis by administering an effective amount of a compound of Formula (I) or a pharmaceutically acceptable salt. The inventive features define the Formula (I) structure and restrict treatment to BRG1 loss-of-function mutation or disorder contexts, with dependent features requiring measurable BRG1 reduction for a specified duration.
Treating neurofibromatosis with Formula (I) compounds
A method of treating neurofibromatosis in a subject in need thereof by administering an effective amount of a compound of Formula (I) or a pharmaceutically acceptable salt thereof.
Formula (I) defined by a depicted chemical structure
The method uses a compound of Formula (I) having the specific chemical structure shown in the provided images.
Neurofibromatosis associated with a BRG1 loss of function mutation
The method is limited to neurofibromatosis that is associated with a BRG1 loss of function mutation.
Subject with a BRG1 loss of function disorder
The method further determines that the subject has a BRG1 loss of function disorder.
Reducing BRG1 level and/or activity by at least 5%
The method is carried out with an effective amount of a compound that reduces the level and/or activity of BRG1 by at least 5% compared to a reference.
Reducing BRG1 level and/or activity by at least 5% for at least 12 hours
The method uses an effective amount of the compound to reduce the level and/or activity of BRG1 by at least 5% versus a reference for at least 12 hours.
The claims cover treating neurofibromatosis with a compound of Formula (I), with refinements that specify the Formula (I) structure and restrict use to BRG1 loss-of-function mutation or disorder contexts. Dependent features further require BRG1 level and/or activity reduction by at least 5%, optionally for at least 12 hours.
Stated Advantages
Reduces BRG1/BRM activity.
Induces apoptosis.
Reduces tumor growth and metastasis.
Documented Applications
Treating neurofibromatosis in a subject in need thereof by administering a compound of Formula (I).
Treating BAF complex-related disorders including neurofibromatosis and cancer, including contexts described as reducing tumor growth and metastasis.
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