JPWO2019234664A5 - - Google Patents
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- JPWO2019234664A5 JPWO2019234664A5 JP2020567815A JP2020567815A JPWO2019234664A5 JP WO2019234664 A5 JPWO2019234664 A5 JP WO2019234664A5 JP 2020567815 A JP2020567815 A JP 2020567815A JP 2020567815 A JP2020567815 A JP 2020567815A JP WO2019234664 A5 JPWO2019234664 A5 JP WO2019234664A5
- Authority
- JP
- Japan
- Prior art keywords
- compound
- composition
- syndrome
- composition according
- mitochondrial
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- 150000001875 compounds Chemical class 0.000 claims description 103
- 208000011580 syndromic disease Diseases 0.000 claims description 49
- 239000000203 mixture Substances 0.000 claims description 47
- 208000012268 mitochondrial disease Diseases 0.000 claims description 37
- 230000007812 deficiency Effects 0.000 claims description 36
- 208000001992 Autosomal Dominant Optic Atrophy Diseases 0.000 claims description 33
- 150000003839 salts Chemical class 0.000 claims description 20
- 125000004105 2-pyridyl group Chemical group N1=C([*])C([H])=C([H])C([H])=C1[H] 0.000 claims description 16
- 206010058799 Mitochondrial encephalomyopathy Diseases 0.000 claims description 16
- 125000001301 ethoxy group Chemical group [H]C([H])([H])C([H])([H])O* 0.000 claims description 16
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 16
- 208000006443 lactic acidosis Diseases 0.000 claims description 12
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims description 12
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 10
- 239000006186 oral dosage form Substances 0.000 claims description 9
- 206010003694 Atrophy Diseases 0.000 claims description 8
- 230000037444 atrophy Effects 0.000 claims description 8
- 239000003814 drug Substances 0.000 claims description 8
- 210000001328 optic nerve Anatomy 0.000 claims description 8
- 230000002265 prevention Effects 0.000 claims description 8
- 230000004064 dysfunction Effects 0.000 claims description 7
- 239000002552 dosage form Substances 0.000 claims description 6
- 201000010099 disease Diseases 0.000 claims description 4
- 125000004435 hydrogen atom Chemical group [H]* 0.000 claims description 4
- 229940124597 therapeutic agent Drugs 0.000 claims description 4
- 239000002775 capsule Substances 0.000 claims description 3
- 239000008187 granular material Substances 0.000 claims description 3
- 238000007918 intramuscular administration Methods 0.000 claims description 3
- 239000008184 oral solid dosage form Substances 0.000 claims description 3
- 229940100688 oral solution Drugs 0.000 claims description 3
- 229940100692 oral suspension Drugs 0.000 claims description 3
- 239000006187 pill Substances 0.000 claims description 3
- 239000007787 solid Substances 0.000 claims description 3
- 238000007920 subcutaneous administration Methods 0.000 claims description 3
- 230000000699 topical effect Effects 0.000 claims description 3
- 101000973439 Homo sapiens NADH-ubiquinone oxidoreductase 75 kDa subunit, mitochondrial Proteins 0.000 claims 3
- 102100022195 NADH-ubiquinone oxidoreductase 75 kDa subunit, mitochondrial Human genes 0.000 claims 3
- 208000001799 Hereditary Optic Atrophies Diseases 0.000 claims 2
- 208000006289 Rett Syndrome Diseases 0.000 claims 2
- 208000006136 Leigh Disease Diseases 0.000 claims 1
- 208000017507 Leigh syndrome Diseases 0.000 claims 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 description 27
- 238000000034 method Methods 0.000 description 17
- 201000006935 Becker muscular dystrophy Diseases 0.000 description 15
