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Methods of treating phelan mcdermid syndrome using farnesyl dibenzodiazepinones

A technology of farnesyl dibenzodiazepine and syndrome, which is applied in the field of using farnesyl dibenzodiazepine to treat Phelan McDermid syndrome, can solve the problems of no approval and few clinical studies, etc.

Active Publication Date: 2020-08-28
AMO PHARMA LTD
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0006] Although studies of the role of intranasal oxytocin in PMS are ongoing in the United States, there are relatively few clinical studies of pharmacological interventions in PMS (ClinicalTrials.gov identifierNCT02710084)
There Are No Approved PMS Treatments

Method used

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  • Methods of treating phelan mcdermid syndrome using farnesyl dibenzodiazepinones
  • Methods of treating phelan mcdermid syndrome using farnesyl dibenzodiazepinones
  • Methods of treating phelan mcdermid syndrome using farnesyl dibenzodiazepinones

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Experimental program
Comparison scheme
Effect test

Embodiment 1

[0114] mouse

[0115] Shank3b with exon 13-16 deletion originally obtained from the Jackson lab - / - Homozygous mice, and wild-type (WT) littermates were generated on the C57BL / 6J background and repeatedly backcrossed to the C57BL / 6J background over eight generations. The resulting Shank3 knockout (KO) mice were placed in a temperature- and humidity-controlled room in groups of the same genotype, and subjected to a 12-hour light-dark cycle (lights on from 7 am to 7 pm). Room temperature and humidity were recorded continuously in the storage room. Tested during the light phase. Food and water were available ad libitum. Shank3 knockout mice and their wild-type littermates were tested, N=10 mice per treatment group, in behavioral experiments at two months of age. Mice were housed in commercial plastic cages and experiments were performed in accordance with the requirements of the UK Animals (Scientific Procedures) Act 1986 (UK Animals (Scientific Procedures) Act). All exper...

Embodiment 2

[0145] AMO-01 was administered to a 23-year-old male with genetically confirmed Phelan McDermid syndrome.

[0146] The drug under study was prepared from a sterile bulk formulation containing 32.8 mg / mL (3.15% w / w) of active drug substance dissolved in sterile 0.9% saline solution to make etc. Infiltrated final dosing formulation. Each vial contained 70 mg of AMO-01 drug substance in 2.134 mL of formulation (plus 2% excess). This corresponds to a total drug content of 71.4 mg of active drug substance per 2.176 mL of formulation. AMO-01 is administered as a single dose by intravenous infusion over 6 hours for a total administered dose of 120 mg / m 2 .

[0147] Seizure frequency and visual evoked potentials (VEPs) were assessed along with clinical testing. VEPs provide a non-invasive technique that assesses the functional integrity of the brain's visual pathways from the retina to the visual cortex via optic nerve / optic radiation. VEP was recorded from the surface of the hea...

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Abstract

Methods for treating Phelan McDermid Syndrome (PMS) in subjects having the disorder or in subjects predisposed to develop the disorder via administration of farnesyl dibenzodiazepinone compounds are provided. The use of farnesyl dibenzodiazepinone compounds in the manufacture of a medicament for treating a subject having PMS is provided as well.

Description

Background technique [0001] Phelan McDermid Syndrome (PMS) is a neurodevelopmental disorder first mentioned in Watt et al. (1985) and later described in more detail by Phelan et al. (2001). [0002] Clinical features of PMS in humans include severe neonatal hypotonia (>97% of individuals), gross developmental delay (>98%), normal to accelerated growth (95%), loss to severe language delay (>98 %) and mildly deformed features (Phelan2008). Behaviorally, PhelanMcDermid individuals typically have low pain sensitivity, poor eye contact, stereotypic movement, decreased social functioning, hyperactivity, altered sensorimotor function, and aggressive behavior. Behavioral phenotypes may regress with age, manifesting as declines in social, motor, or mental function (Soorya et al., 2013). Physically, PMS may be accompanied by mild dysmorphic features and lymphedema. [0003] A key complication of PMS is epilepsy. Seizures (febrile and / or afebrile) occur in up to 41% of affec...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K31/5513A61P43/00
CPCA61K31/5513A61P43/00A61K9/0019
Inventor 迈克尔·斯内普帕特里夏·科格拉姆罗伯特·迪肯
Owner AMO PHARMA LTD