Solid compositions of triglycerides and uses thereof

一种固体组合物、三酸甘油酯的技术，应用在药物组合、酐/酸/卤化物有效成分、含有效成分的医用配制品等方向，能够解决影响、1型葡萄糖转运体缺乏疗效等问题

Inactive Publication Date: 2016-07-13
ULTRAGENYX PHARMA INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Therefore, tolerance to the oil form can be a dose-limiting toxicity or cause side effects, and reducing the dose that can be administered will adversely affect the effects on fatty acid oxidation disorder or deficiency (FAOD), adult glucose polymer disease, mitochondrial fat oxidation Deficiency, glycogen storage disease, mitochondrial myopathy, glucose transporter type 1 (GLUT1) deficiency syndrome or other related diseases

Method used

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  • Solid compositions of triglycerides and uses thereof
  • Solid compositions of triglycerides and uses thereof
  • Solid compositions of triglycerides and uses thereof

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0154] Table 5 demonstrates the high purity of exemplary triheptanoin oil samples and solid samples comprising triheptanoin oil according to the present invention.

[0155] Additionally, the stability of an exemplary solid composition comprising triheptanoin oil (solid sample 2 in Table 5) was tested. Measurements were taken after storage in double LDPE (low density polyethylene) bags in 60 cc induction sealed HDPE (high density polyethylene) bottles at 25°C / 60% RH (relative humidity) and the results are shown in Table 6. Measurements were also carried out at 40°C / 75%RH in 60cc induction sealed HDPE bottles after storage in double LDPE bags and the results are shown in Table 7.

[0156]

[0157]

[0158]

Embodiment 2

[0160] A single-dose study was performed for each group using a complete pharmacokinetic (PK) profile within 48 hours of dosing to determine when metabolites returned to baseline (with a one-week washout period between groups) and the relationship between gastrin and gallbladder Does the rapid release of the kinocin (CCK) hormone cause spasmodic contractions of the stomach. Blood samples were collected from each group at any time between 0 and 90 min plus this window if stomach pain was observed.

[0161]More specifically, animals (n=3 for each sex) were fasted before dosing and fed 4 hours after dosing. Blood samples were collected from pre-dose to 48 hours post-dose for PK analysis of triheptanoin and metabolites. As seen in the multiple dose study (Example 3), there were no major differences between males and females, so the data for all metabolites were combined.

[0162] exist Figures 1 to 21 , animals were administered a single oral gavage dose level of a triheptanoi...

Embodiment 3

[0175] A multiple dose study was performed on oil, powder and powder-ER as used in Example 2 and the results are shown below.

[0176] ·Powder group:

[0177] Day 1: Animal #6501 vomited approximately 1 hour after dosing

[0178] Day 3: Animal #6502 vomits

[0179] Day 3: Animal #6510 stomach pain observed

[0180] · Oil group:

[0181] Day 1: Animal #6501 vomited approximately 3 hours after dosing

[0182] Day 1: Animal #6516 vomited approximately 3 hours after dosing

[0183] Day 5: Stomach pain observed in animal #6509

[0184] ·Powder ER team:

[0185] No vomiting or stomach pain observed

[0186] enanthate

[0187] In conclusion, the results were as expected, triheptanoin released heptanoic acid. refer to Figure 22 , Triheptanoin is mainly metabolized in the liver into C7 fatty acids and ketone bodies, which are distributed to other tissues through circulation to provide energy sources. No triheptanoin values ​​in the oil panel. All triheptanoin oils were con...

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Abstract

The present invention includes solid compositions of triglycerides with one or more fatty acids, such as triheptanoin and glycerol phenylbutyrate, and therapeutic use thereof. The solid compositions can be prepared by spray-drying or other processes.

Description

[0001] Cross References to Related Applications [0002] This application claims the benefit of US Provisional Patent Application Serial No. 61 / 904,369, filed November 14, 2013, which is hereby incorporated by reference in its entirety. field of invention [0003] The present invention relates to solid compositions of triglycerides with one or more fatty acids, such as triheptanoin and phenylbutyrin, and their therapeutic use. Background of the invention [0004] Current formulations of triglyceride drugs such as triheptanoin are liquids with an oily consistency. Due to the physical and physiological properties of liquid dosage forms, there are several problems associated with them. First, liquid dosage forms, such as oils, are difficult to administer due to low miscibility with food or drink. Second, oil formulations are difficult to handle, carry and dispense. Furthermore, after administration, the oil composition is rapidly hydrolyzed and released in the stomach, leadi...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K31/19A61K31/20A61K31/23A61K31/216
CPCA61K31/20A61K9/143A61K9/146A61K9/1611A61K9/1652A61P21/02A61K31/23A61P25/08A61P3/00A61P9/10Y02A50/30A61K9/16A61K9/1664A61K31/201A61K31/202
Inventor J·克洛普G·莫里斯E·卡基斯S·琼格勒斯
Owner ULTRAGENYX PHARMA INC
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