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- 201000006938 muscular dystrophy Diseases 0.000 description 11
- 201000002169 Mitochondrial myopathy Diseases 0.000 description 9
- 206010015037 epilepsy Diseases 0.000 description 9
- 208000004687 long chain acyl-CoA dehydrogenase deficiency Diseases 0.000 description 9
- 201000001119 neuropathy Diseases 0.000 description 9
- 230000007823 neuropathy Effects 0.000 description 9
- KDCGOANMDULRCW-UHFFFAOYSA-N 7H-purine Chemical compound N1=CNC2=NC=NC2=C1 KDCGOANMDULRCW-UHFFFAOYSA-N 0.000 description 6
- 206010003591 Ataxia Diseases 0.000 description 6
- 102000002666 Carnitine O-palmitoyltransferase Human genes 0.000 description 6
- 108010018424 Carnitine O-palmitoyltransferase Proteins 0.000 description 6
- 206010058892 Carnitine deficiency Diseases 0.000 description 6
- 102100022745 Laminin subunit alpha-2 Human genes 0.000 description 6
- 108700006159 Long-chain acyl-CoA dehydrogenase deficiency Proteins 0.000 description 6
- 108700000232 Medium chain acyl CoA dehydrogenase deficiency Proteins 0.000 description 6
- 206010033799 Paralysis Diseases 0.000 description 6
- 208000013234 Pearson syndrome Diseases 0.000 description 6
- 201000007737 Retinal degeneration Diseases 0.000 description 6
- 208000029560 autism spectrum disease Diseases 0.000 description 6
- 230000001684 chronic effect Effects 0.000 description 6
- 201000006815 congenital muscular dystrophy Diseases 0.000 description 6
- DDRJAANPRJIHGJ-UHFFFAOYSA-N creatinine Chemical compound CN1CC(=O)NC1=N DDRJAANPRJIHGJ-UHFFFAOYSA-N 0.000 description 6
- XBDQKXXYIPTUBI-UHFFFAOYSA-N dimethylselenoniopropionate Natural products CCC(O)=O XBDQKXXYIPTUBI-UHFFFAOYSA-N 0.000 description 6
- 208000023692 inborn mitochondrial myopathy Diseases 0.000 description 6
- JVTAAEKCZFNVCJ-UHFFFAOYSA-N lactic acid Chemical compound CC(O)C(O)=O JVTAAEKCZFNVCJ-UHFFFAOYSA-N 0.000 description 6
- 230000008774 maternal effect Effects 0.000 description 6
- 208000005548 medium chain acyl-CoA dehydrogenase deficiency Diseases 0.000 description 6
- 201000002697 mitochondrial DNA depletion syndrome Diseases 0.000 description 6
- 201000011548 mitochondrial DNA depletion syndrome 4b Diseases 0.000 description 6
- 230000002438 mitochondrial effect Effects 0.000 description 6
- 210000003205 muscle Anatomy 0.000 description 6
- 230000035772 mutation Effects 0.000 description 6
- 208000021090 palsy Diseases 0.000 description 6
- 230000000750 progressive effect Effects 0.000 description 6
- 208000016505 systemic primary carnitine deficiency disease Diseases 0.000 description 6
- 238000004519 manufacturing process Methods 0.000 description 4
- 201000002568 3-methylglutaconic aciduria with deafness, encephalopathy, and Leigh-like syndrome Diseases 0.000 description 3
- 208000004724 Ataxia neuropathy spectrum Diseases 0.000 description 3
- 208000014644 Brain disease Diseases 0.000 description 3
- 206010063292 Brain stem syndrome Diseases 0.000 description 3
- 102100027943 Carnitine O-palmitoyltransferase 1, liver isoform Human genes 0.000 description 3
- 101710120614 Carnitine O-palmitoyltransferase 1, liver isoform Proteins 0.000 description 3
- 101710108984 Carnitine O-palmitoyltransferase 1, muscle isoform Proteins 0.000 description 3
- 201000002929 Carnitine palmitoyltransferase II deficiency Diseases 0.000 description 3
- 208000010354 Coenzyme Q10 deficiency Diseases 0.000 description 3
- 102000008186 Collagen Human genes 0.000 description 3
- 108010035532 Collagen Proteins 0.000 description 3
- 208000002155 Cytochrome-c Oxidase Deficiency Diseases 0.000 description 3
