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Edasalonexent dosing regimen for treating muscular dystrophy

A technology for muscular dystrophy and drug delivery, which can be used in pharmaceutical formulations, drug combinations, drug delivery, etc., and can solve problems such as uncontrolled inflammation and pathology

Pending Publication Date: 2020-06-19
CATABASIS PHARMA
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

Although these pathways are essential for the survival and adaptation of organisms, chronic activation of the NF-κB system leads to uncontrolled inflammatory pathology

Method used

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  • Edasalonexent dosing regimen for treating muscular dystrophy
  • Edasalonexent dosing regimen for treating muscular dystrophy
  • Edasalonexent dosing regimen for treating muscular dystrophy

Examples

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example

[0137] The present disclosure is further illustrated by the following examples, which should not be construed to limit the scope or spirit of the present disclosure to the specific procedures described herein. It should be understood that the examples are provided for the purpose of illustrating certain embodiments and are not intended thereby to limit the scope of the disclosure.

example 1

[0138] Example 1: Evaluation of the Pharmacokinetics of Ednaxen Dosing Regimen in C57BL / 6 Mice

[0139] Ednaxen was administered to C57BL / 6 mice in the diet and / or by oral gavage. Pharmacokinetic (PK) modeling was used to identify the appropriate dosing amount and timing for the doses of 67 mg / kg / day and 100 mg / kg / day that have been tested in idenaxan human clinical trials. Specifically, mice were administered 1.5% idenaxen in the diet; 450 mg / kg / day orally delivered as a single daily dose; 450 mg / kg / day orally delivered as three equal doses; or as two 150 600 mg / kg / dose was delivered orally with a 300 mg / kg / dose and a 300 mg / kg / dose. For the dosing regimen of three daily doses, doses are delivered at 7:00am, noon and 5:00pm.

[0140] The dosing regimen for each group is depicted in Table 1. Each group contained 12 mice.

[0141] Table 1

[0142]

[0143] For animals administered idenaxen in the diet, idenaxen concentrations were measured by LC / MS / MS every 4 hours for ...

example 2

[0150] Example 2: Evaluation of idenaxen dosing regimen in mdx mice

[0151]Young mdx mice were treated with different idenaxan dosing regimens for four weeks. The mdx mouse is a useful and well-accepted animal model for the study of Duchenne muscular dystrophy (DMD) (Mann et al. (2001) Proceedings of the National Academy of Sciences of the United States of America (PROC.NATL.ACAD.SCI.) 98(1):42-7). mdx mice lack expression of full-length dystrophin due to an inherited mutation within the dystrophin gene.

[0152] Ednaxen was administered to mice in the diet and / or by oral gavage. Specifically, ednaxen was administered to mice at 1% in their diet; 450 mg / kg / day was orally delivered in a single daily dose; 450 mg / kg / day was orally delivered in three equal doses; and / or 600 mg / kg / day was delivered orally in two 150 mg / kg / doses and one 300 mg / kg / dose. For the dosing regimen of three daily doses, doses are delivered at 7:00am, noon and 5:00pm.

[0153] The dosing regimen for ...

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Abstract

The invention provides methods and compositions for treating a muscular dystrophy, e.g., Duchenne muscular dystrophy (DMD), in a subject, with a fatty acid acetylated salicylate, e.g., edasalonexent,effective to achieve a threshold plasma concentration of the fatty acid acetylated salicylate in the subject, e.g., a threshold plasma concentration of at least about 20 ng / ml for least 12 hours in a24 hour period.

Description

[0001] Cross References to Related Applications [0002] This application claims priority and benefit to U.S. Provisional Patent Application No. 62 / 581,981, filed November 6, 2017, the contents of which are hereby incorporated by reference in their entirety. technical field [0003] The present invention relates to the field of muscular dystrophy, in particular for the treatment of Duchenne muscular dystrophy (Duchennemuscular dystrophy, DMD), in particular for the use of edasalonexent (a fatty acid salicylate conjugate ) dosing regimen for the treatment of DMD. Background technique [0004] Duchenne muscular dystrophy (DMD) is a rare, severe, life-threatening neuromuscular degenerative disease with X-linked recessive inheritance. DMD caused by mutations in the dystrophin gene is characterized by the absence or minimal presence of functional dystrophin, resulting in a progressive deterioration of skeletal muscle function from early childhood. Despite improvements in the s...

Claims

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Application Information

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IPC IPC(8): A61K31/166A61K31/235C07C69/86C07C69/94
CPCA61K31/166A61K9/4816A61P21/00A61K9/4858A61K9/4866A61K9/0053
Inventor 安德鲁·J·尼克尔斯迈克尔·珀尔曼
Owner CATABASIS PHARMA
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