Neurodevelopmental disorder therapy
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Publication Number
US-11839600-B2
Publication Date
2023-12-12
Expiration Date
Abstract
This invention addresses tetrahydro-N, N-dimethyl-2,2diphenyl-3-furanmethanamine hydrochloride (ANAVEX2-73, AV2-73, or A2-73) in a method of treatment for neurodevelopmental disorders. Particular reference is made to the treatment of autism spectrum disorder, cerebral palsy, Rett syndrome, Angelman syndrome, Williams syndrome, pervasive developmental disorder not otherwise specified (PDD-NOS), childhood disintegrative disorder, and Smith-Magenis syndrome. Additional reference is made to multiple sclerosis.
Core Innovation
The disclosed invention treats neurodevelopmental disorders in a subject associated with loss-of-function mutations of the gene encoding methyl-CpG binding protein 2 (MeCP2) by administering a composition comprising a therapeutically effective amount of a therapeutic agent selected from Anavex2-73 (A2-73), an analog of Anavex2-73, or any combination thereof. The disclosure focuses on MeCP2-related disease conditions and describes rationale tied to receptor binding and prior neuroprotection evidence for A2-73.
The disclosure states that the treatment is associated with improvements in reported clinical and physiological readouts, including breathing variability, cardiac pulse variability, seizure reduction, and improvements related to choking and weight gain, sleep, and gadolinium-enhancing lesion reduction on MRI. The disclosure further includes preclinical evaluation in MeCP2 heterozygous knockout mouse studies, assessing locomotor and neurological readouts to support partial rescue of behavioral and gait phenotypes.
The disclosure further describes chronic administration and general pharmaceutical formulation routes, including oral dosing, injectable administration, transdermal formulations, and sustained release. It also states dosing ranges and dosing schedules for subjects, including adults and infants or children, while providing examples of patient regimens with reported improvements.
Claims Coverage
The independent claim covers one inventive feature: administering a therapeutically effective amount of Anavex2-73 (A2-73), an analog of Anavex2-73, or any combination thereof, to ameliorate a condition associated with loss-of-function mutations of the MeCP2-encoding gene.
Administering Anavex2-73 or analogs to ameliorate MeCP2 loss-of-function conditions
A method of ameliorating a condition associated with loss-of-function mutations of the gene encoding methyl-CpG binding protein 2 (MeCP2) in a subject in need thereof, comprising administering to the subject a composition comprising a therapeutically effective amount of a therapeutic agent selected from the group consisting of Anavex2-73, an analog of Anavex 2-73, and any combination thereof.
The claim set centers on administration of Anavex2-73 or analogs for MeCP2 loss-of-function–associated conditions, with dependent claims further specifying associated condition manifestations, the disease list, and administration by formulation route, dose amount, and dosing frequency.
Stated Advantages
Reported improvements include breathing variability, cardiac pulse variability, seizure reduction, improvements related to choking and weight gain, sleep, and gadolinium-enhancing lesion reduction on MRI.
Documented Applications
Treating conditions associated with loss-of-function mutations of the gene encoding MeCP2, including autism spectrum disorder, Rett syndrome, cerebral palsy, Angelman syndrome, Williams syndrome, PDD-NOS, childhood disintegrative disorder, Smith-Magenis syndrome, and multiple sclerosis.
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