- 102100028675 DNA polymerase subunit gamma-2, mitochondrial Human genes 0.000 description 3
- 206010011878 Deafness Diseases 0.000 description 3
- 206010011891 Deafness neurosensory Diseases 0.000 description 3
- 101710088194 Dehydrogenase Proteins 0.000 description 3
- 102000015782 Electron Transport Complex III Human genes 0.000 description 3
- 108010024882 Electron Transport Complex III Proteins 0.000 description 3
- 208000032274 Encephalopathy Diseases 0.000 description 3
- 208000032087 Hereditary Leber Optic Atrophy Diseases 0.000 description 3
- 101000804964 Homo sapiens DNA polymerase subunit gamma-1 Proteins 0.000 description 3
- 101000837415 Homo sapiens DNA polymerase subunit gamma-2, mitochondrial Proteins 0.000 description 3
- 101000595929 Homo sapiens POLG alternative reading frame Proteins 0.000 description 3
- 201000000639 Leber hereditary optic neuropathy Diseases 0.000 description 3
- 208000025121 Leukoencephalopathy with brain stem and spinal cord involvement-high lactate syndrome Diseases 0.000 description 3
- 108010027062 Long-Chain Acyl-CoA Dehydrogenase Proteins 0.000 description 3
- 102000018653 Long-Chain Acyl-CoA Dehydrogenase Human genes 0.000 description 3
- 102100021644 Long-chain specific acyl-CoA dehydrogenase, mitochondrial Human genes 0.000 description 3
- 208000005446 Lupus vulgaris Diseases 0.000 description 3
- 206010028289 Muscle atrophy Diseases 0.000 description 3
- 208000021642 Muscular disease Diseases 0.000 description 3
- 201000009623 Myopathy Diseases 0.000 description 3
- 201000002481 Myositis Diseases 0.000 description 3
- 208000010316 Myotonia congenita Diseases 0.000 description 3
- 102100035196 POLG alternative reading frame Human genes 0.000 description 3
- 208000008425 Protein deficiency Diseases 0.000 description 3
- LCTONWCANYUPML-UHFFFAOYSA-M Pyruvate Chemical compound CC(=O)C([O-])=O LCTONWCANYUPML-UHFFFAOYSA-M 0.000 description 3
- 208000002009 Pyruvate Dehydrogenase Complex Deficiency Disease Diseases 0.000 description 3
- 208000021886 Pyruvate carboxylase deficiency Diseases 0.000 description 3
- 208000009966 Sensorineural Hearing Loss Diseases 0.000 description 3
- 208000012658 Skin autoimmune disease Diseases 0.000 description 3
- 208000029033 Spinal Cord disease Diseases 0.000 description 3
- 102000046669 Surf-1 Human genes 0.000 description 3
- 108060007963 Surf-1 Proteins 0.000 description 3
- 239000002253 acid Substances 0.000 description 3
- 208000012130 acyl-CoA dehydrogenase deficiency Diseases 0.000 description 3
- 201000003554 argininosuccinic aciduria Diseases 0.000 description 3
- 210000000133 brain stem Anatomy 0.000 description 3
- 239000003610 charcoal Substances 0.000 description 3
- 229920001436 collagen Polymers 0.000 description 3
- 201000011474 congenital myopathy Diseases 0.000 description 3
- 229940109239 creatinine Drugs 0.000 description 3
- 230000002950 deficient Effects 0.000 description 3
- 206010012601 diabetes mellitus Diseases 0.000 description 3
- 208000035475 disorder Diseases 0.000 description 3
- 229940079593 drug Drugs 0.000 description 3
- 230000001815 facial effect Effects 0.000 description 3
- 230000009395 genetic defect Effects 0.000 description 3
- 208000007345 glycogen storage disease Diseases 0.000 description 3
- 230000010370 hearing loss Effects 0.000 description 3
- 231100000888 hearing loss Toxicity 0.000 description 3
- 208000016354 hearing loss disease Diseases 0.000 description 3
- 230000001771 impaired effect Effects 0.000 description 3
- 239000004310 lactic acid Substances 0.000 description 3
- 235000014655 lactic acid Nutrition 0.000 description 3
- 231100000518 lethal Toxicity 0.000 description 3
- 230000001665 lethal effect Effects 0.000 description 3
- ZIYVHBGGAOATLY-UHFFFAOYSA-N methylmalonic acid Chemical compound OC(=O)C(C)C(O)=O ZIYVHBGGAOATLY-UHFFFAOYSA-N 0.000 description 3
- 230000020763 muscle atrophy Effects 0.000 description 3
- 201000000585 muscular atrophy Diseases 0.000 description 3
- 230000003274 myotonic effect Effects 0.000 description 3
- 208000018360 neuromuscular disease Diseases 0.000 description 3
- 108010007425 oligomycin sensitivity conferring protein Proteins 0.000 description 3
- 230000003647 oxidation Effects 0.000 description 3
- 238000007254 oxidation reaction Methods 0.000 description 3
- 230000010627 oxidative phosphorylation Effects 0.000 description 3
- 235000019260 propionic acid Nutrition 0.000 description 3
- 102000004169 proteins and genes Human genes 0.000 description 3
- 108090000623 proteins and genes Proteins 0.000 description 3
- 230000006825 purine synthesis Effects 0.000 description 3
- 230000006824 pyrimidine synthesis Effects 0.000 description 3
- 201000006473 pyruvate decarboxylase deficiency Diseases 0.000 description 3
- 208000015445 pyruvate dehydrogenase deficiency Diseases 0.000 description 3
- IUVKMZGDUIUOCP-BTNSXGMBSA-N quinbolone Chemical compound O([C@H]1CC[C@H]2[C@H]3[C@@H]([C@]4(C=CC(=O)C=C4CC3)C)CC[C@@]21C)C1=CCCC1 IUVKMZGDUIUOCP-BTNSXGMBSA-N 0.000 description 3
- 208000001392 short chain acyl-CoA dehydrogenase deficiency Diseases 0.000 description 3
- JHLDJOBIUVJSTG-UHFFFAOYSA-N tdiq Chemical compound C1CNCC2=C1C=C1OCOC1=C2 JHLDJOBIUVJSTG-UHFFFAOYSA-N 0.000 description 3
- 208000014001 urinary system disease Diseases 0.000 description 3
- 208000019932 Aciduria Diseases 0.000 description 2
- 208000001769 Multiple Acyl Coenzyme A Dehydrogenase Deficiency Diseases 0.000 description 2
- 229910001651 emery Inorganic materials 0.000 description 2
- 230000003387 muscular Effects 0.000 description 2
- 208000000943 scapulohumeral muscular dystrophy Diseases 0.000 description 2
- 239000000725 suspension Substances 0.000 description 2
- 239000003826 tablet Substances 0.000 description 2
- 208000023434 Alpers-Huttenlocher syndrome Diseases 0.000 description 1
- 208000004986 Diffuse Cerebral Sclerosis of Schilder Diseases 0.000 description 1
- 241000206607 Porphyra umbilicalis Species 0.000 description 1
- 208000015362 glutaric aciduria Diseases 0.000 description 1
- 201000011540 mitochondrial DNA depletion syndrome 4a Diseases 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP18382397 | 2018-06-06 | ||
| EP18382397.0 | 2018-06-06 | ||
| PCT/IB2019/054696 WO2019234664A1 (en) | 2018-06-06 | 2019-06-06 | 5-[[4-[2-[5-(1-hydroxyethyl)pyridin-2-yl]ethoxy]phenyl]methyl]-1,3-thiazolidine-2,4-dione and its salts for use in the treatment of mitochondrial diseases |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021527049A JP2021527049A (ja) | 2021-10-11 |
| JP2021527049A5 JP2021527049A5 (https=) | 2022-06-14 |
| JPWO2019234664A5 true JPWO2019234664A5 (https=) | 2022-06-14 |
| JP7376934B2 JP7376934B2 (ja) | 2023-11-09 |
Family
ID=62716008
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020567815A Active JP7376934B2 (ja) | 2018-06-06 | 2019-06-06 | ミトコンドリア性疾患の処置における使用のための５－［［４－［２－［５－（１－ヒドロキシエチル）ピリジン－２－イル］エトキシ］フェニル］メチル］－１，３－チアゾリジン－２，４－ジオンおよびその塩 |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US11957670B2 (https=) |
| EP (2) | EP4545142A3 (https=) |
| JP (1) | JP7376934B2 (https=) |
| DK (1) | DK3801515T3 (https=) |
| ES (1) | ES3028538T3 (https=) |
| FI (1) | FI3801515T3 (https=) |
| HR (1) | HRP20250591T1 (https=) |
| HU (1) | HUE071482T2 (https=) |
| LT (1) | LT3801515T (https=) |
| PL (1) | PL3801515T3 (https=) |
| PT (1) | PT3801515T (https=) |
| SI (1) | SI3801515T1 (https=) |
| WO (1) | WO2019234664A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20130158077A1 (en) | 2011-12-19 | 2013-06-20 | Ares Trading S.A. | Pharmaceutical compositions |
| EP3917508A4 (en) | 2019-01-28 | 2022-12-21 | Mitochondria Emotion, Inc. | EAAT2 ACTIVATORS AND METHODS OF USE THEREOF |
| CA3127453A1 (en) | 2019-01-28 | 2020-08-06 | Mitochondria Emotion, Inc. | Trans-4-hydroxycyclohexyl phenyl amide mitofusin activators and methods of use thereof |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2012205798B2 (en) | 2011-01-10 | 2016-02-25 | Takeda Pharmaceutical Company Limited | Methods and drug products for treating Alzheimer's disease |
| EP3013371A4 (en) | 2013-06-26 | 2017-04-26 | Rett Syndrome Research Trust | Rett syndrome and treatments therefore |
| US10188639B2 (en) | 2014-01-15 | 2019-01-29 | Deuterx, Llc | Methods of treating neurological, metabolic, and other disorders using enantiopure deuterium-enriched pioglitazone |
| EP3125888B1 (en) | 2014-04-02 | 2018-05-23 | Minoryx Therapeutics S.L. | 2,4-thiazolidinedione derivatives in the treatment of central nervous system disorders |
| WO2017057587A1 (ja) | 2015-09-30 | 2017-04-06 | 株式会社ニコン | 露光装置、フラットパネルディスプレイの製造方法、及びデバイス製造方法 |
| WO2017083739A1 (en) * | 2015-11-13 | 2017-05-18 | The Trustees Of Columbia University In The City Of New York | A method for predicting a subject's response to valproic acid therapy |
| CN106202285A (zh) | 2016-06-30 | 2016-12-07 | 北京百度网讯科技有限公司 | 搜索结果展示方法和装置 |
| EP3548026B1 (en) | 2016-12-01 | 2021-01-20 | Minoryx Therapeutics S.L. | 5-[[4-[2-[5-(1-hydroxyethyl)pyridin-2-yl]ethoxy]phenyl]methyl]-1,3-thiazolidine-2,4-dione for treating nonalcoholic fatty liver disease |
| EP3559010B1 (en) | 2016-12-23 | 2022-06-08 | Minoryx Therapeutics S.L. | Process for preparing 5-[[4-[2-[5-(1-hydroxyethyl)-2-pyridinyl]ethoxy]phenyl]methyl]-2,4-thiazolidinedione and salts thereof |
| EA202092954A1 (ru) | 2018-06-06 | 2021-04-08 | Минорикс Терапьютикс С.Л. | Применение 5-[4-[2-[5-ацетилпиридин-2-ил]этокси]бензил]-1,3-тиазолидин-2,4-диона и его солей |
| BR112020024917A2 (pt) | 2018-06-06 | 2021-03-09 | Minoryx Therapeutics S.L. | Método de administrar uma quantidade terapeuticamente eficaz de 5-[[4-[2-[5-(1-hidroxietil)piridin-2-il]etoxi]fenil]metil]-1,3-tiazolidina-2,4-diona |
-
2019
- 2019-06-06 FI FIEP19742480.7T patent/FI3801515T3/fi active
- 2019-06-06 EP EP25162244.5A patent/EP4545142A3/en active Pending
- 2019-06-06 WO PCT/IB2019/054696 patent/WO2019234664A1/en not_active Ceased
- 2019-06-06 US US16/972,375 patent/US11957670B2/en active Active
- 2019-06-06 SI SI201930937T patent/SI3801515T1/sl unknown
- 2019-06-06 ES ES19742480T patent/ES3028538T3/es active Active
- 2019-06-06 HR HRP20250591TT patent/HRP20250591T1/hr unknown
- 2019-06-06 PL PL19742480.7T patent/PL3801515T3/pl unknown
- 2019-06-06 HU HUE19742480A patent/HUE071482T2/hu unknown
- 2019-06-06 EP EP19742480.7A patent/EP3801515B1/en active Active
- 2019-06-06 JP JP2020567815A patent/JP7376934B2/ja active Active
- 2019-06-06 DK DK19742480.7T patent/DK3801515T3/da active
- 2019-06-06 PT PT197424807T patent/PT3801515T/pt unknown
- 2019-06-06 LT LTEPPCT/IB2019/054696T patent/LT3801515T/lt unknown
-
2024
- 2024-04-12 US US18/633,922 patent/US20250064791A1/en active Pending
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